World News – CA – Scholar Rock Announces Positive Proof of Concept Data from Phase 2 Spinal Muscular Atrophy Trial


Shares of Scholar Rock Holding Corporation (NASDAQ: SRRK) jumped more than 68% after the clinical-stage biopharmaceutical company reported positive six-month results from interim analysis of the TOPAZ phase 2 clinical trial Treatment with SRK-015 resulted in improvements in Hammersmith scale scores (primary efficacy endpoint) in all three cohorts of patients with spinal muscular atrophy type 2 and type 3 (SMA)

The dose response in the primary efficacy endpoint was observed at all time points evaluated in the randomized, double-blind part of the trial (cohort 3) The high dose arm of cohort 3 a achieved a 5 Average 6 point improvement from baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) compared to the low dose arm, which reached a 2 Average 4 point improvement at the time of interim analysis of six months No safety signal was identified from the interim analysis

« These intermediate results are important because they demonstrate the potential of this muscle-driven approach to improve motor function in individuals with type 2 and type 3 SMA, » said Thomas Crawford, Mré, Professor of Neurology at Johns Hopkins University School of Medicine and Principal Investigator of the TOPAZ trial “Over the past few years, we have celebrated the remarkable success of treating AMS with upregulating approaches to NMS that are stabilize against neurodegeneration These results highlight the potential of an entirely new approach to SMA therapy, used in conjunction with NMS-enhancing therapies, to address the persistent and significant unmet needs of people debilitated by SMA. »

« This is an exciting and important step towards establishing SRK-015 as the first potential muscle-directed therapy for patients with ADS, while providing important validation of our scientific approach to targeting latent forms of growth factors, « said Yung Chyung, Mré, Chief Medical Officer of Scholar Rock » These interim data support the continuation of the TOPAZ trial and we look forward to engaging with regulatory authorities regarding our plans to  » registration tests »

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Spinal Muscular Atrophy, Scholar Rock Holding, NASDAQ: SRRK, Interim Analysis, Stock

News from around the world – CA – Scholar Rock announces positive proof of concept data from phase spinal muscle atrophy 2 Trial


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