Q3 2024 Sarepta Therapeutics Inc Earnings Call
Good day and welcome to the Sarah Pudix, the quarter of 2024, by not to result, comforts after the speaker's presentation, there will be a question and answer session.
To ask a question during this session you will need to press star 1-1 on your telephone and you will hear an automated message inviting you your hand is raised. To withdraw your question please press star 1-1 again. As a reminder today's conference is being recorded.
Speaker Change: At this time, I would like to turn the call over to Mary Jenkins, Associate Director, Investor Relations and Corporate Communications. Please go ahead. Thank you.
Mary Jenkins: Thank you, Michelle, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the third quarter 2024. The press release is available on our website at SREPTA.com, and our 10-Q was filed with the Securities and Exchange Commission this afternoon.
Joining us on the call today are Doug Ingram, Ian Estepan, Dallan Murray, and Dr. Louise Rodino-Klapak. After our formal remarks, we'll open the call for Q&A.
I'd like to note that during this call, we will be making a number of forward-looking statements. Please take a moment to review our slide on the webcast, which contains our forward-looking statements.
These forward-looking statements involve risks and uncertainties, many of which are beyond surreptitious control. Actual results could materially differ from these forward-looking statements, and any such risks can materially and adversely affect the business, results of operations, and trading prices for surreptitious common stock.
Speaker Change: For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent quarterly report on Form 10-Q filed with the SEC as well as the company's other SEC filings.
Speaker Change: The company does not undertake any obligation to publicly update its forward-looking statements, including any financial projections provided today based on subsequent events or circumstances. And now I'll turn the call over to our President and CEO, Doug Ingram, who will provide an overview of our recent progress. Doug?
Speaker Change: For more information visit www.fema.gov
Doug Ingram: Thank you, Mary. Good afternoon, and thank you for joining Sarepta Therapeutics' third quarter 2024 financial results conference call.
Speaker Change: I am pleased to report another strong quarter of commercial performance. Earlier today, we reported total net product revenue of about $430 million, growing at 39% versus the same quarter last year.
Speaker Change: Elevitas net product revenue was 181 million dollars. Our U.S., our ex-U.S. partner Roche has made good progress in the quarter and if one includes the royalty revenue from Roche's ex-U.S. Elevitas performance
Speaker Change: We achieved $190.5 million in the quarter. Even without considering royalty revenue, our levitous performance substantially exceeded our prior guidance.
Speaker Change: as well as Analyst Consensus. We are tracking well to Q4 and 2025 performance consistent with prior guidance.
Speaker Change: Our PMO sales, comprising Exondus 51, Myondus 53, and Amondus 45, achieved approximately $249 million in the third quarter, again, substantially exceeding
Speaker Change: analyst consensus.
Speaker Change: As anticipated, we are not yet seeing material cannibalization of our PMO franchise from a Levitas performance.
Speaker Change: I am also pleased to report that we were again profitable on a gap and non-gap basis in the third quarter. Dallan Murray, our Chief Customer Officer, will provide more color on commercial performance in a moment.
Speaker Change: Turning to R&D, we have rationalized and prioritized our pipeline this quarter and we've made great progress advancing programs.
Speaker Change: Before I discuss our progress, let me discuss our PPMO program. You will have seen in our release that we have made the decision to discontinue the SRP 5051 development program.
Speaker Change: This was done after dialogue with the FDA and their requirements, after our own risk-benefit analysis for the program, and in consideration for the evolving landscape for Duchenne, including the approval of a Levitas.
Speaker Change: Now, one never takes lightly the decision to discontinue a program, but we are confident that our decision is the right one for the patient community and for our stakeholders.
Speaker Change: Moving to our pipeline progress, we are beginning to move rapidly into late-stage clinical with a planned approval BLA in 2025.
Speaker Change: for SRP 9003 to treat limb-girdle muscular dystrophy, or LGMD, type 2 E.
Speaker Change: The commencement of our trial for SRP 9004 to treat LGMD type 2D in the fourth quarter of this year, and the commencement of our trial for SRP 9005.
Speaker Change: to treat LGMD type 2c in early 2025, both of which trials are intended to support accelerated approval.
Speaker Change: We have also made progress with our clinical development efforts for Elevitus, including clinical activity to support XUS approvals and meet our post-marketing commitments.
Speaker Change: data presentations that speak to the value of a Levitas to those living with Duchenne and clinical development work intended to expand the population for a Levitas.
Speaker Change: Additionally, our program to move elevators to suspension manufacturing is proceeding very well. We have had very encouraging interactions with the FDA and we continue our engineering runs in anticipation of commencing a bridging study in 2025.
Speaker Change: We have also made good progress with the rest of our neuromuscular, CNS, and cardiomyopathy pipeline and intend to share more concerning our pipeline and its progress.
Speaker Change: in 2025. Our Head of R&D and Chief Scientific Officer, Dr. Louise Rodino-Klapak, will discuss this decision and our R&D progress in a moment. And with that, I will turn the call to Dallan Murray for more detail on commercial performance and our plans. Dallan?
Dallan Murray: Thank you, Doug, and good afternoon.
Dallan Murray: We're pleased to report an impressive third quarter, led by the continued strength of the Elevitas launch.
Speaker Change: The success and performance we've achieved since approval has been and continues to be driven by robust patient demand from both the ambulant and non-ambulant populations, ample site capacity,
Speaker Change: positive trends in access and reimbursement, and consistent conversion rates as we continue to see patients gaining access within the three to five month conversion timeline.
Speaker Change: Additionally, as the team works to execute on the broad label, it's important to note that we've thoughtfully built a model which allows us to flex as needed to accommodate demand from different segments, such as the older non-ambulatory patient population.
Speaker Change: Turning to the third quarter revenue numbers, net product revenue for Elevitus reached 181 million dollars and grew by nearly 50% compared to the prior quarter, exceeding our guidance of quarter-to-quarter growth of 30%.
Speaker Change: Of note and looking forward, I remain impressed with our team's ability to forecast. As we have shown with our performance this quarter, we have good visibility on the launch dynamics, and as predicted, we are now at the inflection point on the launch curve.
Speaker Change: Thank you very much.
Speaker Change: Further enhanced by the wealth of data presented at WorldMuscle that Louise will discuss, we have strong momentum going into the fourth quarter and reiterate our previously stated Q4 guidance, which is in line with the current Q4 consensus.
Speaker Change: Based on all of these factors and our confidence in meeting the Q4 expectations, we remain in a strong position to achieve guidance for 2025.
Speaker Change: The progress made by this team is unprecedented in our industry. We've anticipated all of the factors necessary to execute on a broad label, and our performance provides clear evidence of our continued ability to meet expectations and execute as promised.
Speaker Change: Turning to our PMO franchise, which also achieved impressive results in the third quarter. Importantly, we expect to see continued strength in the coming quarters and years.
