Spinal Muscular Atrophy
The TOPAZ Phase 2 proof of concept trial evaluated the safety and efficacy of apitegromab (SRK-015) in patients with Type 2 and Type 3 SMA.
The trial enrolled 58 patients between the ages of 2 and 21 in the U.S. and Europe across three distinct cohorts. All patients received apitegromab once every four weeks either as a monotherapy or in conjunction with an approved SMN upregulator treatment over a 12-month treatment period. The primary efficacy endpoints measure motor function through clinically meaningful outcome measures validated in SMA, such as the Hammersmith Functional Motor Scale Expanded (HFMSE) in non-ambulatory SMA and the Revised Hammersmith Scale (RHS) in ambulatory SMA.
A pre-planned interim analysis was conducted following a six-month treatment period, and positive proof-of-concept data were announced in October 2020. Top-line results for the full 12-month treatment period were announced in April 2021.
The trial is anticipated to advance to Part B (dose expansion) mid-year 2021 and initial clinical response and safety data from Part A are anticipated by the end of 2021.
Patients seeking more information on potential clinical trials should contact their physician.