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Scholar Rock to Present Apitegromab TOPAZ Phase 2 Pharmacologic Data at the 2021 World Congress of Neurology
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 an e-poster presentation of clinical results from the TOPAZ Phase 2 trial (NCT03921528) that evaluated apitegromab in patients with Type 2 and Type 3 spinal muscular atrophy (SMA) at the 25th World Congress of Neurology (WCN) taking place October 3-7, 2021.\n\nDetails for the virtual e-poster are as follows:\n\n\nTitle: Relationship of Pharmacokinetics and Pharmacodynamics to Apitegromab Efficacy in Patients with Later-onset Spinal Muscular Atrophy (Types 2 and 3 SMA): Results from the TOPAZ Study (Poster #EP1150)\n\n\nThis poster will showcase positive correlations between the magnitude of target engagement (as measured by serum levels of latent myostatin) and motor function improvements following treatment with apitegromab.\n\n\n\n\nTime: Virtual e-posters are available for viewing for the duration of the congress (October 3-7, 2021) and will be available on demand for 3 months post conference.\n\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 patients with SMA. The U.S. Food and Drug Administration (FDA) has granted Fast Track (FTD), Orphan Drug (ODD) and Rare Pediatric Disease (RPD) designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. The efficacy and safety of apitegromab have not been established and apitegromab has not been approved for any use by the FDA or any other regulatory agency.\n\nAbout SMA\n\nSpinal muscular atrophy (SMA) is a rare, and often fatal, genetic disorder that typically manifests in young children. An estimated 30,000 to 3...