Scholar Rock announced it will present additional data from its Phase 3 SAPPHIRE clinical trial at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in March. The conference will feature a detailed analysis of apitegromab's safety and efficacy in spinal muscular atrophy (SMA) patients.
The company will also present preclinical data on an investigational approach combining a preclinical version of apitegromab with a dystrophin corrector for addressing muscle defects in a Duchenne muscular dystrophy (DMD) model. This highlights potential future applications of its muscle-targeted therapy platform.
These presentations build upon the positive topline data from the SAPPHIRE trial, which was previously announced in October 2024, and the Biologics License Application (BLA) submission to the U.S. FDA in January 2025.
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