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Sarepta Therapeutics (SRPT) Q1 2023 Earnings Name Transcript

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers almost about the proportion of sufferers initially receiving entry? Thanks.

Doug IngramChief Government Officer

I need to say the broadest of strokes and you’ll comply with up on lacking something. To start with, the conversations have gone very effectively. We have been in dialogue with payers concerning the potential for SRP-9001 for a few years now. And I can — , going again so far as mid-2018, Dallan, myself, and others had been assembly with payers.

The — our accident reimbursement crew have been assembly with payers considerably. The quantity of proof that now we have that helps the conclusion that 9001 is a useful remedy for youths and goes to do lots of good may be very, very sturdy. So, these discussions have gone very effectively. With that mentioned, let me be very clear, after all, , as is the case with uncommon illness therapies proper now, truly, reimbursement is a sophisticated and difficult factor.

The excellent news for all of us is that 9001 goes to be launched by Sarepta. And, , on the threat of sounding a bit conceited, in my opinion, there isn’t any crew higher than this Sarepta crew to serve these sufferers, work intelligently with payers, and achieve entry for this remedy for these children as quickly as attainable. And I’m fairly assured that is going to happen. I am fairly assured payers are going to do the proper factor, and positively I consider that they will do the proper factor within the context of accelerated approval, which is, from our perspective, the method that — that one ought to be taking with respect to 9001 given the info that now we have in entrance of us.

Dallan MurraySenior Vice President, Chief Business Officer

Yeah, and I feel — I feel Doug’s actually lined it. There are, as we mentioned in — within the opening remarks, actually constructive, nice dialogue happening with the payers. And proper now, we’re — , we’re — we’re — previous to launch, they’re asking nice questions on timing and the affected person inhabitants. And I feel, extra particularly, Colin, to your query, no matter when the insurance policies are put in place, the payers are going to have a look at every affected person on a case-by-case foundation.

And so, the crew, as Doug mentioned, is able to handle — able to handle that proper from day one.

Doug IngramChief Government Officer

We’re skilled and battle-hardened crew.

Operator

Thanks. Our subsequent query comes from the road of Judah Frommer with Credit score Suisse. Your line is now open.

Judah FrommerCredit score Suisse — Analyst

Hello, thanks for taking the query. One other one type of from the payer angle. Any concept if there could possibly be sort of a distinction in RAM for sufferers which are, , I suppose, naïve to RNA therapies versus these which are — which are on the PMOs gaining entry? After which, any indications round potential value-based funds, given such as you mentioned, that is going to be the most important gene remedy launch ever? So, from a price perspective, might there be any, I suppose, attention-grabbing dynamics to — to the time of funds? Thanks.

Doug IngramChief Government Officer

Yeah, because it pertains to the primary query, I do not assume there’s going to be a distinction in RAM. I feel that is — for individuals who are amenable to this remedy, which at launch we’re profitable, our BLA can be ambulant sufferers, excluding a really slim vary of mutations who’re rh74 detrimental. I feel this remedy goes to be terribly essential. And I feel that there is going to be an equal ramp, whether or not you are naïve or not naïve to — to the PMO.

And in reality, now we have dosed sufferers which have been on the PMO and remained on the PMO post-dose. So, now we have good knowledge that helps that. On value-based agreements and the like, I am not at a spot proper now the place we’ll focus on these points but. I’ll inform you that now we have finished an unlimited quantity of labor, about which I’m extraordinarily impressed and proud to border the worth proposition and the pharmacoeconomic mannequin for one-time therapies like SRP-9001.

And our method to the payer neighborhood pricing value-based agreements and the like can be contained in the body of that worth framework itself. And the one factor I’ll inform you, qualitatively a minimum of, and can on the proper time speak quantitatively, is that the worth that this remedy will carry to Duchenne sufferers goes to be considerably higher than the price of this remedy to the healthcare system, which is what all of us ought to need.

Operator

Thanks. Our subsequent query comes from the road of Salveen Richter with Goldman Sachs. Your line is now open.

Salveen RichterGoldman Sachs — Analyst

Good afternoon. Thanks for taking my query. With regard to manufacturing, are you able to simply present us with some particulars of the place you stand on stock as you look to this launch, after which, the breadth of your manufacturing relationships with a purpose to handle the availability that is required over a minimum of the primary yr or second — or first two years of launch right here?

Doug IngramChief Government Officer

Positive, so we’re constructing stock, as we communicate, to be prepared for launch. So, that is clearly an ongoing exercise with Catalent. It is a excessive precedence for us and, fortuitously, additionally a excessive precedence for Catalent, so we’re in nice form there. And that is nice for launch and we’re in nice form there.

If you happen to look down the street long term, after all, we even have our relationship with Thermo Fisher. We now have a whole stand-alone website with Thermo Fisher. One of many selections we made in reference to our BLA submission was to not attempt to get two websites accepted on the identical time. The complexity related to that may have created a big threat of delay.

And, , I feel, as we have mentioned 1,000,000 occasions, , delay isn’t one thing that sufferers with Duchenne can — can have. So, what we are going to do post-launch is figure with the division to get the Thermo Fisher website up and working and certified as effectively. The excellent news is at launch, this our — our website with Catalent and our suites with Catalent is enough to — to launch the remedy and serve the neighborhood.

Operator

Thanks. Our subsequent query comes from the road of Gil Blum with Needham and Firm. Your line is now open.

Gil BlumNeedham and Firm — Analyst

Good afternoon, everybody, and thanks for taking our query. Doug, in your earlier feedback, you talked about that the corporate is planning to start out a number of the different research together with on nonambulatory sufferers and the clearing of antibody research. What about planning a examine in youthful sufferers? I am assuming that, , as with all gene remedy, youthful is normally higher. Thanks.

Doug IngramChief Government Officer

Yeah, effectively, let me — let me touch upon that final piece first. This can be very essential that we get to youthful sufferers as effectively, I need to be very clear. However I need to be additionally clear that, from our perspective, there isn’t any place throughout this journey of Duchenne the place the intervention of a remedy, like 9 years or one, that may restore purposeful dystrophin to sufferers, will not be useful. There is no such thing as a — there is no little one that is past worth, that is essential to recollect.

