Scholar Rock stuns with positive results for SRK-015 in SMA patients

US clinical-stage biotech Scholar Rock (Nasdaq: SRRK) skyrocketed 119% by close of trading Tuesday and was up a further 13.9% to $34.16 this morning, after it released stunning trial results on its lead investigational drug SRK-015 and announced a public offering of shares.

Scholar Rock announced positive six-month interim analysis results from the TOPAZ Phase II 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 Dr Thomas Crawford, 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,” he opined.