Scholar Rock Announces Positive Proof-of-Concept Data from TOPAZ Phase 2 Trial Interim Analysis of SRK-015 in Patients with Type 2 and Type 3 SMA

Scholar Rock announced positive six-month interim analysis results from the TOPAZ Phase 2 clinical trial yesterday. SRK-015 is a selective inhibitor of myostatin that has been shown to increase muscle mass and strength in animal models. 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). No safety signals were identified from the interim analysis.

For more information, please see the press release from Scholar Rock.