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Scholar Rock Presents Clinical and Preclinical Data for SRK-015 at the 2020 Virtual Cure SMA Research and Clinical Care Meeting
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 presentations as part of the 2020 Virtual Cure SMA Research and Clinical Care Meeting. The two presentations will review previously presented data from preclinical studies, the Phase 1 healthy volunteer trial, as well as preliminary pharmacokinetic and biomarker data and baseline 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).\n\n\nDetails for the two virtual presentations at the meeting are as follows:\n\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: A Phase 2 Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy (TOPAZ): An Introduction\n\n\nClinical Drug Development Session: Virtual presentations on June 12, 2020 at 12:00-3:15pm CST.\n\n\n\nAbout SRK-015\n\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 strength in multiple animal species(1). Preclinical SMA mouse model studies have demonstrated that selective inhibition of myostatin may have therapeutic potential in improving muscle strength(2). Scholar Rock believes by targeting the precursor forms of myostatin, and preventing the release of the mature growth factor, SRK-015 may promote a clinically meaningful improvement in motor function. 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 granted Orphan Drug Designation (ODD), and the European Commission (EC) has granted Orphan Medicinal Product Designation, to SRK-015 for the treatment of SMA. The effectiveness and safety of SRK-015 have not been established. SRK-015 has not been approved for any use by the FDA or any other ...