So, in the event you’re 19 years outdated and you have been in a wheelchair for 5 years, you might be as beneficial to us as a really younger little one. So, that is — in order that’s why we’re very targeted on the nonambulant aspect, however we’re targeted on the very younger as effectively. We have already dosed children which are down to 3 years outdated. Louise would possibly need to touch upon different plans.

We now have that a lot youthful youngsters as effectively.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. We have got three-year-olds in our 103 examine. After which, we’re additionally planning a further examine together with Roche to dose even youthful than the three-year-olds, and that may start briefly time period.

Operator

Thanks. Our subsequent query comes from the road of Tazeen Ahmad with Financial institution of America. Your line is now open.

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Hello, good afternoon. Thanks for taking my questions. I suppose, Ian, I simply needed to make clear a remark that you simply made concerning write-offs of sure batches of the corporate’s merchandise that weren’t assembly high quality specs. Which merchandise had been they, are you able to share? And have you ever resolved that concern? After which, additionally, possibly simply to comply with up on feedback that Doug made concerning inspections being accomplished, are you able to additionally verify whether or not or not FDA has any feedback on the inspection? And if they’ve, have they been resolved? Thanks.

Ian EstepanChief Monetary Officer

Positive, I will take the primary one first, that was concerning the PMO. That is simply a part of our regular manufacturing course of. If you happen to look again over a number of quarters, that is occurred. So, , that is simply one thing that is a part of the conventional manufacturing course of.

And, , there’s some batches that do not meet specs and now we have to write down it off however nothing to be involved and ought to be anticipated to proceed to go far.

Doug IngramChief Government Officer

After which, because it pertains to the second query, the entire inspections are accomplished, and any of the observations have been solely glad. So, we’re in nice form from an inspection perspective.

Operator

Thanks. Our subsequent query comes from the road of Danielle Brill with Raymond James. Your line is now open.

Danielle BrillRaymond James — Analyst

Hello, guys, thanks — thanks a lot. I even have a few questions on EMBARK. Curious what p.c of sufferers have accomplished that trial, simply attempting to gauge how back-end load an enrollment was. After which, additionally, how continuously do you measure NSA? And the way are lacking knowledge imputed? Thanks.

Doug IngramChief Government Officer

I will take the final half however the first half, of the sufferers.

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Yeah. So, the examine was utterly enrolled final fall by way of the — so we are able to count on the one-year endpoint to shut out this fall with the — the examine report early subsequent yr. The NSA — these are the first endpoint for the NSA is at one yr. However we measure it at intervals previous to that, actually, previous to whenever you’re at endpoint.

Doug IngramChief Government Officer

One factor on the workers plan after we announce the outcomes.

Operator

Thanks. Our subsequent query comes from the road of Mike Ulz with Morgan Stanley. Your line is now open.

Mike UlzMorgan Stanley — Analyst

Hey, guys, thanks for taking the query. Simply one other one on the 9001 launch, do you could have a way of what number of sufferers would possibly need to swap from a few of your PMO therapies, and the way do you propose to handle that? Thanks.

Doug IngramChief Government Officer

So, let me say two issues. First, we do not anticipate at launch a big impression on our present PMO income from a income perspective. In the long term, one would possibly — one ought to assume some vital cannibalization. It will not occur early on.

To the extent {that a} affected person needs to change from PMO to have entry to the gene remedy, we are going to embrace that and be, — I am excited for that.

Operator

Thanks. Our subsequent query comes from the road of Neena Bitritto-Garg with Citi. Your line is now open.

Neena Bitritto-GargCiti — Analyst

Hey, guys, thanks for taking my query. I simply needed to return to a number of the questions on the payer dialog to date. Are you able to simply inform us slightly bit about whether or not any of your conversations to date have instructed that payers could wait to really concern protection determinations till they see the EMBARK knowledge? And in that case, how they could take into consideration, , sort of restrictions and the protection coverage post-EMBARK? Thanks.

Doug IngramChief Government Officer

We intend to launch this remedy and work with payers to get entry to this remedy instantly. I’d remind people that now we have three accepted therapies right this moment, Exondys, Vyondys, and Amondys. All of them had been accepted for the accelerated approval pathway. And the crew has finished, in my view, a fully sensible job of working with payers to make sure fast entry for sufferers who’re amenable to these three therapies.

We’ll take that very same execution focus. We are going to apply it to 9001. And we anticipate that payers are going to reply effectively given the robustness of our knowledge and are going to have entry instantly.

Operator

Thanks. Our subsequent query comes from the road of Tim Lugo with William Blair. Your line is now open.

Tim LugoWilliam Blair and Firm — Analyst

Yeah, thanks for taking the query. Better of luck, clearly. Are you able to discuss the way you view capital deployment in a post-approval position? You clearly have lots of research we have talked about; you need to construct the label out your self, these in limb-girdle. And I consider your pipeline is described as 40-compound deep.

So, that appears like lots of R&Ds. And I might love to listen to your ideas round that, which might be cash extraordinarily effectively spent however possibly if that additionally influences your ideas round pricing of 9001.

Doug IngramChief Government Officer

Effectively, I’ll say two issues. I have to flip this to Ian who can remark about capital deployment in additional basic sense. And let’s first — we’re going to considerably deal with analysis and improvement and the like, after all. And we are going to proceed to do this deep into the long run.

That however, we are going to — our present plans, assuming that we’re accepted and our plans come to fruition, would have us being worthwhile subsequent yr. Because it pertains to the pricing for 9001, the pricing of 9001 will happen within the context of the — within the pharmacoeconomic fashions we use to make sure that the worth is acceptable for that remedy and that — as I mentioned earlier than, that the worth delivered to the sufferers and their lives from this remedy is far higher than the fee to the healthcare system. However past that, Ian, you need to touch upon the capital deployment plans?

Ian EstepanChief Monetary Officer

Yeah, no, I feel you are precisely proper, we’re clearly going to proceed to spend money on R&D, however we’re additionally going to be targeted on profitability and comply with metrics that may — will information that and guarantee returns for our shareholders. However clearly, as , investing in our R&D goes to result in continued development. And we’ll be targeted on transferring packages ahead which have excessive likelihood of success primarily based on the info which we generate. We additionally assume the market situations proper now lend itself to, , being ready to accomplice or purchase applied sciences that we expect are scientific breakthroughs as we proceed to construct out our pipeline.

