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Scholar Rock to Present New Apitegromab Data Including 24-Month Efficacy and Safety Data from TOPAZ Phase 2 Trial at the 2022 Annual Cure SMA Conference
- 24-month apitegromab data from Phase 2 TOPAZ trial presented by Thomas Crawford, M.D. - Also presenting patient and caregiver observations from real-world
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n- 24-month apitegromab data from Phase 2 TOPAZ trial presented by Thomas Crawford, M.D.\n\n- Also presenting patient and caregiver observations from real-world evidence study of apitegromab treatment benefits on daily living\n\n-Scholar Rock to host webcast on June 17, 8:30am 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 that the company will deliver two podium presentations on 24-month data from the TOPAZ Phase 2 clinical trial at the 2022 SMA Research and Clinical Care meeting, which is being held in conjunction with Cure SMA’s Annual SMA Conference (June 15-19, 2022).\n\n“We are eager to present new data at this year’s Annual SMA Conference, in particular the 24-month efficacy and safety results from our TOPAZ trial, as well as the initial evaluation of patient treatment benefits of apitegromab on impacts to quality of life, such as social and daily living activities,” said Nagesh Mahanthappa, Ph.D., Founding Chief Executive Officer of Scholar Rock. “With these data and insights, we continue to build strong clinical evidence around the potential of apitegromab for the treatment of spinal muscular atrophy, a disease where significant unmet need still exists.”\n\nDetails of the podium presentations are as follows:\n\nTitle: Treatment Effects Among Patients with Type II and Type III SMA Directly Reported by Patients and Caregivers from the TOPAZ Clinical Trial\nPresenter: Hemal Shah, PharmD, an independent HEOR consultant for Scholar Rock, and CEO of Value Matters, LLC.\nModerator: Thomas Crawford, M.D., lead principal investigator of the TOPAZ trial and Professor of Neurology and Pediatrics; Johns Hopkins University\nClinical Research Session Thursday, June 16th, 4:10 PM - 4:30 PM PST (Abstract #21)\n\nTitle: TOPAZ Extension: 24-Month Efficacy and Safety of Apitegromab in Patients with Later-Onset Spinal Muscular Atrophy (Type 2 and Type 3 SMA)\nPresenter: Thomas Crawford, M.D., lead principal investigator of the TOPAZ trial and Professor of Neurology and Pediatrics; Johns Hopkins University.\nModerator: Katherine Klinger, Ph.D., Cure SMA Scientific Advisory Board Member and Translational Sciences Global Head, Sanofi S.A.\nClinical Drug Deve...