Apitegromab shows sustained benefits in phase 2 SMA trial

Cambridge, Massachusetts–based biopharma Scholar Rock has been making encouraging progress in the development of apitegromab, a potential new therapy for spinal muscular atrophy (SMA), a genetic disease characterized by progressive muscle weakness and atrophy. Apitegromab, a novel monoclonal antibody, works through a unique mechanism of action – it binds to and inhibits the precursor form of myostatin, specifically targeting inactive promyostatin and latent myostatin. By preventing activation of myostatin, apitegromab enables increased muscle growth and strength. 

The company’s recently completed phase 2 TOPAZ trial demonstrated that apitegromab, a novel monoclonal antibody inhibiting myostatin, improved motor function and increased muscle mass and strength compared to placebo in SMA patients across multiple outcome measures. Further data from a 36-month extension of the trial provided strong evidence for the sustained effectiveness and safety of apitegrom…

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