Edgewise Therapeutics, Inc., has announced positive topline results from the double-blind, randomized, placebo-controlled Phase 2 CANYON trial of sevasemten in individuals with Becker muscular dystrophy. Sevasemten is an orally administered small molecule inhibitor designed to protect muscle against contraction-induced damage in muscular dystrophies.

According to Edgewise, the trial met its primary endpoint of change from baseline in creatine kinase (CK), a biomarker associated with skeletal muscle damage. The results demonstrated a significant reduction from baseline in circulating levels of CK in the sevasemten-treated group at 12 months. CANYON is the largest interventional trial to date in Becker and the first to achieve its primary endpoint.

Additionally, sevasemten-treated patients showed stabilization of North Star Ambulatory Assessment (NSAA), compared to placebo which declined similarly to patients observed in natural history studies. Edgewise reported that plasma fast skeletal muscle troponin I (TNNI2), a target-specific biomarker of fast skeletal muscle damage, showed a decline of 77%, a significant reduction compared to placebo. Additional functional measures, including the 10-meter walk/run, 4-stair climb and 100-meter timed test, showed trends towards improvement compared to placebo. Results show that sevasemten was well-tolerated, and no new safety concerns were observed.

Based on these positive Phase 2 results, Edgewise has indicated that it plans to engage the U.S. Food and Drug Administration and European Medicines Agency about marketing authorization filing strategies for sevasemten in Becker. The company intends to submit the complete results of the CANYON study for publication at a future medical congress. Edgewise reports that it is on track to complete recruitment in the GRAND CANYON global pivotal cohort by the first quarter of 2025. 

Read Edgewise’s press release here.

Watch the Webinar

Edgewise joined PPMD for a breaking news webinar, “Sevasemten: CANYON Trial Topline Results,” on Wednesday, December 18th, 2024. Edgewise provided a community update on the recently released topline results from its double-blind, randomized, placebo-controlled design to investigate the effect of sevasemten on individuals with Becker.