Sarepta Therapeutics Inc Q4 2024 Earnings Call Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Tazeen Ahmad - BofA Securities Inc. - Analyst : Okay, great. Thank you for taking my question. I wanted to ask, Doug, about the cadence of what you're seeing in 1Q so far with regards to patients that are being onboarded. You've talked extensively about certain things that cannot be changed, and you've reiterated your confidence about guidance for 2025. But can you give us a little bit more granularity on, again, what you're seeing in 1Q and how we should be thinking about the cadence of uptake for the rest of the quarters this year? Thanks.

Question: Ellie Merle - UBS Securities LLC - Analyst : Hey, guys, thanks so much for taking the question. For limb girdle, just from your work in the space, what's the latest on what you see for the prevalence of 2E and how many in the US are diagnosed today, and the mix of ambulatory versus non-ambulatory patients? Basically, if you will be launching here next year, how should we be thinking about the contribution potentially to revenue and how many patients there might be kind of waiting for therapy? Thanks.

Question: Gena Wang - Barclays Capital, Inc. - Analyst : Thank you for taking my questions. I have one question regarding the data update later this year for both FSHD and the DM1. If I hear correctly, DM1 will be the SAD data. Is FSHD also SAD data? And if they are the SAD data, how many cohorts and how long follow up? And what kind of data you will share with us?

Question: Andrew Tsai - Jefferies LLC - Analyst : Thanks. I appreciate the update. Congrats on all the progress. Maybe one more question on the DM1 FSHD data you'll be sharing later this year. Do you think the initial SAD data will be conclusive to the point where investors can determine whether these programs are looking superior or not, or do you need MAD data? And how would you define superiority in these two programs? Thank you.

Question: Kostas Biliouris - BMO Capital Markets Corp. - Analyst : Thanks for taking our question and congrats on the progress. A question on ELEVIDYS from us, given that it's been about eight months now since the label expansion, can you comment on whether you have seen any reimbursements for PMOs in ELEVIDYS-treated patients? And if not, are you hearing anything from payers around their willingness to reimburse PMOs following ELEVIDYS treatment? Thank you.

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Hey, guys. Thanks for taking the question. Maybe just a quick one on the $500 million share repurchase program. Can you just remind us what the timeframe is there? And then some of the considerations as you decide when you might deploy that. Thanks.

Question: Joe Schwartz - Leerink Partners LLC - Analyst : Great. Thanks very much. So since approval in '23, it seems like the ELEVIDYS launch has had a few different stages with great growth early on following accelerated approval, then a couple of flat-to-down quarters in early 24, followed by a really nice return to growth following the expanded label in mid '24. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 26, 2025 / 9:30PM, SRPT.OQ - Q4 2024 Sarepta Therapeutics Inc Earnings Call So since your '25 guidance seems to imply that growth this year should be more moderate, I'm just wondering if you can give us your view about the stage of the launch that we're in now and how you see it evolving this year. Are there any important constraints to growth that we should keep in mind this year?

Question: Gil Blum - Needham & Co. LLC - Analyst : Hi, good afternoon, everyone. And again, congrats on all the progress. So maybe a question here on the move to suspension manufacturing with the bridging study later this year. Can you remind us what are the potential associated cost savings? And also, how broadly applicable is this? I mean, you guys use a lot of different programs for this in gene therapy. Is this translatable just outside of DMV, or is this very narrow? Thank you.

Question: Ritu Baral - TD Cowen - Analyst : Hi, guys. Thanks for taking the question. Doug and Louise, I wanted to ask your current thoughts as you understand the landscape on the potential for splicing biomarker-based accelerated approval for your FSHD and DM1 programs. I know it's far in the future. But obviously, this is a hot topic of the space. And then, actually, if I could follow up on Gil's question. He asked about the bridging study. Can you describe what that bridging study is? It sounds like it's going to go fast.

Question: Brian Skorney - Robert W. Baird & Co., Inc. - Analyst : Hey, good afternoon. Thanks for taking my question. I was hoping you could kind of walk through what you sort of perceive as the current order of potential rate-limiting steps to commercial ELEVIDYS revenue recognition. I'm just trying to understand. It doesn't seem like it's demand in any REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 26, 2025 / 9:30PM, SRPT.OQ - Q4 2024 Sarepta Therapeutics Inc Earnings Call sense right now. So what's sort of the step-throughs that are kind of creating blocks from realizing the full demand. Is it like -- would one be like insurance authorization? Would it be center capacity? Could you like kind of rank order those?