So, we’ll be very according to the method that we have used beforehand. It is clearly put us ready the place we’re one of many main rising biotech corporations, and we’re not going to stray from that.

Operator

Thanks. Our subsequent query comes from the road of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Kristen KluskaCantor Fitzgerald — Analyst

Hello, good afternoon, and finest needs to your crew this month. Are you able to speak in regards to the newest because it pertains to taking a look at a number of the methods you are seeking to handle pretreatments for these with preexisting antibodies to AAV? I noticed that you simply’re presenting with Hansa some preclinical knowledge at AACT. And the agenda was actually simply launched about an hour in the past. So are you able to discuss a few of these efforts, please?

Doug IngramChief Government Officer

Yeah, so there are two approaches, and if there’s extra to say past this, we are able to have a look. Broadly, there are two approaches that we’re taking proper now. One is, after all, with our accomplice, Hansa, in imlifidase to cleave and due to this fact take away antibodies that stand in the best way of a kid getting 9001. And the opposite is utilizing apheresis to clear antibodies.

So, something you need to add to that, Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

We now have robust preclinical knowledge with each approaches. And as Doug talked about, we’re planning to start out two medical research on each approaches. So, this has been essential to us to make it possible for we are able to serve your complete neighborhood.

Operator

Thanks. Our subsequent query comes from Hartaj Singh with Oppenheimer and Firm. Your line is now open.

Hartaj SinghOppenheimer and Firm — Analyst

Nice, thanks. Thanks for the query. I simply received a fast query on — on the VOYAGENE examine in LGMD. , assuming you get that part 3 began with commercially consultant materials by the top of the yr, how a lot perception will the part 2 and the part 3 provide you with into the opposite LGMDs? I imply, might you progress sooner? , might regulators be amenable to taking a look at them holistically versus, , very individually? After which, how simple will or not it’s to scale the manufacturing for all of the others? Thanks for the query.

Doug IngramChief Government Officer

, after I say broadly, — one’s seen public shows from Dr. Peter Marks, you will know that his — his final aim, his long-term aim is to get to a spot the place you may construct remedy upon remedy. And significantly, in the event you’re utilizing the identical passage which we’re in reference to the LGMDs that you have to be studying from every after which having the ability to vastly shorten the timeline. I feel {that a} type of that may happen with our limb girdles, however we’re within the early days of limb girdles, so it will not be absolutely shaped like that, as we’re — , we’re transferring by means of.

We do get vital worth and studying from every of those packages that we apply to the following one. The limb girdles are benefiting enormously from 9001. Keep in mind, most of our limb girdles, the bulk a minimum of, use the identical promoter as 9001, they usually all use the identical capsid, rh74. So, there may be this virtuous cycle the place we ought to have the ability to begin transferring sooner and sooner over time.

It’ll take a while to do this. I’d say — and eventually, I might say on manufacturing, we positively profit from prior data as we transfer ahead. However every of those packages is its personal program and requires some bespoke components, together with, as an example, a lot of the assay work. Among the asset work could be very translatable.

However lots of this assay work is bespoke from — from program to program. And so, that does take a while, and it’ll take a while with respect to a few of these girdles.

Operator

Thanks. Our subsequent query comes from the road of Debjit Chattopadhyay with Guggenheim. Your line is now open.

Debjit ChattopadhyayGuggenheim Companions — Analyst

Hey, good afternoon and thanks for taking my query. Doug, I simply needed to make clear one of many feedback you made in your ready remarks. You introduced up EMBARK within the context of the result. Might you make clear and body that query once more?

Doug IngramChief Government Officer

Oh, I feel — oh, sure, one of many — the problems that we simply must — to debate on the advisory committee is that — EMBARK, which is our proposed confirmatory trial and clearly wants to finish and full on time. And so, one of many, clearly, cheap questions one would pose is, , are you assured that if we provide you with an approval now and on an accelerated foundation that EMBARK will, in reality, full, that there will not be one thing in regards to the approval of this remedy that may someway affect the flexibility to efficiently full EMBARK. As , EMBARK truly was absolutely enrolled as of final yr — September of final yr. So, I feel relative to different accelerated approval therapies, we’re in a very advantageous, sensible place with respect to the completion of our confirmatory trial.

Operator

Thanks. Our subsequent query comes from the road of Joseph Schwartz with SVB Securities. Your line is now open.

Joseph SchwartzSVB Securities — Analyst

Hello, everybody. Thanks very a lot. Since we’re so near the panel, I used to be questioning when you have seen the FDA’s briefing paperwork at this level and in the event you may give us your gestalt about their tone in order that we could be extra ready for what to anticipate.

Doug IngramChief Government Officer

As I mentioned, we’re 10 days away and counting from the advisory committee assembly. I need to be very clear about this. What we’re all doing collectively proper now’s terribly critical. It is essential to us and it is essential to our buyers, however it’s vastly extra critical and essential to the sufferers residing with Duchenne muscular dystrophy.

That is actually a possible life-or-death concern for them. So, in regard to that, we’re going to be mission-driven, and what which means to us is we’ll keep very targeted on prosecuting our BLA, making ready for our adcom. We’re not going to debate the result or the briefing books or the regulatory course of proper now. We will do a — we’ll prepare for and, in my humble opinion, we’ll do a superb job.

So, I am placing lots of strain on you, Louise. We will do a superb job of presenting what I consider to be the wealth of proof that helps the conclusion that 9001 and the quantities made by this remedy within reason more likely to predict medical profit. So, in gentle of that and with all respect and apologies for not answering the query, I am not going to reply questions in regards to the regulatory course of by means of the advisory — advisory committee till after Could twelfth. After which, we’ll all come collectively, and I’ll be thrilled to speak about all of those points with you.

Operator

Thanks. Our subsequent query comes from the road of Zhi Shu with Berenberg. Your line is now open.

Shu ZhiqiangBerenberg Capital Markets — Analyst

Good afternoon. Thanks for taking my questions. Perhaps going again to the manufacturing ramp. Doug, are you able to set some expectations on what number of sufferers do you propose to deal with for 9001? And secondly, on 9003 limb girdle program, clearly, you commented the part 3 will begin within the second half of the yr.

Is there any chance for accelerated approval pathway for this program as effectively? Thanks very a lot.

