SMA Newsroom

Jun 22, 2022

Scholar Rock: Positive Phase 2 Topaz Trial Extension.

On June 17th, Scholar Rock announced new data from the Phase 2 TOPAZ trial extension period evaluating outcomes after 24-months of treatment.

Apitegromab, or SRK-015, is a muscle-directed therapy, developed by Scholar Rock that aims to reverse or restrict the muscle atrophy and weakness experienced by people living with SMA. The aim of the TOPAZ clinial trial, a phase 2 study, is to evaluate the efficacy and safety of apitegromab in people with later-onset SMA. 

The 24-month results provide long-term data and evidence, underscoring the findings of the 12-month primary treatment period of the TOPAZ trial in which people receiving apitegromab experienced sizable motor function gains. This durability and continued increase in motor function support the transformative potential of apitegromab for people living with SMA.