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 sees continued progress with apitegromab in spinal muscular atrophy
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…
Scholar Rock sees stock soar after announcing positive SMA results
A monoclonal antibody that blocks the activation of the skeletal muscle protein myostatin could be novel muscle-directed therapy for spinal muscular atrophy patients. Cambridge, Mass.–based Scholar Rock (NSDQ:SRRK), which is developing the SRK-015 investigational antibody, saw its shares more than double to $30.02 apiece yesterday. SRK-015 could lead to the formation of a new treatment…