Doug IngramChief Government Officer

So, answering the second query first. Finally, we — we are going to suggest a type of accelerated approval for 9003. That is an extremely, ultra-rare illness. We’re — the 9003 makes non-native protein, the absence of which is the only real and unique explanation for the demise and in the end the loss of life of sufferers that endure from 9003.

So, actually, if we see nice ends in the confirmatory trial within the part 3 that we’re beginning, we’re going to suggest an accelerated approval pathway. As to the primary query, I am not going to supply numbers on — numbers of sufferers apart from to say our aim is to deal with each affected person that is amenable to this remedy as shortly as moderately attainable. And so, we’re making ready ourselves to have a strong launch.

Operator

Thanks. Our subsequent query comes from the road of Gavin Clark-Gartner with Evercore ISI. Your line is now open.

Gavin Clark-GartnerEvercore ISI — Analyst

Hey, thanks for becoming me in. Simply to comply with up on the LGMD2E query, what’s your base case assumption for the part 3 major endpoint and trial design? When will you align with the FDA on this?

Doug IngramChief Government Officer

So, the brief reply is we’ll begin that examine earlier than the top of this yr. We are going to align with the company on that alongside the best way. We have got work to do there. Clearly, we have been prioritizing 9001 proper now.

The purposeful endpoint would probably be a type of NSAA, if I am not mistaken, it has been tailored for limb-girdle. However we’ll clearly even be taking a look at expression and security with respect to 9003.

Operator

Thanks. Our subsequent query comes from the road of Ritu Baral with TD Cowen. Your line is now open.

Unknown speaker

Hello, guys. That is [Inaudible] on for Ritu right this moment. Are you able to verify the timing of EMBARK top-line knowledge? Have there be any adjustments to that? After which, any updates to your long-term income steering of $4 billion in 2025? How will you be revising this if 9001 is accepted this month?

Doug IngramChief Government Officer

Our — our forecast assume approval this month. So, the reply with the primary query was —

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

So far as the readout.

Doug IngramChief Government Officer

There’s been no change within the EMBARK readout. The EMBARK was absolutely enrolled as of September of final yr. It is going swimmingly. It is clearly blinded.

So, it is being executed swimmingly and we anticipate prime line actually on the finish of this yr, early subsequent yr.

Operator

Thanks. Our subsequent query comes from the road of Brian Skorney with Baird. Your line is open.

Brian SkorneyRobert W. Baird and Firm — Analyst

Hey, good afternoon. Thanks for taking my query. I suppose sort of leaping off on a number of the questions across the capacity to efficiently conclude EMBARK that you simply talked about, are you able to simply speak to us about examine conduct? And is there any threat that an accelerated approval might — might put examine conduct in danger? , I feel you’ve got mentioned earlier than that the majority U.S. sufferers have truly had their final go to.

So, might you simply sort of evaluate what you’d say to somebody questioning whether or not or not you had been going to have the ability to efficiently keep examine conduct on EMBARK upon getting commercially accessible 9001 underneath AA?

Doug IngramChief Government Officer

There isn’t any threat. Zero. The examine was enrolled — absolutely enrolled September of final yr. The entire children partially 1 of the examine have obtained their doses.

The children on crossover are being dosed whilst we communicate. Any child that has but to be dosed can be dosed within the subsequent few months, most. So, the march goes very effectively. We’ll learn out on time no matter whether or not we obtain accelerated approval.

There isn’t any — there is no cheap threat to that examine.

Operator

Thanks. Our subsequent query comes from the road of Anupam Rama with J.P. Morgan. Your line is now open.

Anupam RamaJPMorgan Chase and Firm — Analyst

Hey, guys, thanks a lot for taking a query. On SRP-9003, might you see any parts of the VOYAGENE knowledge this yr could also be beginning forward of the industrial materials part 3? I feel the PR solely dedicated to, like, enrollment completion however not knowledge itself probably, however simply questioning if we might see one thing this yr.

Doug IngramChief Government Officer

Yeah, I am sorry — I am sorry for leaping in there, Anupam. We — it is not clear whether or not we would have knowledge this yr, so we’ll — we’ll replace you later within the yr on that.

Operator

Thanks. I’d now like handy the convention again over to Doug Ingram for closing remarks.

Doug IngramChief Government Officer

Effectively, thanks, all, very a lot for becoming a member of us this night. And thanks in your questions and thanks for accommodating, , my request that we not speak in regards to the regulatory course of or the advisory committee as a lot as you need to ask questions on that and as a lot as I need to reply questions on that if I’ll be sincere and direct with you. I stay up for — clearly, all of us stay up for Could 12. That advisory committee assembly is an unbelievably essential second for sufferers residing with Duchenne.

I am terribly assured within the capacity of this crew to current the info effectively and to border it brilliantly, as I mentioned earlier than. After which I stay up for coming again collectively thereafter and discussing the place we’re and the trail ahead. So, thanks, all. Have a beautiful night.

Operator

[Operator signoff]

Length: 0 minutes

Name members:

Mary JenkinsAffiliate Director, Investor Relations

Doug IngramChief Government Officer

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Dallan MurraySenior Vice President, Chief Business Officer

Ian EstepanChief Monetary Officer

Colin BristowUBS — Analyst

Gena WangBarclays — Analyst

Brian AbrahamsRBC Capital Markets — Analyst

Judah FrommerCredit score Suisse — Analyst

Salveen RichterGoldman Sachs — Analyst

Gil BlumNeedham and Firm — Analyst

Tazeen AhmadFinancial institution of America Merrill Lynch — Analyst

Danielle BrillRaymond James — Analyst

Mike UlzMorgan Stanley — Analyst

Neena Bitritto-GargCiti — Analyst

Tim LugoWilliam Blair and Firm — Analyst

Kristen KluskaCantor Fitzgerald — Analyst

Hartaj SinghOppenheimer and Firm — Analyst

Debjit ChattopadhyayGuggenheim Companions — Analyst

Joseph SchwartzSVB Securities — Analyst

Shu ZhiqiangBerenberg Capital Markets — Analyst

Gavin Clark-GartnerEvercore ISI — Analyst

Unknown speaker

Brian SkorneyRobert W. Baird and Firm — Analyst

Anupam RamaJPMorgan Chase and Firm — Analyst

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Sarepta Therapeutics (SRPT -1.14%)
Q1 2023 Earnings Name
Could 02, 2023, 4:30 p.m. ET

Contents:

  • Ready Remarks
  • Questions and Solutions
  • Name Individuals

Ready Remarks:

Operator

Good afternoon and welcome to the Sarepta Therapeutics first-quarter 2023 earnings name. Right now, all members are in a listening mode. After the speaker’s presentation, there can be a question-and-answer session. [Operator instructions] Once more, as a reminder, right this moment’s program is being recorded.

