Edgewise Therapeutics' Lead Candidate Reduces Biomarker Levels In Mid-Stage Study In Patients With Muscle Weakness Disease

On Monday, Edgewise Therapeutics, Inc., EWTX revealed topline results from the Phase 2 CANYON trial of sevasemten in individuals with Becker muscular dystrophy (BMD), is a genetic disorder that causes progressive muscle weakness.

Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to protect muscle against contraction-induced damage in muscular dystrophies.

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The company adds that CANYON is the largest interventional trial to date in BMD and the first to achieve its primary endpoint. The trial was conducted among 40 adults and 29 adolescents.

The trial met its primary endpoint of change from baseline in creatine kinase (CK).

The results demonstrated a significant change from baseline in CK in the sevasemten-treated group (difference vs. placebo, 28% average decrease over months 6 through 12; p=0.02).

North Star Ambulatory Assessment (NSAA, a change in motor function), the key secondary endpoint of function, showed a trend towards improvement over time in the sevasemten-treated group.

Plasma fast skeletal muscle troponin I (TNNI2), a target-specific biomarker of fast skeletal muscle damage, showed 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 toward improvement compared to the placebo.

The treatment population had more advanced disease than placebo.

Sevasemten was well-tolerated, and no new safety concerns were observed.

The company is on track to complete recruitment in the GRAND CANYON cohort by the first quarter of 2025.

Price Action: EWTX stock is up 8.62% at $30 at last check Monday.

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