World Health Organization's official publications

Scholar Rock's Apitegromab Succeeds in Phase 3 SMA Study, Stock Surges Over 300% with p=0.019

Oct 7, 2024, 12:20 PM

Scholar Rock announced that its drug apitegromab met the primary endpoint in a Phase 3 SAPPHIRE study for treating spinal muscular atrophy (SMA). The study demonstrated a statistically significant and clinically meaningful improvement in motor function among patients, with a p-value of 0.019. The trial design included patients with SMA type2/3 who were already on existing treatments such as Spinraza and Evrysdi, despite lowered statistical power for individual arms. Following the positive trial results, Scholar Rock's stock surged over 300%. The successful outcome of this late-stage trial positions Scholar Rock as a significant player in the biotech industry, with expectations of further regulatory approvals.