Scholar Rock Reports Interim Data from Phase 2 TOPAZ Trial of SRK-015 for Type 2 and 3 Spinal Muscular Atrophy

Scholar Rock Reports Interim Data from Phase 2 TOPAZ Trial of SRK-015 for Type 2 and 3 Spinal Muscular Atrophy

Scholar Rock reported positive data from a six-month interim analysis of the ongoing phase 2 TOPAZ clinical trial. The open label trial is evaluating SRK-015 across three cohorts of patients with type 2 and type 3 Spinal Muscular Atrophy (SMA). According to the data, administration of SRK-015 every four weeks (Q4W) led to improvements in Hammersmith scale scores in all three cohorts, meeting the primary efficacy endpoint. Top-line data from the 12-month treatment period are anticipated in the second quarter of 2021.

TOPAZ enrolled 58 patients with Type 2 and Type 3 SMA across 16 study sites in the U.S. and Europe. The patients ranged in age from 2 to 21 years old and were placed in one of three cohorts: Ambulatory Type 3 SMA (cohort 1), Type 2 SMA / Non-Ambulatory Type 3 SMA (cohort 2) or Type 2 SMA (cohort 3). All patient cohorts received 20 mg/kg of SRK-015 Q4W either as a monotherapy or in conjunction with an approved SMN upregulator therapy (nusinersen). In cohort 3, a low dose was added (2 mg /kg SRK-015 Q4W). The primary endpoint for Cohort 1 is change from baseline up to 12 months in the Revised Hammersmith Scale (RHS). The primary endpoint for Cohorts 2 and 3 is change ...

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