NasdaqGS:SRRKBiotechs
Is Phase 3 Apitegromab Success and FDA Priority Review Changing the Investment Case for Scholar Rock (SRRK)?
Scholar Rock announced that pivotal Phase 3 SAPPHIRE trial results for its investigational SMA treatment apitegromab were published in The Lancet Neurology, showing improved motor function in children and adults with SMA compared to placebo, with all patients continuing on standard SMN-targeted care.
This marks apitegromab as the first muscle-targeted therapy candidate in SMA to show clinical success in a pivotal phase 3 trial, and the treatment is currently under FDA priority review with a...