Scholar Rock announces Phase 3 SAPPHIRE data

Scholar Rock (SRRK) announced that new efficacy and safety data from the Phase 3 pivotal SAPPHIRE trial will be presented in multiple clinical presentations at the 2025 Muscular Dystrophy Association, MDA, Clinical & Scientific Conference in Dallas, Texas. The Phase 3 SAPPHIRE trial evaluated the efficacy and safety of apitegromab, an investigational muscle-targeted therapy that is being developed to provide clinically meaningful improvement in motor function for people living with SMA who are receiving SMN-targeted treatments. Primary endpoint showed clinically meaningful improvement in patients with SMA receiving apitegromab as measured by gold standard Hammersmith Functional Motor Scale Expanded versus placebo, with consistent outcomes across all major sub-groups including age, SMN-targeted background therapy, and age at initiation of SMN-targeted therapy