Edgewise Therapeutics, announced positive topline results from the Phase 2 CANYON trial of sevasemten in individuals with Becker muscular dystrophy. Sevasemten, an orally administered first-in-class fast skeletal myosin inhibitor, aims to protect muscle against contraction-induced damage.
The CANYON trial, the largest interventional trial to date in Becker muscular dystrophy, met its primary endpoint of reducing creatine kinase (CK) levels, a biomarker of muscle damage. The trial also showed a trend toward improvement in North Star Ambulatory Assessment (NSAA) functional measures such as, 10-metre walk/run, 4-stair climb and 100-metre timed test over time. Furthermore, a significant reduction was observed in plasma fast skeletal muscle troponin I (TNNI2), a target-specific biomarker of fast skeletal muscle damage, compared to placebo.
Sevasemten was well-tolerated, with no new safety concerns observed. The company is on track to complete recruitment in the GRAND CANYON pivotal cohort by the first quarter of 2025 and plans to engage with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) regarding marketing authorisation filing strategies for sevasemten in Becker muscular dystrophy.
The complete results of the CANYON study will be submitted for publication at a future medical congress.
Regulatory Progress
Sevasemten has achieved notable regulatory milestones by securing FDA Orphan Drug Designation for the treatment of Becker and Duchenne, Rare Paediatric Disease Designation (RPDD) for the treatment of Duchenne, and Fast Track designations for the treatment of Becker and Duchenne. Further, sevasemten secured the EMA Orphan Drug Designations for the treatment of Becker and Duchenne.
CANYON Trial Summary
Trial Design
- Phase 2, double-blind, randomized, placebo-controlled study
- Primary endpoint: Change from baseline in CK levels
- Key secondary endpoint: Change from baseline in NSAA total score
- Participants: 40 adults and 29 adolescents with Becker muscular dystrophy
- Treatment period: 12 months
Key Findings
- Primary endpoint met: Significant reduction in CK levels in the sevasemten-treated group compared to placebo.
- Key secondary endpoint: Trend towards improvement in NSAA total score, indicating potential for improved functional stabilisation.
- Other secondary endpoints:
- Significant reduction in plasma TNNI2 levels (77% compared to placebo)
- Trends towards improvement in 10-meter walk/run, 4-stair climb, and 100-meter timed tests.
- Safety and tolerability: Sevasemten was well-tolerated, with no new safety concerns identified.
About Sevasemten (EDG-5506)
Sevasemten is a novel oral medication designed to protect muscle tissue from damage in patients with Becker and Duchenne muscular dystrophies. This first-in-class fast skeletal myosin inhibitor works by selectively limiting the excessive muscle damage caused by the absence or loss of dystrophin.
Clinical Development:
Sevasemten is currently being evaluated in several clinical trials:
- CANYON and GRAND CANYON: Phase 2 studies investigating the efficacy and safety of sevasemten in adults and adolescents with Becker muscular dystrophy.
- LYNX and FOX: Phase 2 studies exploring the potential of sevasemten in children and adolescents with Duchenne muscular dystrophy.
If you have any questions and queries, please reach out to us at info@actionduchenne.org
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