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Scholar Rock Reports Preliminary Pharmacokinetic and Pharmacodynamic Data from TOPAZ Phase 2 Trial of SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy
Biomarker results confirm presence of latent myostatin in patients with SMA, further supporting relevance of drug target in diseaseSRK-015 demonstrated robust
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"Biomarker results confirm presence of latent myostatin in patients with SMA, further supporting relevance of drug target in diseaseSRK-015 demonstrated robust and dose-dependent target engagementData provide first evidence of successful pharmacologic engagement of a latent growth factor in a human disease setting CAMBRIDGE, Mass., Nov. 19, 2019 (GLOBE NEWSWIRE) -- Scholar 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 preliminary pharmacokinetic (PK) and pharmacodynamic (PD) results from the TOPAZ Phase 2 proof-of-concept trial of SRK-015 for the treatment of patients with spinal muscular atrophy (SMA). The planned preliminary PK/PD analysis, which includes data from 29 patients with SMA across all three cohorts, showed dose-proportional drug exposure and demonstrated target engagement, as evidenced by dose-dependent increases of up to 100-fold in the serum levels of latent myostatin following SRK-015 treatment (2 mg/kg and 20 mg/kg doses). SRK-015 is a highly selective inhibitor of the precursor, or latent form, of myostatin, and was specifically designed to avoid interactions with related targets such as activins, GDF-11, or BMPs, to potentially improve the therapeutic profile compared to traditional non-selective inhibitors.\n “We are pleased with the progress we have made to date towards our goal of developing SRK-015 as a muscle-directed therapy to address the functional deficits that remain a significant unmet need for patients with SMA despite advancements with SMN upregulators,” said Yung Chyung, M.D., Chief Medical Officer of Scholar Rock. “These preliminary PK/PD results positively address two important questions for the program by both confirming the presence of latent myostatin in patients with SMA and further corroborating the ability of SRK-015 to engage this drug target, including in pediatric patients with SMA.” “These results represent a key milestone for Scholar Rock by showing that we can indeed bring together cutting-edge monoclonal antibody technology with deep structural biology insights to target latent forms of growth factors in the context of human disease,” said Nagesh Mahanthappa, Ph.D., President and CEO of Scholar Rock. “We look forward to building upon these insights...