Right now, I will flip the decision over to Mary Jenkins, affiliate director, investor relations. Please go forward.

Mary JenkinsAffiliate Director, Investor Relations

Thanks, Shannon, and thanks all for becoming a member of right this moment’s name. Earlier this afternoon, we launched our monetary outcomes for the first-quarter 2023. The press launch is accessible on our web site at sarepta.com, and our 10-Q was filed with the Securities and Change Fee this afternoon. Becoming a member of us on the decision right this moment are Doug Ingram, Ian Estepan, Dallan Murray, and Dr.

Louise Rodino-Klapac. After our formal remarks, we’ll open the decision for Q&A. I might like to notice that in this name, we’ll be making plenty of forward-looking statements. Please take a second to evaluate our slide on the webcast which comprises our forward-looking statements.

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These forward-looking statements contain dangers and uncertainties, a lot of that are past Sarepta’s management. Precise outcomes might differ from these forward-looking statements and any such dangers can materially and adversely have an effect on the enterprise, the outcomes of operations, and buying and selling costs for Sarepta widespread inventory. For an in depth description of relevant dangers and uncertainties, we encourage you to evaluate the corporate’s most up-to-date quarterly report on Kind 10-Okay filed with the SEC, in addition to the corporate’s different SEC filings. The corporate doesn’t undertake any obligation to publicly replace its forward-looking statements, together with any monetary projections supplied right this moment primarily based on subsequent occasions or circumstances.

And now I will flip the decision over to our president and CEO, Doug Ingram, who will present an summary of our latest progress. Doug.

Doug IngramChief Government Officer

Thanks, Mary. Good afternoon and thanks for becoming a member of Sarepta Therapeutics’ first-quarter 2023 monetary outcomes convention name. Maybe you will note this as a break from the previous, however I intend to maintain my remarks this night temporary. As , in a mere 10 days from now, we can be attending and presenting on the FDA advisory panel for SRP-9001, our gene remedy meant for the remedy of Duchenne muscular dystrophy.

We consider that the first areas of debate on the advisory committee can be these: whether or not the totality of proof helps the conclusion that SRP-9001 dystrophin protein on the ranges expressed by this remedy within reason more likely to predict medical profit. The totality of the proof will embrace pure historical past, the preclinical knowledge, biomarker outcomes, and the purposeful outcomes from our medical trials. The panel may even handle the risk-benefit evaluation related to the administration of SRP-9001 for the remedy of ambulatory sufferers with DMD within the context of accelerated approval. And eventually, an evaluation of the flexibility to carry to conclusion EMBARK, or examine 301, the proposed post-marketing confirmatory trials to assist the accelerated approval of SRP-9001 within the occasion that accelerated approval is granted.

The crew is effectively ready and excited to share with the advisory committee the wealth of compelling proof supporting the conclusion that SRP-9001 dystrophin protein within the quantities expressed by that remedy within reason more likely to predict medical profit. So, to set expectations for this name, we intend to debate first-quarter efficiency, and Dr. Louise Rodino-Klapac will present a pipeline replace. However to respect the method and in gentle of how quickly the assembly will happen, we is not going to focus on or entertain questions on regulatory issues pertaining to SRP-9001 or the upcoming advisory committee assembly till it is concluded on Could twelfth.

Transferring now to the quarter, I’m very happy to announce one other robust quarter of efficiency. First-quarter complete income got here in at $253.5 million. Internet product income got here in at $231.5 million, that is a 23% improve over the identical quarter of the prior yr and exceeding analyst consensus. It’s this very affected person oriented execution that we intend to use to SRP-9001 if accepted.

We proceed to progress our essential post-marketing commitments for our three accepted PMO therapies. We now have already accomplished 11 of our post-marketing commitments. With respect to ESSENCE, which is our two-year blinded placebo-controlled examine for Vyondys and Amondys, we had been absolutely enrolled final yr, and that trial is continuing. With respect to MIS51ON, which is our dose-ranging post-marketing dedication for Exondys, half 2 of that examine is considerably enrolled and progressing.

Because it pertains to SRP-9001, along with making ready for the Could twelfth advisory committee and prosecuting the BLA with the Could twenty ninth PDUFA date in thoughts, we are making certain that we’ll be ready to efficiently launch 9001 and serve the neighborhood if and when accepted. By now, now we have efficiently concluded all FDA inspections that features three GMP inspections and two GCP inspections. With our accomplice, Catalent, we’re producing materials to efficiently launch 9001 upon approval, and we’re finalizing our launch readiness work. And we’re finalizing our plans to start the research essential to increase the 9001 label to the broadest inhabitants supported by the science, together with our graduation of our non-ambulatory examine, ENVISION Research 303, and our a number of research to discover the elimination of neutralizing antibodies to rAAVrh74.

Past that, we have been advancing our broader pipeline, and Dr. Rodino-Klapac will present an replace on our pipeline momentarily. Now the approaching weeks and months are monumentally essential to the sufferers that we serve. As I’ve mentioned many occasions by now, we stand at a bellwether second with the best evidence-based hope but in historical past to carry a greater life to households right this moment residing with and, sadly, right this moment, dying from Duchenne muscular dystrophy.

And we’re additionally effectively conscious that this BLA stands as a bellwether take a look at for gene remedy itself and for the flexibility to successfully lean in and use the instruments accessible to us to translate groundbreaking genetic science to drugs that may prolong and enhance sufferers’ lives now, not merely at some theoretical level sooner or later. We really feel an unlimited obligation to the sufferers that we serve, and our each choice and motion is taken and finished with that obligation entrance of thoughts. And with that, let me flip the decision over to our Head of R&D and Chief Scientific Officer Dr. Louise Rodino-Klapac.

Louise?

