As of the most recent check, shares of Edgewise Therapeutics, Inc. (NASDAQ: EWTX) are up 10.72% in early session, trading at $30.58. Following the publication of positive results from the company’s clinical trial, EWTX shares increased.
Landmark Results from Phase 2 CANYON Trial
Edgewise Therapeutics (EWTX) unveiled positive topline results from its Phase 2 CANYON trial evaluating sevasemten in individuals with Becker muscular dystrophy (BMD). The goal of Sevasemten, a novel oral rapid skeletal myosin inhibitor, is to shield muscles from the harm that muscular dystrophies inflict due to contraction.
Creatine kinase (CK) levels showed notable improvements from baseline, indicating that the experiment met its primary aim. The CANYON trial is noteworthy for being the first to effectively accomplish its primary aim and the biggest interventional study in BMD.
Secondary endpoints also delivered promising insights. The North Star Ambulatory Assessment (NSAA) revealed a trend towards functional improvement in the sevasemten-treated group. Plasma fast skeletal muscle troponin I (TNNI2), a biomarker of acute skeletal muscle damage, showed significant reductions in comparison to the placebo group.
Improvement was shown in functional assessments such the 10-meter walk/run, 4-stair climb, and 100-meter timed test. Crucially, compared to those in the placebo group, individuals undergoing therapy had more advanced illness.
High Tolerance and Continued Engagement
Sevasemten was well-tolerated across both adult and adolescent patient groups, with no new safety concerns reported. An impressive 99% of eligible participants from CANYON and other sevasemten trials have opted to enroll in MESA, an ongoing open-label extension study. This continued participation underscores confidence in the treatment’s safety and efficacy.
Hope for a Transformative Therapy
Becker muscular dystrophy remains an unmet medical need, with no approved therapies currently available. The groundbreaking results from the GRAND CANYON pivotal cohort signal the potential for sevasemten to stabilize functional decline in BMD patients.
Edgewise Therapeutics plans to complete recruitment for the GRAND CANYON cohort by Q1 2025 and engage regulatory authorities for marketing authorization strategies in the U.S. and Europe. Additionally, EWTX intends to present the full CANYON study findings at an upcoming medical congress.
Edgewise Therapeutics’ novel approach to treating muscular dystrophies highlights its potential to revolutionize care for Becker muscular dystrophy patients, offering hope for the first approved therapy in this challenging field.
