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Investing.com -- Edgewise Therapeutics Inc (NASDAQ:EWTX) stock tumbled 7.2% after the company revealed that the U.S. Food and Drug Administration (FDA) deemed its CANYON trial data insufficient for accelerated approval of sevasemten for Becker muscular dystrophy.
The muscle disease biopharmaceutical company reported that despite positive results from its MESA open-label extension trial showing sustained disease stabilization in Becker muscular dystrophy patients, the FDA indicated a traditional approval pathway would be necessary. The agency encouraged Edgewise to continue sharing MESA data and natural history modeling while awaiting results from the ongoing GRAND CANYON pivotal trial.
GRAND CANYON, which the FDA emphasized support for, is designed as a placebo-controlled study that could potentially serve as a single adequate well-controlled study to support registration. Topline data from this trial is expected in the fourth quarter of 2026.
The company also announced encouraging topline data from its Phase 2 Duchenne muscular dystrophy trials, LYNX and FOX. These dose-finding studies identified 10 mg as the optimal dose to evaluate in Phase 3 trials. Edgewise plans to meet with the FDA in the fourth quarter of 2025 to discuss Phase 3 design for Duchenne muscular dystrophy, with plans to initiate the pivotal study in 2026.
Edgewise’s Chief Medical (TASE:BLWV) Officer, Joanne Donovan, expressed optimism despite the regulatory setback, stating, "We are thrilled with the tremendous excitement from physicians and the patient community around the data on sevasemten to date and their unwavering commitment to our ongoing pivotal program."
The company continues to position sevasemten as potentially the first approved therapy for individuals with Becker muscular dystrophy, pending successful completion of its ongoing clinical program.
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