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Scholar Rock to Present Apitegromab Clinical Data from the TOPAZ Phase 2 Trial in Patients with Type 2 and 3 Spinal Muscular Atrophy at the 2021 Virtual SMA Research & Clinical Care Meeting
- Lead principal investigator, Dr. Thomas Crawford, to present TOPAZ results showing apitegromab’s therapeutic potential to improve motor function in
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n- Lead principal investigator, Dr. Thomas Crawford, to present TOPAZ results showing apitegromab’s therapeutic potential to improve motor function in patients with Spinal Muscular Atrophy (SMA)\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 an oral presentation of apitegromab TOPAZ Phase 2 clinical trial results at the 2021 Virtual SMA Research and Clinical Care Meeting, being held in conjunction with Cure SMA’s Annual SMA Conference June 7-11, 2021.\n\nDetails for the virtual Cure SMA oral presentation are as follows:\n\n\nTitle: TOPAZ: A Phase 2 Study to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy (Type 2 and Type 3 SMA): Topline Results\n\n\nPresenter: Thomas Crawford, M.D., Co-Director, Muscular Dystrophy Association Clinic and Professor of Neurology, Johns Hopkins Medicine (Lead TOPAZ Principal Investigator)\n\n\nClinical Drug Development Session: Virtual oral presentation on June 11, 2021 at 2:00pm CST\n\n\nScholar Rock recently announced positive 12-month top-line data from the TOPAZ Phase 2 trial (NCT03921528), which evaluated apitegromab, a selective inhibitor of myostatin activation, in patients with Type 2 and 3 SMA. The results show the transformative potential of apitegromab to further improve motor function in patients with non-ambulatory Type 2 and 3 SMA who were already receiving chronic maintenance therapy with a background SMN upregulator (nusinersen). A Phase 3 registrational trial in patients with non-ambulatory Type 2 and Type 3 SMA is anticipated to initiate by the end of 2021.\n\nAbout Apitegromab\n\nApitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in ...