Louise Rodino-KlapacGovernment Vice President, Chief Scientific Officer

Thanks, Doug. Good afternoon. As we stay up for the weeks and months forward, we stay resolute in our conviction and our values to comply with the science and current the target proof that helps SRP-9001’s capacity to alter the trajectory of Duchenne muscular dystrophy. Our aim with SRP-9001 is to change the course of this deadly illness by treating the underlying explanation for Duchenne with a one-time gene remedy that delivers purposeful dystrophin to the muscle groups.

Sarepta generated essentially the most compelling preclinical biomarker and medical purposeful outcomes to this point, greater than another gene remedy in improvement for Duchenne. We have been in a position to exhibit primarily based on the robust scientific underpinning of our assemble that early SRP-9001 knowledge supplied read-through for our optimistic medical expertise with the remedy. After years of analysis, we recognized an optimum gene cassette in a position to retain essentially the most essential, protecting, and purposeful components and match inside AAV, thereby enabling its supply. This gene cassette was packaged into our AAV of selection, rAAVrh74, and we selected MHCK7 as our promoter.

The early knowledge confirmed sturdy expression throughout skeletal, diaphragm, and cardiac muscle. And because of that expression in addition to the dystrophin protein demonstrating purposeful advantages, we noticed medical good thing about the goal dose in sufferers with Duchenne. I’ll clarify in a bit extra element. People with Duchenne do not have a functioning dystrophin-associated protein complicated for that state.

Understanding this, after we inserted a purposeful dystrophin protein, we noticed upregulation of DAPC in animal fashions. Extra particularly, we noticed an virtually one for one upregulation of the DAPC when there was expression of the SRP-9001 dystrophin, confirming the protecting properties of the protein. Additional, we noticed vital discount in creatine kinase, or CK. CK is an enzyme related to muscle injury.

The rejection of CK supplied additional proof that SRP-9001 was moderately more likely to predict medical profit. Since 2018 and throughout a number of research who does the most important variety of Duchenne sufferers greater than another gene remedy in improvement for this illness, and the medical outcomes have surpassed our expectations. In abstract, SRP-9001 demonstrated sturdy expression of dystrophin, far above what literature would recommend is important to be protecting of the muscle. All of it’s correctly localized on the muscle membrane, or sarcolemma, the place it acts as a shock absorber.

We developed a cell-based efficiency assay that exhibits that SRP-9001 is energetic, purposeful, and protecting on the muscle membrane and as in animal fashions with sturdy expression of SRP-9001 with vital discount of CK. Lastly, expression of SRP-9001 in sufferers results in upregulation of the DAPC. Along with all of this compelling proof, we had been in a position to present purposeful advantages versus what pure historical past would predict on NSAA, or the North Star Ambulatory Evaluation, which is our major purposeful endpoint. We noticed profit throughout one, two, and four-year time factors.

Primarily based on the totality of the info, we consider that SRP-9001 qualifies as a disease-modifying agent as on the ranges of dystrophin expressed are moderately more likely to predict medical profit in sufferers with Duchenne. Now transferring to limb-girdle muscular dystrophy, or LGMD. We stay dedicated to advancing our LGMD portfolio throughout a wide range of subtypes. I stay up for offering updates on these essential packages within the months forward.

At the moment, we’re making wonderful progress on Journey, our LGMD pure historical past examine; and in VOYAGENE, our part 1 examine evaluating SRP-9003 for the limb-girdle muscular dystrophy Sort 2E in ambulant grownup sufferers and non-ambulant sufferers inside medical course of SRP-9003. Mixed with optimistic expression and purposeful knowledge shared from our preliminary examine, SRP-9003-101, we consider the info from VOYAGENE will give us insights right into a broader affected person inhabitants. Our subsequent milestones for VOYAGENE embrace finishing enrollment within the second half of the yr and starting our part 3 examine utilizing commercially consultant processed supplies later within the yr. Lastly, we’re on observe to start a systemic pilot examine for SRP-606-004 dual-vector rh74-mediated gene remedy to deal with LGMD 2B characterised by the absence of the proteins [Inaudible].

Turning now to the progress we made with our RNA platform. We’re happy to finish enrollment within the first quarter of 2023 for our MOMENTUM examine for SRP-5051. And we stay on observe to announce knowledge from the examine within the again half of 2023. In regard to our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSENCE trials for post-marketing requirement for [Inaudible] market.

We proceed to make good progress with our MIS51ON examine, which is on observe to be absolutely enrolled this yr. In closing, we’re trying ahead to the advisory committee assembly on Friday, Could twelfth, as it would present us the chance to share the science and the info in assist of SRP-9001. I might wish to take this chance to thank our Sarepta crew who’ve been diligently working these previous months. I will now flip the decision over to Dallan for an replace on our industrial actions.

Dallan.

Dallan MurraySenior Vice President, Chief Business Officer

Thanks, Louise, and good afternoon. Within the first quarter of 2023, the crew executed on our core RNA enterprise and delivered one other robust efficiency throughout all three of our RNA-based PMO therapies. As Doug talked about, we delivered $231.5 million in internet product income within the first quarter, representing effectively over 20% development over the primary quarter of 2022. Notably, Q1 of 2023 exceeded our expectations and represented essentially the most profitable first quarter within the historical past of our marketed therapies.

In Q1, now we have traditionally seen an impression within the U.S. resulting from anticipated insurance coverage adjustments to start with of every yr. As a result of extraordinary efforts of the crew in navigating these entry headwinds, we noticed higher-than-expected income within the U.S. within the first quarter.

Every year, our crew is ready for these challenges, and I am very pleased with their steadfast dedication and sense of urgency with which they serve [Inaudible] neighborhood. They know that each minute issues for every one of many sufferers we serve. Complete ex-U.S. internet product income within the first quarter was roughly $31 million.

This represented a lower over the prior quarter, which was anticipated and absolutely mirrored in our annual steering forecast. As mentioned on final quarter’s name, we count on to see continued fluctuations in ex-U.S. order issues for the quarter. Total, the basics of the enterprise popping out of Q1 are utterly in step with what we anticipated at this level within the yr.

