Scholar Rock (SRRK) Shares Surge on Successful SMA Drug Trial

Group 1 - Scholar Rock Holding Corp. (SRRK) shares increased by 335% following positive Phase 3 trial results for their drug apitegromab, which targets spinal muscular atrophy (SMA) and significantly improves motor function in patients [1] - In the SAPPHIRE study, patients receiving apitegromab experienced an average increase of over 3 points in the Hammersmith Functional Motor Scale Expanded (HFMSE) at week 52, while only 12.5% of placebo patients achieved similar improvements [2] - Patients on apitegromab began to see enhancements in motor function as early as week 8, with these improvements sustained throughout the 52-week study [2] Group 2 - Scholar Rock plans to submit regulatory applications for apitegromab to the U.S. FDA and the European Medicines Agency (EMA) in Q1 2025, potentially providing new treatment options for SMA patients and fostering growth in the biopharmaceutical sector [3]