Shares of Edgewise Therapeutics, Inc. (NASDAQ:EWTX) experienced a notable 21% increase in premarket trading today following the announcement of successful results from its Phase 2 CANYON trial. The trial, which tested the company's drug sevasemten on individuals with Becker muscular dystrophy, achieved its primary endpoint, leading to a significant rise in the company's stock value.
The primary endpoint of the trial was the change from baseline in levels of creatine kinase, a biomarker associated with skeletal muscle damage. The results showed that patients treated with sevasemten demonstrated a stabilization of the North Star Ambulatory Assessment (NSAA), a measure of motor function, with a trend toward improvement at 12 months in comparison to the placebo group.
Sevasemten was reported to be well-tolerated, with no new safety concerns identified in either adult or adolescent participants. The company has expressed plans to discuss marketing authorization filing strategies with both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for sevasemten in the treatment of Becker muscular dystrophy.
Truist analyst Srikripa Devarakonda, Ph.D. reaffirmed a Buy rating on Edgewise Therapeutics, citing the positive Phase 2 data as a significant development in a field with no approved drugs and a high unmet need. Devarakonda highlighted the statistical significance of the study's primary endpoint and noted the numerical improvement in the NSAA score, though not statistically significant. The analyst also pointed out the substantial reduction in creatine kinase and TNNI2, a biomarker of fast skeletal muscle damage, over a 6 to 12-month period.
The CANYON trial stands as the largest interventional study in Becker muscular dystrophy to date and the first to meet its primary endpoint. Additional functional measures in the trial indicated trends toward improvement when compared to placebo. Notably, the treatment group had more advanced disease than the placebo group.
Craig M. McDonald, M.D., a Principal Investigator in the CANYON trial, emphasized the rapid progression of Becker muscular dystrophy and the importance of the study's findings, which suggest potential for functional stabilization with sevasemten. Joanne Donovan, Ph.D., M.D., Chief Medical (TASE:PMCN) Officer at Edgewise, echoed this sentiment, noting the promising signals for functional preservation in patients.
The company is on track to complete recruitment for the GRAND CANYON cohort by the first quarter of 2025 and intends to submit the complete CANYON study results for publication at a future medical congress.
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