Scholar Rock (SRRK) Q4 FY25 earnings call: guidance and Q&A

Is Novo ready for reinspection, and what specific confidence is needed from the reinspection to trigger the BLA resubmission?

Management is gratified by the FDA and Novo's urgency. The gating item is a reinspection following routine manufacturing activities, which have resumed. A successful reinspection will trigger a rapid BLA resubmission, and the company is confident given the progress.

“We are at the ready to submit our BLA submission very, very quickly. But it really would be with some level of confidence that it was a successful reinspection.”

asked by Eric Schmidt · answered by David Hallal

If the backup fill-finish facility becomes the primary, how would that affect the 2026 timelines, given the supplemental filing is guided for later in the year?

The company views the second facility as an important effort regardless. While the plan is for it to be a supplemental BLA, management does not believe the timing would be tremendously altered if it needed to become the primary, indicating preparedness to pivot.

“We don't really believe that, that timing would be altered tremendously in terms of if it were not an sBLA. So we've thought about it. It is our plan that it will be an sBLA.”

asked by Tazeen Ahmad · answered by David Hallal

What was the purpose of the FDA sending a field team to Catalent Indiana, and what are the next procedural steps and communication methods post-reinspection?

The field team visit demonstrates high FDA priority and engagement, following a constructive meeting with Novo. Management expects the rapid pace of progress to continue for reinspection timing, resubmission, and review, but cannot comment on specific communication methods (verbal vs. written).

“we do think what it shows is for just weeks after a really constructive meeting with Novo in early Q1, where there were no new requests by the FDA of Novo into their remediation plan. We think it just continues to show high priority by the FDA to send a field team out to interact with the site.”

asked by Tessa Romero · answered by David Hallal

What are the expectations for BLA turnaround time post-submission, and what is the strategy for SRK-439 (in-house vs. partnership, rare vs. broader application)?

For BLA timing, the sole approvability issue was the Catalent facility, and management points to the rapid progress over the last three months as an indicator. For SRK-439, it's a high-potency, proprietary asset, currently intended to be kept in-house for rare neuromuscular diseases, with further plans to be shared after Phase I data.

“439 is a subcutaneously administered myostatin inhibitor binding to both pro and latent myostatin with high affinity and selectivity. We recently presented data demonstrating that 439 is 10x more potent than apitegromab.”

asked by Ryan Mcelroy · answered by David Hallal, Akshay Vaishnaw

Can the 2026 launch still happen with a Class II submission, and was there any indication of a Commissioner's Priority Voucher (CNPV) for apitegromab?

Management is comfortable with 2026 guidance, noting that Class II submissions can be decided before the full 6-month timeline, citing Regeneron as an example. The sole approvability issue remains the Catalent facility. No specific comment on CNPV, but they are in close communication with the FDA on all initiatives.

“even when Class IIs are sort of granted, oftentimes, the decision is taken up before that 6-month time line.”

asked by Srikripa Devarakonda · answered by David Hallal

What are the expectations for the apitegromab label regarding ambulatory/non-ambulatory patients and age subgroups, and how should weight-based dosing impact pricing models?

The original PDUFA cycle had advanced draft label discussions. The final label will depend on FDA, but guiding principles include trial design (covering non-ambulatory, ambulatory, children 2+, risdiplam/nusinersen patients) and broad applicability. Pricing will be discussed at launch, but it will be weight-based, leading to a range, and will consider SMA's rarity, severity, and apitegromab's clinical benefits.

“we anticipate that it would be relatively straightforward to get aligned with the FDA on the final label after our BLA resubmission.”

asked by Michael Yeh · answered by Akshay Vaishnaw, Robert Keith Woods

What commercial analogs should be considered for initial uptake and launch trajectory, and what is the likely approval path for subcu apitegromab (full study, bridging study, human factors)?

