Scholar Rock (SRRK) TD Cowen 46th Annual Health Care Conference summary

Strategic vision and pipeline development

• Plans to build a global biotech leader anchored by innovative anti-myostatin therapies, with apitegromab as the lead asset for SMA and additional rare neuromuscular disorders targeted for future expansion.

• Expects 2026 to be transformative, with first regulatory approvals in the U.S. and Europe and ambitions for launches in 50 countries over the next decade.

• Ongoing development of apitegromab and SRK-439 for other severe neuromuscular diseases, aiming for perennial launches and multi-billion dollar franchise potential.

• Long-term vision includes sustained global launches and growth, leveraging a robust clinical and commercial pipeline.

Regulatory and manufacturing updates

• Initial U.S. launch for apitegromab delayed from 2025 to 2026 due to compliance issues at the Catalent Indiana manufacturing facility, now owned by Novo Nordisk.

• FDA granted priority review with a September 2025 action date, but approval was held up solely due to facility compliance, not clinical or filing issues.

• Steady progress in remediation, with positive FDA interactions, a constructive site visit, and resumed manufacturing; awaiting re-inspection before BLA resubmission.

• A second fill-finish facility is being validated to ensure supply chain resilience and maintain 2026 launch guidance even if primary facility issues persist.

• European approval is also contingent on resolution at the Indiana facility, with EMA showing flexibility and ongoing coordination with FDA.

Commercialization and pricing strategy

• Pricing will consider disease rarity, severity, and the unique clinical benefit of apitegromab, which targets muscle function in SMA, unlike existing therapies.

• Robust demand anticipated, with over 90% of clinical trial patients remaining on long-term extension and strong interest from both patients and neurologists.

• Initial launch may be gradual due to payer policies, infusion logistics, and reimbursement processes, but long-term revenue potential is seen as multi-billion dollar.