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Scholar Rock's Apitegromab Succeeds in Phase 3 SMA Trial with p=0.019, Stock Soars 200%

Oct 7, 2024, 12:01 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 trial, which achieved statistical significance with a p-value of 0.019, demonstrated a clinically meaningful improvement in motor function among patients. As a result, Scholar Rock's stock soared more than 200%. The late-stage study involved patients already on standard-of-care treatments such as Spinraza and Risdiplam. The success of the trial positions Scholar Rock as a potential key player in the SMA treatment market.