Scholar Rock Archives - Medical Design Sourcing

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…

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 in Hammersmith Functional Motor Scale-Expanded (HFMSE) scores at 24 months for non-ambulatory patients. The HFMSE scores continued to improve for patients who moved from 2 mg/kg to 20 mg/kg in Cohort 3 in the second year of the study. In addition, patients continued to see increases in Revised Upper Limb Module (RULM) scores at 24 months.

The HFMSE is an assessment for spinal muscular atrophy types 2 and 3 comprising 33 distinct activities scored 0, 1 or 2. HFMSE measures common activities such as lying and rolling, crawling, kneeling and standing…

Scholar Rock

Apitegromab shows sustained benefits in phase 2 SMA trial

Scholar Rock sees continued progress with apitegromab in spinal muscular atrophy