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Scholar Rock Presents Data for SRK-015 at the World Muscle Society 2020 Virtual Congress
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in

About this update from Scholar Rock Holding Corporation
[{"type":"text","content":" 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 two poster presentations as part of the World Muscle Society (WMS) 2020 Virtual Congress. The SRK-015 clinical development poster will showcase previously presented data from the Phase 1 healthy volunteer trial, as well as baseline characteristics and demographics from the TOPAZ Phase 2 proof-of-concept trial evaluating SRK-015 for the treatment of patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA). The second poster shares data on the pharmacologic effects of SRK-015 in patients with SMA, healthy volunteers, and across animal species.\n\nDetails for the two virtual posters at the WMS meeting are as follows:\n\n\nTitle: Clinical Development of SRK-015, a Fully Human Anti-proMyostatin Monoclonal Antibody, for the Treatment of Later-Onset Spinal Muscular Atrophy\n\n\nTitle: Myostatin Dynamics in Health and Disease: Pharmacologic Effects of SRK-015, a Highly Selective Monoclonal Antibody Inhibitor of Myostatin Activation\n\n\nVirtual poster presentations on October 1, 2020 at 17:30-19:30 BST.\n\n\nThe SRK-015 TOPAZ Phase 2 clinical trial is fully enrolled, and a 6-month interim efficacy and safety analysis is planned for the fourth quarter of 2020 with top-line data for the 12-month treatment period expected in the first half of 2021. As of September 25, 2020, 22 patients across the three cohorts have completed the 12-month treatment period, and all 22 have opted into the 12-month extension period.\n\nAbout SRK-015\n\nSRK-015 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(1). Scholar Rock believes the inhibition of the activation of myostatin with SRK-015 may promote a clinically meaningful increase in muscle strength(2). A Phase 2 clinical trial in patients with Type 2 and Type 3 SMA is ongoing (NCT03921528). The U.S. Food and Drug Administration (FDA) has g...