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Scholar Rock Looks To Augment Existing Therapies In Spinal Muscular Atrophy

 
• By Joseph Haas

Phase III study under way may lead to filing of drug meant to address muscular component of the rare disease, with existing therapies such as Spinraza targeting neurological aspects.

Muscle atrophy
Scholar Rock's approach to SMA focuses on the muscle-function aspects of the disorder • Source: Shutterstock

Scholar Rock, LLC says it hopes to bring its myostatin activation inhibitor apitegromab to market as an additive muscular function therapy for spinal muscular atrophy in 2025, with plans to commercialize the drug itself in the US and Europe – not an easy feat for a small biotech in a big pharma-dominated indication, but executives hope their drug will address a gap in treatment.

Cambridge, MA-based Scholar Rock also has SRK-181 in Phase I testing a theory that it can address checkpoint inhibitor resistance in multiple cancer types

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