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.
In the SAPPHIRE study, patients treated with apitegromab showed an average increase of over 3 points in the Hammersmith Functional Motor Scale Expanded (HFMSE) at week 52. In contrast, only 12.5% of those on placebo achieved similar improvement. Patients on apitegromab began to experience enhancements in motor function as early as week 8, which persisted across the 52-week study period.
Given these encouraging data, Scholar Rock plans to submit regulatory applications for apitegromab to the U.S. FDA and the European Medicines Agency (EMA) in the first quarter of 2025. This could introduce new treatment options for SMA patients and drive further growth for Scholar Rock in the biopharmaceutical sector.
