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 in Indiana, now owned by Novo Nordisk; a Type A meeting with FDA in November 2025 established a collaborative remediation plan.

• FDA issued a warning letter to Novo Nordisk, which responded promptly, leading to a positive site visit and plans for a formal re-inspection after resumption of manufacturing.

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

Manufacturing and supply chain

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

• Confident that both facilities will support a 2026 launch, with sufficient drug inventory already vialed and ready for market release.

• Existing supply supports ongoing clinical trials and early access programs, with no need for additional manufacturing before launch.

• Second facility expected to release product in 2026, further supporting supply chain resilience.

Commercial readiness and launch strategy

• Commercial team hired, trained, and deployed, focusing on disease awareness and education at 140 SMA treatment centers.

• Built a nationwide home infusion network and established ongoing payer engagement to facilitate access and reimbursement.

• Launched patient assistance and disease awareness programs to support uptake and patient navigation at launch.

• Anticipates initial reimbursement challenges but expects strong demand and conversion rates post-approval.