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Scholar Rock Heading To Finish Line In SMA With Positive Pivotal Data

 
• By Joseph Haas

Phase III success positions Scholar Rock’s selective myostatin inhibitor for regulatory filings in early 2025. Analysts see a blockbuster opportunity for additive therapy in spinal muscular atrophy.

Idea takes off
Clinical-stage Scholar Rock may be close to commercial takeoff with Phase III SMA data • Source: Shutterstock

Scholar Rock, LLC unveiled Phase III data on 7 October that appears to make a strong case for its selective myostatin inhibitor apitegromab as additive therapy that can improve motor function in patients with spinal muscular atrophy, a rare neuromuscular disease in which patients typically experience decline in motor function on standard of care.

In a top-line readout from its Phase III SAPPHIRE trial, Scholar Rock noted that apitegromab produced a 1.8-point improvement from...

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