Speaker Change: We deliver roughly $249 million in net product revenue.
Speaker Change: With approximately 4% growth in Q3 compared to the third quarter of 2023, the PMOs represent a healthy and solid segment of our Duchenne franchise.
Speaker Change: Exonis 51 led the way for the PMOs with $140.7 million in net product revenue.
Speaker Change: Vionda's 53 delivered $32.2 million in net product revenue in the third quarter, and we delivered $75.9 million in net product revenue for Amanda's 45 and Q3, representing growth of nearly 15% compared to Q3 of 2023.
Speaker Change: Thank you for watching. Please subscribe to my channel. I hope to see you again soon.
Speaker Change: In closing, I'd like to thank the SIRREPTA team and all of our partners, whether at the sites of care or elsewhere.
Speaker Change: And most importantly, I'd like to thank the patient community who have been with us on this pioneering journey every step of the way.
Speaker Change: We're grateful to be exactly where we had always wanted to be, and that is with approved therapies serving the vast majority of the Duchenne patient community.
Speaker Change: And with that, I'll hand the call over to Dr. Louise Rodino-Klapak for the R&D update. Louise.
Speaker Change: Thanks, Dallan.
Speaker Change: I'll begin my remarks with a Leviticus and then provide an update on our pipeline.
Speaker Change: We continue to advance the Elevitus Clinical Program and share new datasets as they become available.
Speaker Change: We recently published the primary one-year EMBARQ results in Nature Medicine, a high-impact journal. In addition, we had multiple presentations at the World Muscle Society Congress in early October. This included additional EMBARQ data, muscle MRI, and cardiac MRI.
Speaker Change: Muscle MRI changes were consistent with functional outcomes from EMBARQ Part 1, showing stabilization or slowing of disease progression with SRP 9001, while progression occurred in placebo-treated patients, evidenced by accumulation of fat and fibrosis.
Speaker Change: Cardiac MRI demonstrated that at one year post gene therapy there was no evidence of harm observed.
Speaker Change: Future longitudinal cardiac MRI studies will assess long-term protection cardiac muscle.
Speaker Change: As we previously described, Elevitis contains an MHTK7 promoter that expresses well in the heart to protect cardiac muscle. We have shown in the MDX RAP model of Duchenne improvements in cardiac function as well as overall survival.
Speaker Change: In addition to the EMBARQ data, we've also presented safety and expression data from Study 103, or ENDEAVOR, demonstrating consistent safety and expression data across ambulatory and non-ambulatory patients.
Speaker Change: As of the end of October 2024, we have dosed over 80 late ambulatory and non-ambulatory patients within our clinical program and continue to see a consistent safety profile.
Speaker Change: Finally, we shared five-year longitudinal data from Study 101 demonstrating that SRP 9001 stabilizes or slows DMD progression with an increase in divergence from natural history over time.
Speaker Change: as shown by external control analysis and supported by the independent CTAP analysis.
Speaker Change: No new safety signals were identified.
Speaker Change: As mentioned on our last call, the Elevitus Accelerated Approval for Non-Ambulatory Patients includes a post-marketing commitment to confirm clinical benefits, which will be addressed via our Non-Ambulatory and Late Ambulatory Study 303, also known as ADVISION.
Speaker Change: As a reminder, ENVISION is a global, randomized, double-blind, placebo-controlled, two-part study.
Speaker Change: Envision is progressing well, with U.S. enrollment complete and the remaining recruitment occurring ex-U.S. Enrollment is expected to be completed in 2025, with our last patient last visit expected in 2027, following an 18-month placebo-controlled period.
Speaker Change: We also continue to advance clinical studies that monitor long-term follow-up with the Leviticus.
Speaker Change: Our long-term follow-up studies include Endure and Expedition.
Speaker Change: As a reminder, ENDURE is a Phase IV observational study that will follow individuals treated with a Levitas for up to 10 years.
Speaker Change: In addition, EXPEDITION is a Phase III study enrolling approximately 400 patients that were previously enrolled in a limited clinical trial and followed for consistent safety and efficacy measures for up to five years.
Speaker Change: Regarding patients currently ineligible to receive elevatives under the expanded label, we continue to advance multiple studies.
Speaker Change: For the approximately 15% of patients who are screened out for pre-existing anti-AEV Rh-74 antibodies, we have commenced two studies, one with embolicidase to cleave antibodies and a second with plasmapheresis to remove antibodies.
Speaker Change: We expect to have expression and safety data from Sentinel patients in early 2025.
Speaker Change: In addition, for patients under the age of 4, we have treated patients as young as 2 in our Study 103, and together with our partner Roche, we are executing Study 302, or EVOL, to gain experience dosing patients under 4 and as young as 3 months.
Speaker Change: We continue to communicate the range of trial experience in patients treated with elevative from those that are under four to those with more advanced disease.
Speaker Change: Moving now to our programs for the limb-girdle muscular dystrophies or LGMDs.
Speaker Change: starting with SRP 9003.
Speaker Change: As we mentioned in the first quarter call, we initiated dosing early this year in study SRP 9003-301.
Speaker Change: also known as Emergene, or Phase III Multinational Open Label Clinical Trials, SRP-9003, for the treatment of limb-girdle muscular dystrophy type II-E, or beta-circle glycanopathy.
Speaker Change: The agreed primary endpoint of emerging is expression of beta-circle glycan, the absence of which is the sole cause of the disease.
Speaker Change: The study is on track to be fully enrolled by the end of 2024.
Speaker Change: Assuming a positive pre-BLA meeting, we will anticipate a BLA filing in 2025.
Speaker Change: We are encouraged by the agency's willingness to support a viable pathway for SRP 9003, an ultra-rare genetic condition that is progressively debilitating, results in loss of ambulation, and leads to early mortality.
Speaker Change: The ability to progress a small NF15 biomarker study together with our ability to demonstrate delivery of a functional beta-circle glycan protein is extremely important, not just for this program, but for the other circle glycanopathies in our pipeline.
Speaker Change: including LGMD2D and LGMD2C, both of which are progressing to the clinic.
Speaker Change: Having successfully advanced SRP 9003, we submitted our SRP 9004 IND update reflecting our suspension process this year, with Phase I initiation expected by year's end.
Speaker Change: As a reminder, SRP 9004 is designed for the treatment of limb girdle muscular dystrophy type 2D or alpha-circular glycanopathy.
Speaker Change: Finally, we are also rapidly progressing our program for SRP 9005 for the treatment of limb-girdle muscular dystrophy type 2c or gamma-circumglycanopathy.
Speaker Change: We plan to engage with FDA in Q4 of this year with plans to initiate a clinical study in Q1 2025.