Question: David Hoang - Deutsche Bank Securities, Inc. - Analyst : Hi there. Thanks for taking my question. So I just had one on if you have any thoughts on a recent data set that was generated by a next-generation DMV gene therapy competitor, and do you have any insights from your field force on how a family of patients might think about receiving treatment with commercial ELEVIDYS versus enrolling in a clinical trial for one of these other products. Thank you.

Question: Rai Forsett - Guggenheim Securities LLC - Analyst : Hi, this is Rai from Debjit's team. We have two questions. Number one, how is Sarepta modeling the impact of competitive gene therapies in DMD, especially on the annual incidence population under the assumption that the prevalence pool is saturated? And number two, for the 2E program, should we expect higher vector genomes per nucleus relative to historical Sarepta data and protein expression above 50%? And is there a threshold for regulatory submission?

Question: Priyanka Grover - JPMorgan Securities LLC - Analyst : Hi, guys. This is Priyanka on for Anupam. Thank you for taking our quick question. As the R&D Day is in the second half of the year, can we assume potential new data could be presented there from Arrowhead or other non-Arrowhead pipeline programs? Thank you.

Question: Biren Amin - Piper Sandler Companies - Analyst : Yes. Hi, guys. Thanks for taking my questions. For the EMERGENE trial, I think you're enrolling both ambulatory and non-ambulatory patients, while the Phase 1 enrolled ambulatory. So should we express -- should we expect expression would be similar in non-ambulatory patients to what was observed in the Phase 1 ambulatory data? And then, when you report these data, will you be comparing these to the NCH national history cohort?

Question: Gavin Clark-Gartner - Evercore ISI - Analyst : Hey, guys. Thanks for taking the question. I just wanted to focus on terminal value. So if I look at the outer year of consensus estimates, take 2033, I see about $2.2 billion in US ELEVIDYS sales. So that implies about 850 to 900 treated patients annually. Maybe, Dallan, you could just remind us specifically what you're seeing on US incidents. But more broadly, do you believe this consensus estimate is plausible? And maybe explain what has to happen for ELEVIDYS to reach and stay into that range into the next decade. Thank you.

Question: Sami Corwin - William Blair & Co. LLC - Analyst : Great. Thank you. Congrats on the progress, and thanks for taking my questions. I was curious how you're thinking about the evolution of your gross margins in 2025 and 2026 as ELEVIDYS begins to compromise a larger percentage of your revenue. And then a quick question on your FSHD program. Is this DUX4 assay new, or was it developed in-house, and could you just elaborate on it a little more? Thank you.

Question: Daniel Smith - H.C. Wainwright & Co., LLC - Analyst : Good afternoon. This is Dan on for Mitch. Thanks for taking our question. Congratulations on the positive cash flow for the year. So payers we've spoken with have said that they have had patients experience two rounds of appeals and were ultimately denied. Would an IRO denial not count as permanent denial? And if not, what qualifies as a permanent denial? Thank you.

Question: Leo Watson - Mizuho Securities USA LLC - Analyst : Hi, guys. This is Leo on for Uy. Thanks for taking our question. Could you provide some detail on the learnings from the pre-BLA meeting with the FDA on 9003 and how these learnings might be applied to the follow-on limb girdle programs? And then also, based on the recent changes within the agency, how do you think FDA interactions might change going forward? Thanks. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 26, 2025 / 9:30PM, SRPT.OQ - Q4 2024 Sarepta Therapeutics Inc Earnings Call

Question: Tommie Reerink - Goldman Sachs & Co. LLC - Analyst : Thanks for taking our questions. This is Tommie for Salveen. Just overall, what do you see as Arrowhead's differentiation in terms of kind of their chemistry or structure versus some other RNA approaches in DM1 and FSHD? And on ELEVIDYS, is there flexibility to expand upon existing infusion center capacity, including, for instance, staffing needs? Thank you.