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Scholar Rock Presents Baseline Demographics Data from the SRK-015 TOPAZ Trial at the American Academy of Neurology Conference
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 a poster presentation as part of the 2020 American Academy of Neurology Science Highlights virtual platform. The presentation will include baseline demographics as well as preliminary pharmacokinetic and biomarker data 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 SRK-015 TOPAZ presentation at the conference are as follows:\n\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\n\n\nAAN Science Highlights virtual platform: Virtual presentation will be available starting May 18, 2020.\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 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 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 regulatory agency.\n\n\n\n(1) \n \n\nHan, H.Q., Zhou, Xiaolan, Goldberg, A. Myostatin/activin pathway antagonism: Molecular basis and therapeutic potential. International Journal of Biochemistry & Cell Biology, 2013, 45(10):2119-2348\n\n\n\n\n\nAbout Scholar Rock\n\n\nScholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of seri...