Speaker Change: To summarize, we are very pleased with the progress of our LGMD portfolio and expect to have three of our LGMDs in the clinic in less than six months. We are maximizing the synergies across this platform from both an R&D and manufacturing perspective, and our sights are firmly set on accelerating the remainder of the LGMD assets to the clinic.
Speaker Change: Continuing with our RNA platform and beginning with our PMOs, the ESSENCE trial, our post-marketing requirements for Goladursin and Casimirsin, as well as Mission, our post-marketing commitment for Exondys, are both fully enrolled and remain on track. We look forward to sharing data as soon as the study is complete.
Speaker Change: Turning now to PPML.
Speaker Change: As announced today, and in line with our unwavering commitment to Patients First, we have decided to discontinue development of SRP 5051, also known as vesiceplasin, or Investigational Peptide Conjugated PMO, or PPMO.
Speaker Change: to treat Duchenne. This means dosing and our momentum study SRP 5051-201 has stopped.
Speaker Change: The safety of study participants is our highest priority, and while we are encouraged by the dystrophin expression results with SRP 5051, the long-term safety in a chronic treatment setting does not support further development.
Speaker Change: Our initial hypothesis was that the hypomagnesemia was manageable and monitorable.
Speaker Change: Although events thus far have remained medically manageable in a small number of patients, we saw persistent hypomagnesemia despite treatment discontinuation, and our risk-benefit analysis led us to end the study.
Speaker Change: The MOMENTUM study provided important information around the use of RNA-targeted therapies to increase dystrophin production in Duchenne, and we are extremely grateful to the patients, families, and clinicians who participated in our study.
Speaker Change: Now, spend a moment discussing our current and future pipeline.
Speaker Change: During the past six years since I joined Sarepta, we've been diligently building expertise and capabilities to advance our current portfolio and identify new assets.
Speaker Change: We've significantly advanced the field of genetic medicine through clinical trials and advanced research for patients with pre-existing antibodies and the potential for redosing.
Speaker Change: We've also advanced regulatory precedent for gene therapy for rare disease, as evidenced by a rapidly developing LGND platform.
Speaker Change: On the research side, we have optimized, developed, and characterized new AAV capsids that will change the landscape for neuromuscular gene therapy and unlock potential in cardiac and central nervous system disease areas.
Speaker Change: As an example, we've optimized the construct for Charcot-Marie-Tooth type 1A, or CMT1A, using AAV ARG74, and are now rapidly advancing to the clinic following exciting preclinical data.
Speaker Change: As a reminder, we are using a surrogate approach for delivery of the neurotrophin 3 or NT3 gene to improve myelination and nerve regeneration in CNT1A.
Speaker Change: This pipeline-in-the-project approach has applicability to other CMTs as well as other demyelinating indications.
Speaker Change: We look forward to highlighting our impressive pipeline in an R&D day in 2025.
Speaker Change: I'll close by thanking all the patients who participate in our trials and my incredibly talented R&D colleagues who make all of this possible. The future is bright because of their work.
Speaker Change: I'll now turn the call over to Ian Estepan for an update on our financial results. Ian?
Ian Estepan: Thanks, Louise, and good afternoon, everyone.
Speaker Change: The financial results press release provided details for the third quarter of 2024 on a GAAP basis as well as a non-GAAP basis. Please refer to our press release available on our website for full reconciliation of GAAP to non-GAAP financial results.
Speaker Change: As a reminder, beginning in the fourth quarter of 2023, amortization of unlicensed rights and income tax expense are no longer excluded from non-GAAP results.
Speaker Change: The company has added the Income Tax Effective Adjustments, which represents the estimated income tax impact of each of our pre-tax non-GAAP adjustments based on the applicable effective income tax rate.
Speaker Change: Non-GAAP financial results for the third quarter of 2023 have been updated to reflect this change for comparability purposes.
Speaker Change: Thank you.
Speaker Change: So for the three months ended September 30, 2024, the company recorded total revenues of $467.2 million.
Speaker Change: which consists of net product revenues and collaboration and other revenues compared to revenues of $331.3 million for the same period of 2023, an increase of $135.4 million.
Speaker Change: Net product revenue for the third quarter of 2024 for Molebius was $181 million compared to $69 million.
Speaker Change: Same period of 2023.
Speaker Change: Net product revenue for the third quarter of 2024 from our PMOX on Skipping Franchise was $248.8 million compared to $240.2 million for the same period of 2023. The increase in net product revenue primarily reflects the net product revenue associated with sales of Elevitas.
Speaker Change: For the three-month end of September 30, 2024, the company recognized $37.4 million of collaboration and other revenues.
Speaker Change: which primarily relates to the commercial elevated supply delivered to Roche and royalty revenue from Roche compared to collaboration revenues of $22.5 million for the same period of 2023, an increase of $14.9 million.
Speaker Change: The reimbursable co-development costs under the Roche Agreement totaled $61.5 million for the third quarter of 2024, compared to $34.9 million for the same period of 2023. As a reminder, these Roche reimbursable co-development costs are an offset to our operating expenses.
Speaker Change: On a gap basis, we reported a net income of $33.6 million, or $0.35 per basic share and $0.34 per diluted share, and a net loss of $40.9 million, or $0.46 per basic and diluted share for the third quarters of 2024 and 2023, respectively.
Speaker Change: We reported a non-GAAP net income of $67 million or $0.62 per diluted share in the third quarter of 2024 compared to a non-GAAP net income of $31.5 million or $0.31 per diluted share in the third quarter of 2023.
Speaker Change: In the third quarter of 2024, we recorded approximately $91.7 million in cost of sales compared to $37 million in the same period of 2023.
Speaker Change: The increase in cost of sales primarily reflects the cost of sales related to elevatives during a three-month ended September 30, 2024, following the label expansion in June.
Speaker Change: On a gap basis, we recorded $224.5 million and $194.3 million in R&D expenses for the third quarter of 2024 and 2023, respectively, a year-over-year increase of $30.2 million. The increase primarily reflects a $55.4 million
Speaker Change: associated with the termination of an inherent commercial manufacturing and supply agreement met of the reimbursable termination cost by Roche, partially offset by a decrease in clinical and manufacturing activity for our PPMO and its Hefferson program.
Speaker Change: On a non-GAAP basis, R&D expenses were $199.8 million for the third quarter of 2024, compared to $163.9 million for the same period of 2023, an increase of $35.9 million.
Speaker Change: Now, turning to SG&A, on a gap basis, we recorded approximately $128.2 million and $120.9 million of expenses for the third quarters of 2024 and 2023, respectively, an increase of $7.3 million.
Speaker Change: The increase was primarily driven by an increase in professional services used to support the continued efforts to commercialize Alevis, and an increase in compensation and other personnel expenses primarily related to the changes in headcount.
Speaker Change: On a non-gap basis, the SG&A expenses were $100.2 million for the third quarter of 2024, compared to $92.8 million for the same period of 2023, an increase of $7.4 million.