Management expects robust demand for apitegromab due to high unmet need, but anticipates initial access speed bumps from payers and J-code. The long-term opportunity is significant. For subcu apitegromab, Phase I data show viability. The company aims to leverage existing IV PK/PD data for regulatory alignment on a path forward, but cannot guide timelines yet.

“we believe robust demand, but we think that will be met with initially some access speed bumps that could impact our launch curve.”

asked by Amy Li · answered by David Hallal, Akshay Vaishnaw

Would the second fill-finish facility completely derisk the supply chain for US and EU launch, and what specific leading indicators are being tracked for payer and physician readiness?

The second fill-finish facility, if relied upon solely, would derisk US and EU commercial opportunities. For launch readiness, the company is pleased with access to national and regional payers, and Medicare/Medicaid, having in-depth discussions and sharing SAPPHIRE data. Market research shows neurologists desire multiple modalities for SMA treatment.

“Should we rely on this facility solely, we are confident that we would be derisking as well our U.S. and EU commercial opportunities.”

asked by [Indiscernible] on for Jeff Meacham · answered by David Hallal, Robert Keith Woods

Will the European market also be served by the Novo Catalent Indiana facility, and has EMA taken any actions in response to FDA inspection findings?

There is mutual recognition between FDA and EMA, so progress with FDA on Catalent Indiana serves the MAA review well. The continued remediation and eventual successful reinspection will support the EMA decision. EMA is fully aware and supportive, awaiting resolution.

“There is a mutual recognition between both FDA and EMA. And so this steady and rapid progress we're making with FDA actually serves us very well for the current MAA review with regulators.”

asked by Salvatore Caruso · answered by David Hallal, Akshay Vaishnaw

Has FDA requested additional safety data that could extend review, and will the FSHD Phase II study look at functional endpoints, potentially making SRK-439 more appropriate for FSHD long-term?

The November meeting aligned on safety database updates for BLA resubmission, so no issues are foreseen. For FSHD, the Phase II study's primary endpoint is lean muscle volume, but it will include quantitative myometric testing to evaluate functional impact, which is a validated approach. SRK-439's high potency makes it interesting for rare neuromuscular diseases, including potentially FSHD, but it's currently proprietary.

“The primary endpoint will focus on increasing lean muscle volume, measured very sensitively by imaging techniques. But we will have on-site biomonitoring there, which is a validated approach in FSHD to understand the functional impact of any potential change in muscle mass.”

asked by Etzer Darout · answered by David Hallal, Akshay Vaishnaw

Is there precedent for FDA taking less than 6 months for a Class II resubmission, and does 2026 launch guidance assume a full 6-month review? What are OPAL enrollment trends?

Yes, there is precedent; Regeneron's 2023 CRL at the same facility was approved within ~60 days, despite being a Class II resubmission. Management is comfortable with 2026 guidance. OPAL enrollment is going very well, driven by high awareness and appreciation for a muscle-based approach in the SMA community, with good progress in enrolling infants and toddlers.

“in 2023, at the same facility, Regeneron did have a CRL, had a resubmission. I believe it was a Class II resubmission and yet it was approved within essentially sort of a 60-day window.”

asked by Allison Bratzel · answered by David Hallal, Akshay Vaishnaw

How does apitegromab aim to address the historical hurdle of previous myostatin inhibitors increasing muscle mass without meaningful functional improvement in FSHD, and what would be clinically meaningful functional improvement?

Previous inhibitors lacked potency/selectivity or were locally applied. Apitegromab, with its potency and selectivity, is expected to show global effects. Global strategies like intense physical therapy or anabolic agents have shown both muscle mass and functional capacity increases. The FORGE Phase II study will use quantitative myometric testing to evaluate functional change, alongside lean muscle volume as the primary endpoint.

“globally applied strategies like intense physical therapy or anabolic agents that increase muscle mass... clearly show an increase in muscle mass and also increase in functional capacity.”

asked by Gugan Raghuraman · answered by Akshay Vaishnaw