And we reiterate our full-year steering upgraded to 925 million in internet product income for our PMO therapies. This steering displays the entire elements that we navigate and monitor in supporting sufferers globally. With this growing world income base, we are going to proceed to see fluctuations within the internet product income recorded within the quarter. Importantly as effectively within the U.S.

market, now we have now hit a mature part with all three merchandise, and as such, we count on extra modest development and new affected person begins within the coming quarters for the PMO enterprise. Turning now to particular person internet product revenues for the primary quarter of 2023 for our three accepted RNA-based chemotherapies. Exondys 51 totaled $132.6 million, representing greater than 13% development over Q1 of 2022. Our Vyondys 53 gross sales had been $33 million, rising roughly 18% over the primary quarter of 2022.

And our Amondys 45 gross sales totaled $65.9 million, representing greater than 50% development versus Q1 of 2022. Along with the robust efficiency in our core enterprise, the crew has been concurrently making ready and laying the groundwork for the SP-9001 launch. The total crew is in place, being rigorously educated as we communicate, and I can say with confidence that they’re able to execute if SP-9001 is accepted. This launch will signify a historic second not just for Sarepta however for the Duchenne neighborhood and for genetic drugs.

The extent of enthusiasm and confidence of the crew is at an all-time excessive, and they’re anticipating this chance to exhibit what we’re able to and what can be our fourth launch within the Duchenne market. Over the previous a number of months, our area groups and Sarepta management have meaningfully engaged with roughly 75 websites of care on strategic and operational website readiness issues. These essential interactions are to make sure that the websites are able to effectively, safely present SRP-9001 gene remedy to sufferers as quickly as attainable. We have additionally been working intently with websites to supply schooling and coaching in addition to making certain that they’ve the required gear and assets to ship the remedy to sufferers.

Along with website readiness, we all know from our expertise with PMO therapies that entry and reimbursement are essential to efficiently delivering SRP-9001 to sufferers. We’re dedicated to making sure that our gene remedy for Duchenne is accessible to all sufferers who want it, and we acknowledge that meaningfully partaking with payers is a essential a part of attaining that aim. We discovered that the payers are asking essential questions pertaining to the SRP-9001 medical knowledge, potential affected person inhabitants dimension, launch timing, and infusion websites. We’re inspired by the optimistic response we obtained to date and are happy on the progress now we have made in partaking with each industrial and Medicaid payers.

If accepted, 9001 will carry forth the possibly transformative remedy to sufferers who’ve been ready for much too lengthy. The crew has finished an incredible job, making ready for what would be the largest gene remedy launch to this point if SRP-9001 is accepted. I might wish to take this chance to personally thank the entire group who aren’t solely executing to assist the 30% of sufferers on our [Inaudible] right this moment however have additionally risen to the event in order that we could be prepared as a crew for this paradigm-shifting second. And now I will flip the decision over to Ian for an replace on our financials.

Ian.

Ian EstepanChief Monetary Officer

Thanks, Dallan, and howdy, all. This afternoon’s monetary outcomes press launch supplied particulars for the primary quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please discuss with the press launch accessible on Sarepta’s web site for a full reconciliation of GAAP to non-GAAP monetary outcomes. For the three months ended March thirty first, 2023, the corporate recorded complete revenues of $253.5 million, which include internet product revenues and collaboration revenues, in comparison with revenues of $210.8 million for a similar interval of 2022, a rise of $42.7 million.

Internet product income for the primary quarter of 2023 from our PPMO exon skipping franchise was $231.5 million, in comparison with $188.8 million for a similar interval of 2022. The rise in internet product income primarily displays growing demand of our merchandise. In every of the quarter ended March thirty first, 2023 and 2022, we acknowledged $22 million of collaboration income which pertains to our collaboration association with Roche. The reimbursable co-development prices underneath the Roche settlement totaled $20.3 million for the primary quarter of 2023, in comparison with $17.7 million for a similar interval of 2022.

On a GAAP foundation, we reported a internet lack of $516.8 million, or $5.86, and $105 million, or $1.20 per primary and diluted share, for the primary quarter of 2023 and 2022, respectively. This transformation is primarily as a result of loss on debt extinguishment of $387.3 million, a noncash expense incurred within the three months ended March thirty first, 2023 with no comparable exercise for a similar interval of 2022. We reported a non-GAAP internet lack of $85.5 million, or $0.97 per primary and diluted share, within the first quarter of 2023, in comparison with a non-GAAP internet lack of $48.6 million, or $0.56 per primary and diluted share, within the first quarter of 2022. Within the first quarter of 2023, we recorded roughly $35 million in price of gross sales, in comparison with $31.4 million in the identical interval of 2022.

The rise in price of gross sales is primarily resulting from an growing demand for our merchandise in addition to proper off of sure batches of our merchandise not assembly the standard specs for the three months ended March thirty first, 2023 with no comparable exercise in the identical interval of 2022, partially offset by a lower within the royalty funds in the course of the three months ended March thirty first, 2023 resulting from adjustments within the BioMarin royalty time period. On a GAAP foundation, we recorded $245.7 million and $194.3 million in R&D bills for the primary quarter of 2023 and 2022, respectively, a year-over-year improve of $51.4 million. The rise is primarily resulting from a rise in our manufacturing bills. On a non-GAAP foundation, R&D bills had been $220.7 million for the primary quarter of 2023, in comparison with $173.2 million for a similar interval of 2022, a rise of $47.5 million.

Now turning to CIGNA,SG&A. On a GAAP foundation, we recorded roughly $110.7 million and $71.8 million for bills for the primary quarter of 2023 and 2022, respectively, a rise of $38.9 million. The rise was pushed primarily by a rise in skilled service bills to organize for a possible launch of SRP-9001. On a non-GAAP foundation, the SG&A bills had been $83.3 million for the primary quarter of 2023, in comparison with $53.2 million for a similar interval of 2022, a rise of $30.1 million.

We count on that our R&D and SG&A expense will improve subsequent quarter as we proceed to organize for a possible launch of SRP-9001. On a GAAP foundation, we recorded $12.7 million in different earnings, internet, for the primary quarter of 2023, in comparison with $17.3 million in different expense, internet, for a similar interval of 2022. The change is primarily resulting from a rise in curiosity earnings and accretion of funding low cost as a result of funding mixture of our funding portfolio in addition to a discount of curiosity expense incurred because of the reimbursement of our December 2019 time period mortgage throughout 2022. Within the first quarter, we exchanged a portion of our 2024 notes with an combination principal worth of $313.5 million and issued roughly 4.5 million shares of our widespread inventory.

