Scholar Rock announced it will present a comprehensive update on its Phase 3 SAPPHIRE clinical trial data at Cure SMA’s Annual SMA Research & Clinical Care Meeting, held from June 25-27, 2025. The presentation will detail the efficacy and safety of apitegromab in individuals with Type 2 and Type 3 spinal muscular atrophy.
The company previously shared positive topline data from the SAPPHIRE trial in October 2024, which demonstrated a statistically significant 1.8-point improvement in motor function. The U.S. FDA accepted the Biologics License Application (BLA) for apitegromab under priority review in March 2025, with a PDUFA target action date of September 22, 2025.
The announcement also highlighted the positive Phase 2 EMBRAZE trial results, where apitegromab demonstrated statistically significant preservation of lean mass during tirzepatide-induced weight loss. This reinforces the broad potential of Scholar Rock's myostatin inhibition platform across multiple therapeutic areas.
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