Regulatory Progress for Apitegromab
The BLA for apitegromab has been accepted under priority review by the FDA with a target action date of September 22, 2025. The company is also working towards potential approval in Europe by mid-2026.
Positive Clinical Trial Results
The SAPPHIRE trial showed statistically significant improvement in motor function in SMA patients, and the Phase II EMBRAZE study met its primary endpoint, demonstrating a 54% greater lean mass preservation with an encouraging safety profile.
Commercial Launch Preparations
The U.S. customer-facing team is fully on board and trained, ready to launch apitegromab following FDA approval. Preparations for a European launch are also underway for 2026.
Financial Position and Runway
Scholar Rock ended the quarter with $295 million in cash, with an anticipated runway into 2027. Additional funding options include a $50 million debt facility and expected $16 million from warrant exercises.