Edgewise Therapeutics, Inc., has announced positive results in the company’s sevasemten program for both Becker and Duchenne. These data include results from:

• MESA, an open label extension trial of participants with Becker who were previously enrolled in the ARCH trial, or completed the CANYON, GRAND CANYON, or DUNE trials 
• Phase 2 Duchenne trials, LYNX and FOX 

Sevasemten is a small molecule inhibitor taken by mouth designed to protect muscle against contraction-induced damage in muscular dystrophies.

According to Edgewise, key data takeaways in trials for individuals living with Becker include: 

• MESA data demonstrated sustained disease stabilization. 
• CANYON participants who rolled over to MESA showed increased North Star Ambulatory Assessment (NSAA) scores over 18 months and placebo participants who switched to sevasemten showed a trend toward improvement. 
• NSAA scores for ARCH participants who rolled over to MESA remained stable after three years of treatment.

Edgewise expects to share topline data from GRAND CANYON, the ongoing global pivotal placebo-controlled cohort for individuals with Becker muscular dystrophy, in the fourth quarter of 2026. 

The company’s Phase 2 Duchenne trials, LYNX (ages 4-9) and FOX (ages 6-14, post-gene therapy), are intended to assess safety and identify a potentially beneficial dose of sevasemten for a Phase 3 study. Across both studies, at target doses, results indicate that sevasemten was well-tolerated. Data from LYNX showing consistent observations across functional measures, including Stride Velocity 95th Centile (SV95C), NSAA and 4 stair-climb, supports a dose of 10mg to evaluate in Phase 3. 

According to Edgewise, the company plans to meet with the FDA in the fourth quarter of 2025 to discuss a Phase 3 design for its Duchenne studies, with plans to initiate the pivotal study in 2026. Edgewise also plans to continue to collect longer-term open label extension data.

Upcoming Webinar

Members of the Edgewise team will join PPMD for a webinar on Friday, June 27, 2025, at 1:00 PM ET to discuss these updates and answer questions from the community. Register here and submit questions in advance here.