Positive Phase 3 SAPPHIRE Trial Results
The SAPPHIRE trial showed statistically significant and clinically meaningful improvement in motor function for SMA patients using apitegromab, with a threefold higher chance of improvement versus placebo.
FDA Priority Review and Upcoming Launch
The FDA granted priority review for apitegromab with a PDUFA date set for September 22nd, indicating significant potential impact for SMA treatment.
Global Launch Strategy for Apitegromab
Efforts are underway for a global launch of apitegromab, beginning with the US in Q3 2025, followed by Europe, Asia Pacific, and Latin America.
Strong Financial Position
Scholar Rock ended the quarter with $364.4 million and an additional $100 million available through a debt facility, ensuring financial runway into 2027.
Exploration of New Indications
Scholar Rock is evaluating the use of apitegromab in other neuromuscular disorders and advancing SRK-439 for clinical trials, with an IND filing expected in Q3.