We accounted for the trade of the debt extinguishment recognizing the distinction of the honest worth of the shares of widespread inventory transferred on the trade date and the web carrying quantity of the extinguished debt as a lack of $387.3 million, inclusive of the $6.9 million of third-party debt conversion prices, simply, once more, to reiterate this can be a noncash expense. We had roughly $1.9 billion in money, money equivalents, and investments and long-term restricted money as of March thirty first, 2023. We stay effectively capitalized to execute on our objectives for the yr and assist our transition to profitability assuming approval of SRP-9001. And with that, I will flip the decision again over for Doug to start out the Q&A.

Doug?

Doug IngramChief Government Officer

Thanks, Ian. Now earlier than we start the Q&A, let me reiterate and in gentle of — and in respect of the upcoming FDA advisory committee, we is not going to be entertaining questions on the regulatory course of for the upcoming advisory committee assembly for SRP-9001 tonight. I do stay up for discussing these issues with you as soon as the Could twelfth advisory committee assembly has concluded. And with that, Shannon, let’s open the road for questions.

Questions & Solutions:

Operator

Thanks. [Operator instructions] Our first query comes from the road of Colin Bristow with UBS. Your line is now open.

Colin BristowUBS — Analyst

Hey, good afternoon. And, , simply congrats on being on the cusp of this approval. So, in your provide at launch, are you able to speak in regards to the capability and skill to fulfill demand? I am simply asking in gentle of the actual fact of — or a number of the feedback out of your accomplice round a slower-than-expected manufacturing ramp on the Maryland website. After which, simply type of inside this, by way of affected person logistics, are you able to discuss your expectations for the preliminary timelines from a doctor prescribing 9001 to a affected person receiving the remedy given the necessity for antibody testing and different potential labeling necessities? Thanks.

Doug IngramChief Government Officer

Yeah, thanks very a lot for that, Colin. So, on the primary a part of the query, as we have mentioned, our aim — and we are going to meet that aim — is to have the ability to absolutely launch and provide the neighborhood assuming that we’re in a position to get an approval. After all, our aim proper now’s to get an approval on the PDUFA date, which is Could twenty ninth. We now have seen the feedback made by Catalent that we’re very clear that these feedback and the problems that they had been discussing don’t play any position in or have any impact on our plans or our manufacturing plans within the slightest.

So, we ought to be in fine condition there. With respect to the launch, , our aim is to launch this remedy as quickly as attainable. And I feel now we have confirmed ourselves over the previous few years very able to doing precisely that. With that mentioned, the 9001 gene remedy has its personal specific problems.

You have received it, as an example, along with not merely having a starch kind for that. You going to do plenty of different issues together with getting a pre-infusion antibody take a look at that is sufficiently near the infusion, that it is legitimate. After which, after all, they don’t seem to be — they usually — you need to — should work by means of entry to reimbursement and codes and the like. So, from a planning perspective, whereas we’ll be launching this quickly, one ought to anticipate actually seeing, , 1 / 4 or two earlier than we actually begin seeing a big ramp.

Operator

Thanks. Our subsequent query comes from the road of Gena Wang with Barclays. Your line is now open.

Gena WangBarclays — Analyst

Thanks. I additionally needed to ask one industrial query. What’s your estimate affected person numbers for preliminary indications within the U.S.? And likewise, concerning the manufacturing capability, any main enlargement you should do with a purpose to provide affected person for the preliminary indication within the U.S.?

Doug IngramChief Government Officer

Let me reply the second half first. The answer is no. There aren’t any main capital expenditures or extra enlargement that’s required to launch this remedy and serve the neighborhood at launch. On the addressable affected person inhabitants, I can provide the broadest of strokes.

They’re someplace within the 12,000, 10,000 to fifteen,000 sufferers in america. Our aim — once more, assuming that we’re accepted at a PDUFA date, the aim is to serve all ambulatory sufferers. The ambulant sufferers are about 50%, so it is roughly 50% ambulant, 50% non-ambulant. We can be masking the ambulant inhabitants, after which, there can be, after all, sufferers that may be excluded as a result of they’ve preexisting neutralizing antibodies.

Primarily based on our most up-to-date knowledge, that is about 13.5% of sufferers can be at the moment excluded on the idea of getting preexisting antibodies to binding antibodies to rh74. After which, there’s a subset of sufferers who would have a few of these earlier mutations in a variety — in a area which will have a threat of an innate immune response. That’ll be lower than 5% of sufferers. So, that the rest is the addressable affected person inhabitants at launch.

Now, with that mentioned, I’ll go forward and provide you with our plans for the long run as effectively as a result of, , very quickly we’ll be beginning plenty of research in an effort to totally construct out the addressable affected person inhabitants to the extent that science permits us to. We’re fairly assured about that. In order that the most important alternative clearly is to get to the nonambulant sufferers. That’s terribly essential to us and to them.

Nonambulant sufferers do not have the posh of time, so we have got to maneuver as quick as attainable. We’re beginning our examine 303 for the nonambulant inhabitants very quickly. The aim is to have enough security and expression knowledge from that examine to hunt an replace to our label early subsequent yr to get nonambulant sufferers within the label so we are able to start to dose them. And we’re beginning as effectively two extra research for 2 various approaches to clear preexisting neutralizing antibodies.

And if we’re able to doing that, then that may additionally carry again into to border for the flexibility to dose sufferers which are at the moment excluded as a result of they’ve — they’ve one thing that reacts and is a preexisting neutralizing antibody. That is a very essential concern as effectively. , oftentimes, you will — whenever you speak to sufferers and their households, dad and mom will say that the worst day of their lives was getting the information that their little one had Duchenne muscular dystrophy and that the second worst day of their lives was discovering out that their children are considered one of these, , uncommon children, about 13% of youngsters who’ve examined optimistic for preexisting neutralizing antibodies. So, we have to transfer quick to attempt to clear up that concern for them.

Thanks for these questions, Gena.

Operator

Thanks. Our subsequent query comes from the road of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian AbrahamsRBC Capital Markets — Analyst

Hello, good afternoon. Thanks for taking my query. Are you able to increase on what you’ve got been listening to in your preliminary conversations with payers? Is your sense that they’d be open to paying for 9001 underneath accelerated approval or would favor to attend for full approval? And will we count on type of an analogous combine as with the exon skippers