Speaker Change: On a gap basis, we recorded $11.8 million in other income met for the third quarter of 2024 compared to $12.3 million of other loss met for the same period of 2023.
Speaker Change: The change is primarily due to an impairment or a strategic investment during the three months ended September 30, 2023, with no similar activity in 2024.
Speaker Change: We had approximately $1.4 billion in cash-based equivalency investments and long-term restricted cash as of September 30, 2024. So to conclude, unsurprisingly, our strong commercial execution and our continued focus on expense management has put us in a strong financial position.
Speaker Change: We now expect to be sustainably profitable from both the GAAP and non-GAAP perspective and will turn cash flow positive early in 2025. And with that, I'll turn the call back over for Doug to start the Q&A.
Doug Ingram: Thank you very much, Ian. And Michelle, let's open the call for questions. Thank you. As a reminder, to ask a question, please press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again. We ask that you please limit yourself to one question. One moment for our first question.
Speaker Change: Our first question is going to come from the line of Tazeen Ahmad with B of A Securities. Your line is open. Please go ahead.
Tazeen Ahmad: for 4Q results, now that you've beaten expectations. Can you tell us what that base number is? Is it 100% upside from what you just reported? Thanks.
Speaker Change: Yeah, we feel very comfortable with the guidance we did before, which was we'd be 100% above
Speaker Change: The guidance that we had for Q3, I will also linger and note that we continue to be very comfortable with the guidance we gave for 2025, the long-term guidance, $3 billion, about two-thirds of which will be a levitas, and one-third of which
Speaker Change: would be our PMOs and we're also very confident in our long-term projections.
Speaker Change: which would have peak year sales across the four approved therapies at $5 billion or more.
Speaker Change: and that we would be treating with the Levitas, the prevalent population over the course of the 2020s into around 2030 or so and we'll be growing for some number of years.
Speaker Change: Thank you and one moment for our next question.
Speaker Change: For more information visit www.FEMA.gov
Speaker Change: Our next question comes from the line of Gina Wang with Barclays. Your line is open. Please go ahead.
Gina Wang: Thank you. Since I can only ask one question, I will ask about the CPMO discontinuation of 5051. So maybe, can you give a little bit more color regarding the hypomagnesium? Is that the main concern the FDA raised? And can you give a little bit more color since, you know, at what point that you see more severe cases? And also, when we...
Speaker Change: The old data back at the 30 mg at the 12 weeks was 6.55% protein expression and at that time you projected it could be over 10% at the 96 weeks with the protein level is still in line with what you projected and is mainly is the safety and are you planning to complete discontinue the PPMO franchise?
Speaker Change: yeah so I will first of all thank you for your question Gina I'll turn the question over to Louise to make some comments about our decisions regarding the PPMO program
Louise Rodino-Klapak: Sterling, I'll try to make sure I remember all of the parts. So on the franchise, we are discontinuing the entire PPMO franchise. They all use the same cell penetrating peptide and so that this also includes the discontinuation of the other PPMO programs.
Louise Rodino-Klapak: The decision to stop the program was based on, you know, multi-factorial, so it included our discussions with the FDA, which we've had on the development of that. At this point, the FDA...
Louise Rodino-Klapak: primarily based on our own benefit risk assessment, and that was including safety, which included the hypomegnesemia that we saw, which was consisted of prolonged hypomegnesemia in some cases.
Speaker Change: the approval of the love list. So taking all of those things together led to our decision.
Speaker Change: Let me just contextualize all this, just to remind folks.
Speaker Change: Commencing really in February of 2018, the FDA had confirmed that it had a precedent for the use of the accelerated approval pathway for PMOs, and that precedent was really rested on two things. It's obviously rested on the value of the district in production.
Speaker Change: for Duchenne Muscular Dystrophy, but it also rests as well on the safety of the morpholino oligonucleotides, the PMOs, and that safety profile itself is very laudable. It's second, really, to almost none.
Speaker Change: When one attaches a delivery MODI to a PMO, then you will change that safety profile, potentially, in that risk-benefit analysis. And I think the agency.
Speaker Change: was not comfortable with the concept of accelerated approval in light of this change. We independently did a risk-benefit analysis and concluded that the program should not continue.
Speaker Change: So that's where we are. I think this is in the first and foremost, first and foremost, it is in the benefit of the patients that we serve. And then secondly, of course, it benefits our other stakeholders as well.
Speaker Change: Thank you and one moment for our next question.
Speaker Change: And our next question is going to come from the line of Andrew Tsai with Jeffrey's. Your line is open. Please go ahead.
Andrew Tsai: Hey, thanks. Good afternoon. Congrats on the execution. Thanks for taking my question. So, about the 11th launch, your partner Roche made a comment about two weeks ago how 500 patients have been treated worldwide so far, including 450 in the U.S. So, can you help us reconcile that 450 U.S. patient number? Presumably a good chunk are in clinical trials, but even backing out those patients, we could be getting a nice implied jump in Q4 sales. That could be above your Q4 guidance. So, maybe help us reconcile that. Thank you.
Speaker Change: Yeah, Andrew, I'm not going to comment or confirm that we haven't provided...
Speaker Change: Those numbers like that, we're going to use revenue as our metric and we're, as it stands today, standing on the guidance that we've provided.
Speaker Change: previously, I mean it certainly is the case qualitatively, that we have dosed an enormous number of patients. We have an extraordinary amount of experience with elevators.
Speaker Change: Louise will have mentioned to you that we have already dosed, between clinicals and some commercial, 80 or so, probably more than that by now, about 80 patients that are either late ambulatory or non-ambulatory, in addition to all of the other patients we've dosed.
Speaker Change: And, as you know, we've not seen a difference in any safety metrics. So the things that look great, the profile of the therapy looks great, and the launch is going great.
Speaker Change: So that's where we are right now with it, and we're excited to give you an update after 2.4.
Speaker Change: Thank you and one moment for our next question.
Speaker Change: Our next question is going to come from the line of Brian Abrams with RBC Capital Markets. Your line is open. Please go ahead.
Speaker Change: Hi, this is Kevin on for Brian. Thanks for taking our questions I just wanted to ask you know Can you speak to what progress you've made in conversations with payers on broader coverage policies? And maybe can you speak more to your efforts in ensuring Medicaid coverage policies? And if you can remind us what proportion of patients with DMD you estimate to be on Medicaid. Thanks a lot
Speaker Change: Thank you.
Speaker Change: Yeah, so it's about, and Dallan, following my remarks, if there's anything to add or if I've made a fundamental error, you can correct me.
Speaker Change: Broadly speaking, it's about 50% commercial, 50% Medicaid, things are going very well, interactions both from a Medicaid perspective and a commercial perspective are very productive.
Speaker Change: And one of the things that we're really benefiting right now is a couple of things. One, the amount of evidence and data that we have that supports...
