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…

Scholar Rock recently announced Q2 results, highlighting continued progress in its Phase 3 SAPPHIRE trial for the selective myostatin-inhibitor apitegromab in spinal muscular atrophy (SMA). SAPPHIRE is a randomized, double-blind, placebo-controlled study. The Cambridge, Massachusetts–based company also shared new data from its Phase 2 TOPAZ trial focused on apitegromab that showed significant and sustained improvement…