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 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…

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