Scholar Rock's Phase 3 SAPPHIRE Study Of Apitegromab In SMA Meets Primary Goal; Stock Surges

(RTTNews) - Scholar Rock Holding Corporation (SRRK), a late-stage biopharmaceutical company, Monday said its Phase 3 SAPPHIRE study evaluating apitegromab in patients with spinal muscular atrophy (SMA) met primary goal. Following the news, the company's shares jumped more than 200 percent in pre-market.

The primary endpoint of the study was statistically significant and clinically meaningful improvement in motor function in SMA patients treated with apitegromab compared to placebo. Further, treatment with apitegromab was well-tolerated across all age groups, and no new safety findings were observed in the trial.

The Company plans to submit a U.S. Biologics License Application (BLA) and a European Union marketing authorisation application (MAA) in the first quarter of 2025.

Scholar Rock had closed at $$7.42, up 4.95 percent on Friday. It has traded in the range of $6.48 - $21.17 in the last 1 year.