Scholar Rock Holding Corporation (SRRK) Earnings

Management highlights

- **Lead Candidate Regulatory Progress (Epidigromab for SMA)** - The company is awaiting a U.S. FDA decision on Epidigromab, the first-in-class muscle-targeted therapy for spinal muscular atrophy (SMA), with a PDUFA decision date of September 30, 2026. - The original complete response letter (CRL) from September 2025 cited only GMP observations at the Novo Nordisk-owned Catalent Indiana fill-finish facility as the sole approvability issue. - The company resubmitted the BLA in March 2026 with two independent fill-finish facilities (Catalent Indiana and a second qualified facility), creating two separate approval paths to launch. - More commercial vials of Epidigromab are currently available from the second fill-finish facility than from Catalent Indiana, and vials from both facilities are already at third-party packaging sites awaiting approval. - In Europe, the MAA for Epidigromab currently only includes the Catalent Indiana facility, and the EMA is awaiting the FDA's inspection classification for this site. The company is actively engaging with European regulators to add the second fill-finish facility to the MAA, which has already passed recent FDA and EMA inspections. - **Pipeline Progress** - Enrollment is robust in the Phase II OPAL study evaluating Epidigromab in infants and toddlers with SMA under 2 years old, including patients who have already received SMN-targeted therapies. - The company has initiated the randomized, double-blind, placebo-controlled Phase II FORGE study of Epidigromab for facioscapulohumeral muscular dystrophy (FSHD), an inherited neuromuscular disease with no currently approved therapies. The trial will enroll 60 patients with mild to moderate disease. - Phase 1 study of SRK439, a novel high-potency anti-myostatin inhibitor, is progressing well with healthy volunteers, and top-line data is expected by the end of 2026. - A high-concentration subcutaneous formulation of Epidigromab has demonstrated favorable bioavailability and pharmacodynamic profiles comparable to the IV formulation in Phase 1 testing; regulatory engagement is planned after approval of the IV Epidigromab candidate. - **Commercial Readiness** - The U.S. commercial organization is fully launch-ready across all functions, prepared to support patients, caregivers, and prescribers immediately upon FDA approval. - The U.S. field team has already engaged with approximately 140 SMA treatment centers and 2,600 prescribing physicians, establishing center-specific treatment workflows for post-approval access. - The Scholar Rock Supports patient services program is fully staffed and trained to provide insurance navigation, financial assistance, and treatment logistics support for eligible patients at launch. - Reimbursement discussions are complete with most national and regional U.S. payers, as well as Medicare and Medicaid, to support broad access post-approval. - European launch preparations are focused on an initial launch in Germany, with buildout of a 50-country operating platform underway to serve the estimated 35,000 SMA patients globally who have already received SMN-targeted therapies. - **Financial Position** - Disciplined capital allocation has allowed the company to increase commercial launch investments while maintaining operating expense levels consistent with the prior year, and continuing to invest in pipeline development.

Guidance

- The company reaffirms that the FDA will complete review of the resubmitted Epidigromab BLA by the September 30, 2026 PDUFA date, and the firm expects no timeline delays regardless of whether one fill-finish facility is removed from the application. - Top-line data from the Phase 1 study of SRK439 is expected to be released by the end of 2026. - Regulatory engagement for the high-concentration subcutaneous Epidigromab formulation will begin immediately after approval of the IV candidate, with further updates provided after discussions with regulators. - The company maintains that it has sufficient cash to support operations through the Epidigromab launch and ongoing pipeline progress, with additional liquidity available via a $150 million existing debt facility that can be drawn down post-approval, and plans to monetize a priority review voucher to further strengthen the balance sheet.

Segment performance

Scholar Rock is a clinical-stage biotech focused on neuromuscular disease therapies, and the firm does not report separate product segment financial performance as Epidigromab, its lead candidate, has not yet launched. For the second quarter of 2026, the company reported total operating expenses of $108.9 million, which included $19.7 million in non-cash stock-based compensation. Excluding stock-based compensation, total operating expenses were $89.2 million, which was generally consistent with operating expense levels in the second quarter of 2025. As of the end of the second quarter of 2026, the company held $492 million in cash, cash equivalents, and marketable securities, including $63 million in net proceeds from the firm's at-the-market (ATM) equity program completed during the quarter. There are no revenue-generating product segments to report at this time.

Risks & headwinds

- The FDA's inspection classification for the Catalent Indiana facility, originally expected by the end of July 2026 (90 days after the April 2026 reinspection), is still pending, which creates minor uncertainty around the regulatory timeline. - If the FDA maintains the current OAI classification for Catalent Indiana, the company will need to withdraw the facility from the BLA, but management confirms this will not impact the September 30 PDUFA timeline as the second facility provides a fully independent approval path. - Post-launch, prior authorization requirements from payers may lead to delays in patient access, and initial denials and subsequent appeals can extend the timeline from prescription to patient treatment to an average of more than 60 days in the first six months of launch before a permanent J-code is established. - European approval timelines for Epidigromab are currently dependent on FDA's classification of Catalent Indiana, and the process to add the second fill-finish facility to the MAA will require additional regulatory review, with timelines still to be confirmed with the EMA.

Analyst Q&A

• Q: How confident is management that the second fill-finish facility alone can support approval by the PDUFA date, and what progress has the FDA made in reviewing its data package?

  A: Management confirmed that the FDA aligned with the company's two-facility approach in the March 2026 Type C meeting, and all required data for the second facility has been submitted with review progressing well. The second facility has passed recent successful routine GMP inspections by both the FDA and EMA, with no post-approval inspections required for recent product approvals, demonstrating strong operational performance. If Catalent Indiana needs to be withdrawn from the application, management confirmed this would not impact the September 30 PDUFA timeline, and the company already has more commercial vials from the second facility than Catalent Indiana at packaging sites awaiting approval.

• Q: If Catalent Indiana retains its OAI classification, is there a set deadline to withdraw the facility that would avoid delaying the PDUFA?

  A: Management stated that the company is in ongoing open correspondence with the FDA, and both parties have pre-agreed on the process for withdrawing the facility if needed. There is no expected impact to the September 30 timeline even if withdrawal is required, as the FDA has already pre-cleared this process. The 90-day inspection classification deadline is only guidance, not a statutory requirement, and the FDA is taking additional time to review Novo Nordisk's robust response to the inspection observations, which is not unexpected.

• Q: What remaining procedural steps are needed between now and September 30 for approval, and is there any reason to expect the FDA will not complete review by the PDUFA date?

  A: Management confirmed that the company has followed all the FDA's guidance step-by-step since the November 2025 Type A meeting, and all required submissions for the second fill-finish facility are complete. The FDA's review of the second facility is progressing well, and management sees no reason the FDA cannot complete review and issue an approval by or before the September 30 PDUFA date. The company remains grateful for the FDA's collaborative and expedited engagement given the high unmet need for SMA therapies.

• Q: Is the second fill-finish facility a fully independent approval path, and what regulatory work is needed to advance the subcutaneous Epidigromab formulation?

  A: The second fill-finish facility provides a fully independent approval path, with all details agreed upon with the FDA in the March 2026 Type C meeting. For the subcutaneous formulation, all required briefing documents for regulators are being finalized and will be submitted immediately after approval of the IV Epidigromab candidate. Management will engage with the FDA to align on the development pathway (either a PK/PD bridging path or a full pivotal trial) and will provide a timeline update after receiving regulatory guidance.