Scholar Rock Holding Corporation Q4 FY2025 earnings call

David Hallal discussed the company's priorities for 2026, including bringing upitigramab to the SMA community, progress with the regulatory process, commercial launch preparations, and progress in advancing the anti-myostatin pipeline. Akshay Vaishnaw provided an R&D update, including progress in the Phase 2 OVAL study, IND clearance for upitigromab in FSHD, and updates on subQ formulation of epitogromab and SRK four thirty nine. R. Keith Woods discussed commercial launch preparations, including disease education initiatives, specialty pharmacy network expansion, patient access support program, and launch readiness in European markets. Vikas Sinha provided financial results, noting operating expenses and the new debt facility secured.

Reaffirmed guidance of BLA resubmission and U.S. launch following approval in 2026; anticipate filing a supplemental BLA for the second fill-finish facility later this year; expect a decision from the European Medicines Agency on the MAA for upitigromab in mid-2026

Q: Can you elaborate on whether Novo is now ready for reinspection and what triggers resubmission?

A: Novo is making progress with remediation, gating item is reinspection following routine manufacturing activities, trigger is successful reinspection.

Q: What would happen to timelines if reverting to backup facility?

A: Assumption is timing wouldn't be altered tremendously.

Q: Elaborate on FDA sending field team and post-reinspection steps?

A: Field team shows high priority, progress continues, reinspection to follow routine manufacturing.

Q: Expectations for turnaround time following BLA submission?

A: No specific timing commented but progress is positive.

Q: Strategy for SRK four thirty nine?

A: Plan to keep in house, share further development plans after Phase 1 data.

Q: Timelines for inspection, BLA resubmission, U.S. launch?

A: Continue to expect in 2026, steady progress.

Q: Label expectations and pricing?

A: Label details to be finalized with FDA, pricing to be discussed on launch call.

Q: Commercial analogs and subcu api approval?

A: Commercial analogs have interest, subcu api regulatory strategy to be discussed with FDA.

Q: Second fill finish facility and payer/physician readiness indicators?

A: Second facility derisks supply chain, tracking payer and physician engagement.

Q: MAA review and EMA action?

A: Progress with FDA supports MAA review.

Q: FDA requesting additional safety data and FSHD Phase 2 study?

A: BLA resubmission will include updated safety data, FSHD Phase 2 study focuses on muscle volume and function.

Q: Time to profitability and financing?

A: Anticipate 2-3 years from launch, will weigh profitability vs investing.

Q: Review timing and OPAL enrollment?

A: FDA has shown quick review for Class 2, OPAL enrollment going well.

Q: How epitigramab addresses historical hurdle in FSHD?

A: Incorporates contact myometric testing to evaluate muscle function, primary endpoint is muscle volume change.