Speaker Change: Bringing a Levitas to a broad group of Duchenne patients is great and gives us a lot to talk about. The depth of the conversations that we're having with payers and the quality of those conversations is absolutely fantastic. And we're all benefiting from this.
Speaker Change: If one focuses down on SREP itself, you know, we have become...
Speaker Change: very expert over the last nearly a decade, about eight years.
Speaker Change: in working with payers and gaining access to patients for our therapy.
Speaker Change: At the same time...
Speaker Change: In fairness, physicians have become increasingly more sophisticated in how to manage through the process to get kids on therapy.
Speaker Change: and payers, both Medicaid.
Speaker Change: and commercial have become far more experienced with Duchenne muscular dystrophy and how that disease works and the like, so that the entire quality of the discussions.
Speaker Change: are far more productive than they may have been some eight years ago. And it's also resulting in the performance that you're seeing today, which is of course a very, very good launch, which is matching the trajectory.
Speaker Change: that we had imagined that we would have with all of the work that we did to prepare ourselves for this launch.
Speaker Change: Thank you and one moment for our next question.
Speaker Change: Our next question comes from the line of Sylvain Richter with Goldman Sachs. Your line is open. Please go ahead.
Speaker Change: Thanks for taking our question. This is Tommy on for Salveen. So just on the 2Q call, I believe it was mentioned that there were some infusions that were rescheduled from 2Q to 3Q. Is it possible to quantify that impact? And more broadly, what's the latest thinking on how to think of the tail end of the launch and the peak year? Thanks so much.
Speaker Change: I'm a...
Speaker Change: The first part of the question I will turn it over to Dallan, the second part of the question is that what I will say to you right now is that we're going to go through a period
Speaker Change: of significant growth over the course of this decade and we will
Speaker Change: We will get through the prevalent population by 2030. With that said...
Speaker Change: Dallan, if you want to comment a little more on Q2-Q3. Yeah, the question was if that impacted our performance or, you know, our guidance in any way. Remember, we guided off of Q2 sales, growth off of Q2 sales, so that was factored into our guidance and expectations for Q3 and Q4 already.
Speaker Change: Thank you. One moment for our next question.
Speaker Change: © Dallan Murray, Tazeen Ahmad, Mary Jenkins, Ian Estepan, Douglas Ingram, Louise Rodino
Tazeen Ahmad: Hey guys. Thanks so much for taking the question. Just a quick one from me. You've given a little bit more details on kind of your internal pipeline focus here in the near term. It looks like we're going to hear a little bit more at an R&D day in 2025. I'm just wondering how we should think about sort of the time and resources spent on the internal pipeline versus you guys doing some external business development. And I know you guys only said one question, but since it's third quarter earnings, if I could just ask if you might pre-announce at a small healthcare conference in January, that would be cool. Thanks.
Speaker Change: Okay, so first of all, thank you very much for your question, Donna Palm, as it relates to your first question.
Speaker Change: Let me say that we are really focusing right now on two major and very important things.
Speaker Change: One, of course, is the launch of Elevitus, which, as you can see right now, hopefully is going swimmingly, and that's the result of an enormous amount of work over many years by a lot of folks. And also in that first part is the continuing performance of the PMOs, which themselves are doing very well, bringing a better life to kids, very durable, and so far not seeing any significant impact from the launch of Elevitus, so all fantastic there.
Speaker Change: The second big effort of this organization right now is advancing our internal pipeline.
Speaker Change: We're getting a lot of great traction there. There is a lot of excitement. We're going to do kind of a curtain raiser next year and, you know, really show you some of the deeper pipeline and some of the things that we're very excited about. But even in the near term, in the late stage, you can see.
Speaker Change: We are in the next few months, really, we'll be in clinical trials on three of our limb-dortal programs. So that's really beginning to accelerate, and we're very, very...
Speaker Change: I'm excited about that. That's what we're focused on as an organization right now. Yes.
Speaker Change: We are going to go, as Ian has said, we're going to be profitable from a gap and a non-gap basis on a go-forward basis as we have been in prior quarters now. And we're going to be cash flow positive, really starting very early next year. And that gives us some opportunities to think even more broadly. But if you're asking us about the things that we're focusing on right this minute, right now,
Speaker Change: It's ensuring the success of our four approved therapies, including the launch of the Levitas, and really advancing and accelerating our internal pipeline, which we are.
Speaker Change: excited about.
Speaker Change: Now, on the second one, I'm not going to make any commitments as we stand here right now. We have had, as you know, a history of doing some preannouncements at that conference in January, but of course, we'll wait and see in January what we do there. But, you know, we will likely be consistent with history.
Speaker Change: That's where we are.
Speaker Change: Thank you. One moment for our next question.
Speaker Change: Our next question is going to come from the line of Danielle Brill with Raymond James. Your line is open. Please go ahead.
Danielle Brill: For all intents and purposes, it sounds like the launch is going great. So, I guess we just thought that the step-up in treatment rates could be more pronounced following the label expansion. Can you just talk a little bit more about what you're seeing in the market and what the main bottlenecks are to getting these kids treated? Thanks.
Speaker Change: Let me just say again, when we talk about performance, we're going to talk in revenue as our marker. We're not going to use other metrics.
Speaker Change: And we're very, very excited about the progress of our launch, and it's going exactly to plan. And we're seeing the trajectory that we had envisioned and we spoke about.
Speaker Change: a few quarters back. So everything is going fantastic.
Speaker Change: The shape of that growth
Speaker Change: is a combination, broadly, of three things, and it always will be.
Speaker Change: Some of it is, you know, a piece of it is.
Speaker Change: site capacity. And that's not simply, you know, a matter of getting more sites up and running. It's the making sure that you have sites that are well-educated, well-informed, expert, and that they have the ability to manage and monitor kids post-dosing so that we always have
Speaker Change: This extraordinarily positive, you know, safety and efficacy profile that we have today with a Levitas, at least from my perspective.
Speaker Change: The second one, of course, is just, you know, manufacturing and batch release and making sure that we're thoughtful about that. And the third one is payer interactions, which are going great, both from a Medicaid perspective and a commercial perspective.
Speaker Change: very well. So things are tracking exactly as we had hoped and that's why we're able to reiterate
Speaker Change: The guidance that we provided previously, both about Q4 and our guidance for 2025.
Speaker Change: $3 billion. Thank you.
Speaker Change: is the mean of that, and our long-term forecast, and the fact that we will be going through a period of multiple years of significant growth as we, over the course of this decade, treat the prevalent population, even as we are also treating the incident population now and deeply into the future.
Speaker Change: Thank you and one moment for our next question.
Speaker Change: Our next question comes from the line at L.A. Murrow with UBS. Your line is open. Please go ahead.
L.A. Murrow: Thank you. Thank you.
