Shares of Scholar Rock Holding Corp. (SRRK, Financial) soared by 335% as their candidate drug for spinal muscular atrophy (SMA), apitegromab, achieved the primary endpoint in a Phase 3 trial. The positive results indicate that the apitegromab monoclonal antibody significantly improves motor function in patients.
Apitegromab met the primary endpoint in its phase 3 SAPPHIRE study for spinal muscular atrophy, setting up BLA & MAA filings in Q1 2025. Apitegromab is the first treatment targeting muscle fiber atrophy in SMA, potentially changing the standard of care and offering a competitive edge. Scholar Rock also has apitegromab being explored in a phase 2 study for obesity, with clinical data expected in Q2 2025, presenting another catalyst.
U.S. stocks traded lower midway through trading, with the Dow Jones index falling more than 200 points on Monday.
The Phase 3 trial of Apitegromab showed statistically significant improvement in motor function.