Scholar Rock (SRRK) Leerink Global Healthcare Conference 2026 summary

Clinical and regulatory progress

• Achieved positive phase III SAPPHIRE trial results for Apitegromab in SMA, meeting primary endpoints with significant motor function improvement in patients on SMN-targeted therapies.

• Filed for approval in January 2025 and received priority review with an action date of September 22, 2025.

• Approval delayed due to compliance issues at the fill-finish facility; a Type A meeting with FDA in November 2025 established a collaborative remediation plan.

• FDA and Novo Nordisk have shown urgency, with a positive site visit and plans for a formal re-inspection following resumption of manufacturing.

• Guidance reaffirmed for BLA resubmission and U.S. launch in 2026, pending successful re-inspection.

Manufacturing and supply chain

• Second fill-finish facility qualified as a contingency, with engineering and validation runs underway to ensure supply redundancy.

• Sufficient inventory of Apitegromab already vialed to support launch and ongoing patient needs, with no immediate need for additional manufacturing.

• Both facilities expected to support product release in 2026, aligning with launch plans and supply chain resilience.

Commercial readiness and launch strategy

• Commercial team hired, trained, and deployed, focusing on disease awareness and education at 140 SMA centers and with thousands of specialists.

• Home infusion network established with 10,000 nurses nationwide to facilitate patient access.

• Patient assistance program and enhanced disease awareness initiatives launched to support reimbursement and access.

• Expectation of strong demand at launch, with anticipated payer headwinds but confidence in eventual broad access.