L.A. Murrow: Hey guys, thanks for taking the question. Can you just elaborate a bit more on your comments on site capacity just now? Just the latest that you're seeing, you mentioned you're seeing ample site capacity, but I guess how many sites dose patients in 3Q and I guess how dispersed or concentrated has this dosing been across the sites? Thanks.
Speaker Change: Yeah, so we have about, so I'll just say broadly, we have about 75 sites. They might modestly increase over time, reactively, and Dallan can provide an answer to us on, as we sit here today, what percentage of those sites have actually been dosing sites?
Speaker Change: Yeah, the vast majority of centres have dosed patients, and we really don't get into...
Speaker Change: specifics quarter to quarter on, you know, how concentrated these sites are. So as Doug had said earlier, we're guiding our, you know, we're focusing our guidance on revenue, net product revenue.
Speaker Change: Thank you.
Speaker Change: Thank you and one moment for our next question.
L.A. Murrow: For more information, visit www.FEMA.gov
Speaker Change: Our next question is going to come from the line of Gil Blum with Needham & Company. Your line is open. Please go ahead.
Gil Blum: Hey guys, thanks for taking our question and congrats on the progress.
Gil Blum: So, you know, now that 50-51 seems out, are there any thoughts for lifecycle management of the PMOs? And it kind of feels like SREPTA is strategically moving away from ExxonSkippers. Thank you.
Speaker Change: Thank you for watching. Please subscribe to our channel.
Speaker Change: First, I don't want to suggest for a moment that we're moving away from Exxon Skippers.
Speaker Change: And we do have a lot of thoughts on other modalities for exon skipping. We're not in a position right now to discuss them in any detail, but...
Speaker Change: We are constantly looking at opportunities to enhance the benefit that...
Gil Blum: that our therapies provide to patients. The reason that we're not moving away from, actually I'm skipping right now, even though we are very excited.
Gil Blum: both about Elevitus and what Elevitus can do but and also the rest of our deep pipeline including
Gil Blum: cardiomyopathies and CNS and other neuromuscular as well.
Gil Blum: The reason we're so excited about PMOs is they're doing an enormous amount of good right now. Okay, so, and that's an important thing to consider, you know.
Gil Blum: The PMOs make a small amount of dystrophin, but a small amount of dystrophin is very meaningful as we have recently published the real-world evidence on exondus, which will.
Gil Blum: presumably be the same answer for Valiadis and Amadis over time as...
Gil Blum: We remain very excited about the benefit that our PMOs can safely provide to patients, and that's one of the things that's really great about the PMOs, and that is that they not only are providing a really significant benefit,
Gil Blum: to patients, but the safety profile is great. And that's why one should remember, even as we think about our own pipeline and we think about others, that while we're always ambitious, the bar for beating the PMOs is very high and the road.
Gil Blum: to any approval.
Gil Blum: for an alternative to our PMOs on the exon skipping side is very long. So, I don't want to suggest for a minute that we're in any way less excited about our PMOs. We're excited every week that a patient gets an infusion and benefits from either Ixondas or Vyondas or Imondas.
Gil Blum: And we will remain that way, and I think one of the things that I'm excited about right now, particularly during the launch and the fact that we're not seeing a ton of cannibalization is that there is a long road for the PMOs, and I think they're going to be very durable.
Speaker Change: For more information, visit www.fema.gov
Speaker Change: Thank you and one moment for our next question.
Speaker Change: Our next question is going to come from the line of Red 2 Borough with TD Cowan. Your line is open. Please go ahead.
Speaker Change: Hi, guys. Thanks for taking the question. I wanted to ask about forward guidance, especially given the insight you guys have.
Speaker Change: on
Speaker Change: on basically revenues. Doug, you mentioned, you know, three to five months time to fill. And just given where...
Speaker Change: You guys are notified about when a patient is seeking reimbursement and when you have to, you know, make their kit, ship the kit, etc. and when they're dosed. Are we going to, one, are we going to continue to get
Speaker Change: some pretty granular guidance as you've given. And two, given the healthy beat congratulations this quarter, what sort of swing factors should we be thinking about despite that three to five month insight, you know, logistical swing factors beyond
Speaker Change: You know, Ian's...
Speaker Change: tendency to give conservative guidance.
Ian Estepan: Look, you know, of course it's always our goal to...
Speaker Change: provide, you know, reasonable and accurate guidance, but guidance that we're very comfortable
Speaker Change: with, and we will continue to ensure that we are comfortable.
Speaker Change: with the guidance that we provide. And if that makes us seem conservative, I'm probably proud of that. We have an enormous amount of insight, as you know, about not only broadly about the Shedmuster Distribute and epi and prevalence, but all the way down to things like start forms and the like. And that does provide us with a lot of confidence as we think about forecasting and guidance and the like.
Speaker Change: With that said, as we sit here today, notwithstanding our very positive, in my view, and significant beat, we're not going to update our guidance at this moment, and we'll talk again.
Speaker Change: probably at J.P. Morgan if we do pre-announce at J.P. Morgan about 2025.
Speaker Change: All of us. All of us. All of us.
Speaker Change: Thank you and one moment for our next question.
Speaker Change: Our next question comes from the line of Joe Swartz with Leering Partners. Your line is open. Please go ahead.
Joe Swartz: Great. Thanks very much. I have a question about the next MRI study you plan to do. I'm wondering how will this...
Joe Swartz: differ from the MRI data that you presented at WMAS, and will you be using gadolinium enhancement to evaluate tissue characteristics like edema and fibrosis?
Speaker Change: Perry Dosing and Will You Evaluate Global Longitudinal Strain?
Speaker Change: Louise.
Louise Rodino-Klapak: Sure, so from the EMBARQ study, the longer-term data...
Louise Rodino-Klapak: We'll be following the patients over time and then that will be compared if there's a wealth of natural history data compared to natural history since all patients have been dosed now, so they'd be compared to their baseline and then to
Louise Rodino-Klapak: Natural History.
Speaker Change: inflammation and edema that you noted. So that's a precursor to changes that you would see by MRI and MRS. So we'll continue to use those three measurements and strain as a portion.
Speaker Change: of the MRI results.
Speaker Change: [inaudible]
Speaker Change: Thank you, and one moment for our next question.
Speaker Change: Our next question comes from the line of Kostas Bilioris with BMO Capital Markets. Your line is open. Please go ahead.
Kostas Bilioris: Given the 300% quarter-over-quarter growth of Elevity Sales x US,
Kostas Bilioris: and potential further acceleration with upcoming approvals in Europe and Japan. Can you comment on your manufacturing capacity to meet this high level of demand? And a follow-up on this same topic, can you clarify whether the suspension manufacturing will be only with thermo features, or there is optionality to do that with catalan as well? Thank you.
