Scholar Rock Announces Positive Proof-of-Concept Data from Spinal Muscular Atrophy Phase 2 Trial | Financial Buzz

Scholar Rock Announces Positive Proof-of-Concept Data from Spinal Muscular Atrophy Phase 2 Trial

Scholar Rock Holding Corporation (NASDAQ: SRRK) stock jumped more than 68 percent after the clinical-stage biopharmaceutical company announced positive six-month interim analysis results from the TOPAZ Phase 2 clinical trial. Treatment with SRK-015 led to improvements in Hammersmith scale scores (primary efficacy endpoint) in all three cohorts of patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA).

Dose response in the primary efficacy endpoint was observed across all evaluated timepoints in the double-blind, randomized portion of the trial (Cohort 3). The high-dose arm of Cohort 3 attained a 5.6 point mean improvement from baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) as compared to the low dose arm, which attained a 2.4 point mean improvement at the six-month interim analysis timepoint. No safety signals were identified from the interim analysis.

“These interim results are important because they demonstrate the potential of this muscle-directed approach to improve motor function of individuals with Type 2 and Type 3 SMA,” said Thomas Crawford, M.D., Professor of Neurology at the Johns Hopkins University School of Medicine and Lead Investigator of the TOPAZ trial. “In the last few years, we’ve celebrated the remarkable success in treating SMA with SMN-upregulating approaches that stabilize against neurodegeneration. These findings highlight the potential for a whole new approach to SMA therapy, used in conjunction with the SMN-enhancing therapies, to address the persistent and significant unmet needs of individuals weakened by SMA.”

“This is an exciting and important step towards establishing SRK-015 as the potential first muscle-directed therapy for patients with SMA, while also providing important validation of our scientific approach of targeting the latent forms of growth factors,” said Yung Chyung, M.D., 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 registrational trial plans.”

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