Scholar Rock Holding Corp Q1 FY2025 earnings call

David Hallal highlighted that 2025 has started strongly with progress on apitegromab for SMA, including positive Phase 3 SAPPHIRE trial results, BLA priority review by FDA with a September 22nd PDUFA date, and plans for US commercial launch in Q3 2025 with global launches to follow. Akshay Vaishnaw discussed R&D progress, including SAPPHIRE trial showing motor function improvement, BLA validation by EMA, and progress on SRK-439 IND filing, as well as the EMBRAZE study on obesity with top-line results expected in June 2025. Keith Woods provided an update on commercial readiness for apitegromab in the US, including stakeholder engagement, market access team work, and hiring of personnel. Vikas Sinha discussed financials, ending Q1 with $364.4 million, and prioritizing commercial launch and clinical programs with $100 million available under debt facility.

Anticipate US commercial launch of apitegromab in Q3 2025, EU approval in 2026. Initiate study of apitegromab for infants/toddlers with SMA under age 2 in Q3 2025. File IND for SRK-439 in Q3 2025. Share top-line results of EMBRAZE study in June 2025.

Q: Ahead of apitegromab launch, could you just frame for us how your discussions with US payers have gone? How receptive are they to coverage of combination therapy for both an SMN targeting and a muscle targeting therapy in SMA patients? And can you also describe that feedback for ex-US payers and governments as well?

A: David Hallal and Keith Woods discussed pricing reflective of disease rarity, value, and US payer interactions positive; EU plans to sequence launches.

Q: Maybe just two questions on obesity coming up in June. Given that it's towards the end of the second quarter but also that there's two different endpoints, I think 24 and 32 weeks. Are you thinking about providing more data to be a more complete package of information to help people out in terms of longer term follow up data on some of the metabolic parameters that could come out in June, have you thought about that? And then just holistically, the prior CEO, Jay, has mentioned like 20% to 40% reduction of new muscle loss would be great. But I think people are still sort of trying to grasp what you think a good result is and what is exciting. Can you give us some color on that?

A: Akshay Vaishnaw mentioned providing 24-week data as the main study and any necessary follow-up information.

Q: So in the US, you noted collaboration with regulators and that you remain on track here. Can you confirm or not if you have completed your mid cycle meeting? And if so can you comment on any high level discussions you have had around labeling? And at this time, what has the agency said about the need or lack thereof for an adcom?

A: Akshay Vaishnaw said constructive conversations on track for PDUFA date Q: Given that we're getting close to the PDUFA date, just a follow-up question from one of the prior analysts. Would you be able to provide any kind of metrics or guidance once the drug is approved or any color on how you see the early demand? And not sure how much you expect the recent executive order on drug pricing to have an impact on orphan disease area. But in light of that executive order, any comments on how you think about pricing in the US versus ex-U.S? I know it's early to give us exact numbers but just qualitatively.

A: David Hallal said pricing reflective of disease, early to comment on pricing order Q: Maybe just following up a little bit on Kripa's question before, just on launch trajectory. As you mentioned, the SMA population now has one 100% newborn screening in the US, they've gotten very concentrated into these centers of excellence to receive SMN therapy. Like those would seem to be kind of tailwinds to the launch relative to prior SMA launches but then, while there's certainly unmet need maybe it's not quite as dramatic as it was before the original SMN correctors launched, which would maybe be a headwind. I mean, do you think those net out and this we should look at prior SMA launches as a good proxy for the trajectory that apitegromab might have? And I guess, to the extent that you don't think those are good proxies, is there another other launches out there in the rare disease space that you think maybe are more analogous to what we should expect with apitegromab?

A: David Hallal and Keith Woods discussed urgency due to progressive motor function loss despite SMN therapies, focusing on long-term patient service Q: Recently, we had noted some stronger rebound in Spinraza sales to start 2025. And we're hoping to get your updated thoughts on long term changes that may occur in the SMA market, specifically with apitegromab being used in conjunction with Spinraza. Are you internally expecting any longer term growth in Spinraza that may benefit apitegromab upon launch?

A: David Hallal said they'll assess dynamics but feel apitegromab can help all patients independent of SMN therapy Q: Maybe just following up a little bit on Kripa's question before, just on launch trajectory. As you mentioned, the SMA population now has one 100% newborn screening in the US, they've gotten very concentrated into these centers of excellence to receive SMN therapy. Like those would seem to be kind of tailwinds to the launch relative to prior SMA launches but then, while there's certainly unmet need maybe it's not quite as dramatic as it was before the original SMN correctors launched, which would maybe be a headwind. I mean, do you think those net out and this we should look at prior SMA launches as a good proxy for the trajectory that apitegromab might have? And I guess, to the extent that you don't think those are good proxies, is there another other launches out there in the rare disease space that you think maybe are more analogous to what we should expect with apitegromab?

A: David Hallal and Keith Woods discussed urgency due to progressive motor function loss despite SMN therapies, focusing on long-term patient service Q: As we look towards the EMBRAZE data in June, could you just help frame expectations for the tirzepatide monotherapy arm? Specifically, how much of the lean mass loss that we expect at one year with tirzepatide do you think typically occurs by week 24? And related to that, do you think eight weeks of follow-up post treatment is sufficient time to demonstrate a weight regain in patients in the tirzepatide monotherapy arm?

A: Akshay Vaishnaw discussed expected lean mass loss and follow-up