Speaker Change: For more information visit www.fema.gov
Speaker Change: Yeah, thank you very much for your questions, Custis. First, as relates to the first part of your question, we're very comfortable with our forecast and our manufacturing to supply. We're in a very good position from a manufacturing perspective to serve ours and our partners' needs.
Speaker Change: As it relates to suspension, to remind everyone, things are going very well with suspension right now. We're in engineering runs, both in 500 liter and 2000 liters.
Speaker Change: And we're going to start our bridging study next year, assuming everything goes well, and we believe it will. And we have not made a decision, as it stands today, on who we will use as a commercial supplier for suspension. So that's an issue we're continuing to ponder and evaluate.
Speaker Change: Thank you for watching. Please subscribe to our channel. This helps us to keep you up to date on all our latest videos. Please visit us on Facebook, Twitter, Instagram and Google+.
Speaker Change: Thank you and one moment for our next question.
Speaker Change: Our next question comes from the line of Brian Scorney with Baird. Your line is open. Please go ahead.
Brian Scorney: Hey, good afternoon, everyone, and thanks for taking my question. Actually, my question is on redosing, and I guess, when can we think about seeing initial data here from either the AFERESA study or the HANSA collaboration study you're planning to go about? And how do you kind of think about clinical development here? Do you focus on patients who had received prior levodas but had inadequate dystrophin response? Do you target naive patients with higher RH-74 serology? Do you have access to just inflidase in your HANSA deal, or is HNSA 5487, their next-gen cleaver, included in that? Thanks.
Speaker Change: All right, I'm going to turn this to Louise. Before I do, I want to make sure I have a clarification here. And Brian, thank you very much for your question and the opportunity to clarify.
Speaker Change: So, there is definitely an opportunity down the road to use some modality to either cleave or clear neutralizing antibodies in a manner that might allow for redosing.
Speaker Change: and we're very excited about that potential. But just so we're clear, as we sit here today, that is not actually the goal of our near-term studies.
Speaker Change: Our near-term studies are intended to do something slightly different, which is...
Speaker Change: to clear or cleave antibodies that have been acquired by a...
Speaker Change: a Duchenne patient.
Speaker Change: through, you know, environmental exposure to put them in a position where we could dose them with a Levitas, and that would provide an opportunity for another 15% or so of Duchenne patients who would right now be screened out to actually get the opportunity to have a Levitas. And with that, Louise, if you want to provide some color on...
Speaker Change: Brian's questions.
Louise Rodino-Klapak: Sure. So the question about when we'll have data for both studies, we'll have safety and expression data in early 2025 on the initial cohorts.
Louise Rodino-Klapak: and really that data will inform us for anything we do in the future with regards to a potential study for re-dosing. As Doug mentioned, that's not the goal right now, but we've been...
Louise Rodino-Klapak: positioning ourselves to be ready in case we do. We've shown good data in non-human primates.
Louise Rodino-Klapak: So, the challenge for redosing is much higher antibody levels, and so this data from these pre-existing antibody charts will be critical in seeing how far we can go and how we might design a study if we were to do so with redosing.
Louise Rodino-Klapak: And then, Brian, to answer your final question, we have a right of first negotiation on a next-generation therapy.
Speaker Change: Thank you and one moment for our next question.
Speaker Change: Our next question comes from the line of Christine Kluska with Cantor Fitzgerald. Your line is open, please go ahead.
Christine Kluska: Hi, congratulations on the revenue beat. You seem to not be getting a whole lot of credit for the limb girdle portfolio. So I wanted to ask if you could remind us about the number of patients you expect could be identified around the time of these launches and how we should be thinking about that market opportunity. Thank you.
Speaker Change: Yeah, that's a great question. Thank you very much. And it's a particularly poignant question now because we're really starting to make traction and move fast on our limb girdle portfolio.
Speaker Change: Broadly speaking, let me say that the Lindgren portfolio that we have today is about 70% of the opportunity of Duchenne muscular dystrophy, so quite significant, and if you think about the size of Duchenne muscular dystrophy, it's maybe in the 12,000.
Speaker Change: Douglas Goldstein, CFP®, is the director of Profile Investment Services and the host of the Goldstein on Gelt radio show.
Speaker Change: opportunity to do good and do well by our investors at the same time.
Speaker Change: Our near-term program, CyberGlycan, are themselves actually quite significant.
Speaker Change: something in the range of 25% or more of the Duchenne Muscular Dystrophy and we're gonna be in late stage development with all three of those programs.
Speaker Change: in the coming few months. So, there's a big opportunity that I think people will start focusing on as they clear and get more excited about and more confident about the launch of Levitas and hopefully this quarter has helped them do that.
Speaker Change: Thank you, and one moment as we move on to our next question.
Speaker Change: Our next question comes from the line of Aaron Emmon with Piper Stanley. Your line is open. Please go ahead. Yeah. Hi, guys. Thanks for taking my questions. Can you maybe just talk about the split of patients that you saw in the third quarter across ambulatory versus non-ambulatory from a commercial standpoint? And what type of access are you seeing across both groups? Thanks.
Speaker Change: Yeah, we're not going to provide a granular breakdown of that, but I can give you some broad qualitative color. I mean, one of the things that one would anticipate, that we anticipated, and others have anticipated at launch, is that there may be a bias in favor of.
Speaker Change: ambulatory patients over non-ambulatory patients at launch. And certainly that is the case, but it isn't probably as significant as some may have imagined. We're seeing a very significant percentage of start forms.
Speaker Change: for late ambulatory, non-ambulatory patients, which is fantastic.
Speaker Change: I think that as more information comes out about the number of patients that have been dosed.
Speaker Change: in the ambulatory, non-ambulatory setting and the safety profile that we're seeing there, which is the same as the ambulatory patients, it's only gonna increase that awareness and excitement. We've already dosed kids in their mid-20s.
Speaker Change: who are obviously non-ambulatory, and we have stark forms of men in their late 30s, which is, you know, a very, very...
Speaker Change: advance for Deschamps-Moscow District.
Speaker Change: Thank you.
Speaker Change: While there is, as we have guided to and anticipated, there is some bias towards ambulatory versus non-ambulatory, in the very early days, you know, there is a very significant percentage of non-ambulatory, and we're quite confident that's going to continue to increase over time.
Speaker Change: Dallan, is there anything else about that that I've missed?
Dallan Murray: No, not at all. And, you know, just to add, it's not really going to fluctuate quarter to quarter, as you, as you had said, that bias towards the ambulatory population is kind of in the early going stages of the launch, but we don't expect much fluctuation in that breakdown from quarter to quarter. So thanks.
Speaker Change: Thank you. One moment for our next question.
Speaker Change: Our next question is going to come from the line of Gavin Clark Gunter with Evercore ISI. Your line is open. Please go ahead.
