Business
Scholar Rock Announces Positive 12-Month Top-Line Results From the TOPAZ Phase 2 Clinical Trial Evaluating Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
- Data further demonstrate proof-of-concept for the therapeutic potential of apitegromab in patients with Type 2 and Type 3 SMA - Phase 3 registrational
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n- Data further demonstrate proof-of-concept for the therapeutic potential of apitegromab in patients with Type 2 and Type 3 SMA\n\n- Phase 3 registrational trial initiation expected by the end of 2021 \n\n- European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation to apitegromab, recognizing unmet medical needs of patients with SMA \n\n- Scholar Rock to host webcast today at 8:00am ET\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar Rock (NASDAQ:SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced positive top-line data from the TOPAZ Phase 2 clinical trial evaluating apitegromab (SRK-015) in patients with Type 2 and Type 3 spinal muscular atrophy (SMA).\n\n“These top-line 12-month data provide further support towards establishing apitegromab as a potential first muscle-directed therapy for patients with SMA,” said Yung Chyung, M.D., Chief Medical Officer of Scholar Rock. “The findings also offer important insights into myostatin biology and our scientific approach of targeting the latent forms of growth factors. We look forward to advancing the development and investigation of apitegromab as we plan to initiate a pivotal trial in SMA by the end of 2021 and explore its potential in additional disease areas.”\n\nKey findings from the 12-month top-line analysis include:\n\n\nCohort 1: 5-21 years of age, ambulatory Type 3, 20 mg/kg dose monotherapy and in conjunction with nusinersen:\n\n\nObserved a mean change from baseline in Revised Hammersmith Scale (RHS) of a 0.3-point decline.\n\n\nMajority of patients across the cohort (57%, 13/23 of patients) maintained or improved their motor function, as reflected by a >0-point change from baseline in RHS score, and 22% of patients (5/23) attained a >3-point increase from baseline.\n\n\nResults suggest potential clinical effect in certain patients in this population.\n\n\n\n\nCohort 2: 5-21 years of age, Type 2 and non-ambulatory Type 3 who initiated nusinersen ≥5 years old, 20 mg/kg dose:\n\n\nObserved a mean change from baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) of a 0.6-point improvement.\n\n\nMajority of patients (64%, 9/14 of patients) attained a >1-point increase from baseline and 29% of patients (4/14) attained a >3-point inc...