Scholar Rock's Muscle Wasting Drug Excels In Pivotal Trial, Plans FDA Submission Next Year

Scholar Rock Holding (NASDAQ:SRRK) stock is trading higher on Monday after the company released topline results from the Phase 3 SAPPHIRE trial of apitegromab in spinal muscular atrophy.

The study achieved its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement for apitegromab versus placebo in motor function in SMA patients on chronic dosing of standard-of-care therapies.

Standard therapies include Biogen Inc‘s (NASDAQ:BIIB) Spinraza (nusinersen) and Roche Holdings AG‘s (OTC:RHHBY) Evrysdi (risdiplam).

In the main efficacy population (ages 2-12), the mean ...

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