Speaker Change: Hey guys, thanks for taking my question. I just wanted to ask on the Essence confirmatory trial, is the latest guidance still for data in 2026? I'm just looking at the trial page and it still notes the primary completion date is next week actually. And just more broadly for this trial, do you have any sense what the requirements to actually pull these drugs off the market may be, especially in light of the fact we haven't really heard anything following the Viltepso results? Thanks.
Speaker Change: Douglas Ingram, Tazeen Ahmad, Mary Jenkins, Ian Estepan, Douglas Ingram, Louise Rodino
Speaker Change: That's correct.
Speaker Change: And then as it relates to the standards, the standard for evaluating a therapy and whether it, you know, should be removed from the market is based on the totality of evidence. When all of the evidence is in on the therapy, the FDA will look at the totality of all of the evidence.
Speaker Change: Associated with that therapy, including study results, including trends in the study results, including presumably information like the real-world evidence that we've gathered with respect to the.
Speaker Change: The PMOs, which has been very positive so far, and then we'll make a decision. It won't be a yes or no based on one single trial. That isn't the standard.
Speaker Change: Thank you.
Speaker Change: Thank you and one moment for our next question.
Speaker Change: Our next question is going to come from the line of Leo Watson with Mizuho. Your line is open. Please go ahead.
Speaker Change: Hi, this is Leo Anturoi. Congrats, Mr. Encorder, and thanks for taking our question. How are you thinking about the competitive dynamics in the Exxon skipping business, given the recent competitor data readouts?
Speaker Change: and the discontinuation of the PPMO. And while you expect no material cannibalization through 25, we're just curious on how you're thinking about cannibalization going forward in the long term. Thanks.
Speaker Change: Let me answer the second question first. So we don't think we'll see net cannibalization over the course of 2025. Remember we also have
Speaker Change: Ex-U.S. sales of the PMOs, and those won't be in the near term, at least, subject to any cannibalization.
Speaker Change: How significant, if at all, that will be is something we're going to watch and monitor. But we have some reason to believe that these therapies are going to be very durable, certainly on a net basis over this entire decade. But we'll see how that goes post-treatment.
Speaker Change: 25. As relates to competition I should note that we're not very focused on competition right now. There is no one near us today either on the TMOs or you know certainly on a Levitas.
Speaker Change: I will not comment on other folks' programs, and I wish them well. One of the things I do want to point out as it relates to the PMOs is that how much good those PMOs are doing today. You know, there was an open issue about that perhaps in late 2016, and when...
Speaker Change: Extrandus was approved, you know, Teplerson.
Speaker Change: But the real-world evidence has been really supportive.
Speaker Change: You can see it in the compliance rates for these therapies. They're doing an enormous amount of good.
Speaker Change: with a really, really laudable safety profile. And so the one thing I would say is that when we think about the PMOs and we think about competition, one ought to remember that thus bar to overachieve from both an efficacy and safety perspective.
Speaker Change: for the PMO. It's a very high bar.
Speaker Change: And the road for even getting to clinical data that would allow one to think about approval is a very long one. This is not, this requires one to go, you know, exon by exon over a very long period of time. So, you know, I wish these folks well. I think that they have a high bar and a long road.
Speaker Change: to get to the right place.
Speaker Change: Thank you. One moment for our next question.
Speaker Change: Our next question is going to come from the line of Sammy Corwin with William Blair. Your line is open, please go ahead.
Sammy Corwin: Hi there, congrats on the quarter and thanks for taking my questions.
Sammy Corwin: Looking ahead to a potential launch in Lynn Girdle, do you expect any synergies between LGMD and Elevitus in terms of sale reps or treatment centers? And then with the discontinuation of 5051, could any cost savings there be used to accelerate the development of any of those Lynn Girdle programs? Thanks.
Sammy Corwin: Well, the
Speaker Change: There's in a way a yes and a yes to both of your questions.
Speaker Change: Is there synergy between the limb girdle launch and what we've done with the Levitas? A hundred percent.
Sammy Corwin: Absolutely. It's going to be very, very synergistic.
Sammy Corwin: You know, there'll be nuanced things we need to do in advance to make sure we're very successful, but we have a very seasoned
Sammy Corwin: group of folks that know how to service the rare disease community and the neuromuscular community and to gain access and reimbursement and to focus on distribution. It's a fascinating thing to consider that the group that launched Exondus back in late 2016
Sammy Corwin: that have become so expert, you know, 80% of those folks.
Sammy Corwin: remain here with Sarepta today. So yes, there'll be a lot of really nice synergy and really frankly synergy at the therapy level as well. I would remind folks that these limb girdle programs that we're talking about share the same capsid.
Sammy Corwin: So they will really be standing on the shoulders of the safety profile of a Levitas and the same promoter.
Sammy Corwin: as a Levitist, so the productivity of these therapies is.
Sammy Corwin: standing on the shoulders of the Lebanese.
Sammy Corwin: And then as it relates to 50-51, I don't want to suggest that we can accelerate the plans based on the cost savings from 50-51 only because we're already doing everything we can to move these as fast as possible and you're seeing it in the progress that we've made and announced.
Sammy Corwin: in this earnings call. But certainly there will be some cost savings from 50-51 that we get to use to focus back down into research and development and advance our program, including our pipeline, which we're very excited to talk about next year.
Speaker Change: Thank you and one moment for our next question.
Sammy Corwin: Our next question comes from the line of Mike Ault with Morgan Stanley. Your line is open. Please go ahead. Hi. This is Rohit on for Mike. Thanks for taking our questions. Since we're at the inflection point for Elevated Sales, can you provide any color on early 4Q trends and how they're tracking versus expectations? Thanks.
Speaker Change: We're not going to do that right now. We have sort of reiterated the guidance that we provided previously.
Sammy Corwin: Notwithstanding that some appear to have criticized us for being conservative,
Sammy Corwin: for Q4, and we are reiterating our guidance for 2025, which I would remind you.
Sammy Corwin: is a very significant $3 billion, two-thirds of which will come from Elevitas and one-third of which will come from our three approved PMOs. So we're feeling very good about where we are as an organization and very consistent with all of our internal forecasts over the last year.
Sammy Corwin: a year or so.
Speaker Change: Thank you. I would now like to hand the conference back to Doug Ingram for closing remarks.
Doug Ingram: Well, thank you all very much for your very insightful questions and for spending time with us this evening. We've had a very positive quarter from our perspective.
Doug Ingram: We're very excited about our late stage Lin-Gerdal programs as well, which are moving into what will be clinical trials that can support the approval of those therapies as well.
Sammy Corwin: As one commenter made today, and I agree with, I don't think there has been enough attention spent on the limb girdles or the opportunity there both to do good for some patients who really need us and do well by the investors who stand by us.
Sammy Corwin: I look forward to keeping you all up to date and talking to you about the fourth quarter performance when we announce fourth quarter performance next week.
Speaker Change: This concludes today's conference call. Thank you for participating. You may now disconnect.
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