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