Edgewise Therapeutics Reports Second Quarter 2025 Financial Results and Recent Business Highlights
– Announced positive top-line data from the open-label extension MESA trial of sevasemten in participants with Becker muscular dystrophy who previously completed ARCH and CANYON studies,
– Announced encouraging top-line data from the Phase 2 LYNX and FOX trials of sevasemten in Duchenne muscular dystrophy,
– Advanced the Phase 2 CIRRUS-HCM trial of EDG-7500 in Hypertrophic Cardiomyopathy (HCM),
BOULDER, Colo., Aug. 7, 2025 /PRNewswire/ -- Edgewise Therapeutics, Inc., (NASDAQ:EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the second quarter of 2025 and recent business highlights.
"In the first half of 2025, we reached key milestones that bring us closer to delivering on our mission," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. "With strong funding in place, we're advancing our skeletal and cardiac muscle programs and building the commercial infrastructure with precision and purpose to support a potential U.S. launch of sevasemten in Becker. We're also actively exploring Phase 3 trial designs in HCM and Duchenne, while continuing to advance our pipeline through bold, innovative R&D."
Recent Highlights
Muscular Dystrophy ProgramSevasemten and BeckerSevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to protect against contraction-induced muscle damage in muscular dystrophies including Becker and Duchenne. Becker is a rare, genetic, life-shortening, debilitating and degenerative neuromuscular disorder. Genetic mutations in the dystrophin gene result in contraction-induced muscle damage, which is the primary driver of irreversible muscle loss and impaired motor function. The disease predominantly affects males, with functional decline beginning at any age. Once that muscle loss occurs, the decline in function is irreversible and continues throughout the individual's life. Currently, there are no approved therapies on the market to treat Becker.
MESA open-label extension trial in adults with Becker: In June 2025, the Company announced positive data from MESA, an open-label extension trial that provides continued access to sevasemten to participants with Becker who were previously enrolled in ARCH, or completed CANYON, GRAND CANYON, or DUNE. As of the March 2025 data cut, 99% of eligible participants (n=85) are enrolled in MESA, which was designed to collect long-term safety and efficacy data.
The MESA data demonstrated sustained disease stabilization in North Star Ambulatory Assessment (NSAA), reinforcing prior ARCH and CANYON findings. Importantly, CANYON participants who rolled over to MESA showed stable NSAA scores over 18 months with a trend toward improvement in placebo participants switching to sevasemten. During the 18 months of sevasemten treatment, participants' NSAA scores continued to diverge relative to the expected functional declines seen in multiple Becker natural history studies. Further, NSAA scores for ARCH participants who rolled over to MESA remained stable after three years of treatment. Sevasemten continues to demonstrate a favorable safety profile after up to three years of treatment.
GRAND CANYON, a global pivotal placebo-controlled cohort in Becker: In February 2025, the Company completed enrollment in GRAND CANYON, an expansion of the CANYON placebo-controlled trial. The 18-month GRAND CANYON cohort is active in 12 countries, GRAND CANYON enrolled 175 adults, reflective of the Becker community's enthusiasm to have access to a therapy with the potential to stabilize their debilitating decline in function and ability to perform everyday activities.
GRAND CANYON is highly powered to be able to show a statistically significant difference in NSAA versus placebo over 18 months and is on track for topline data in the fourth quarter of 2026. In the second quarter of 2025, the Company completed a successful Type C meeting with the FDA, which provided a clear path to registration of sevasemten as the first ever therapy for Becker. To learn more, go to clinicaltrials.gov (NCT05291091).
DuchenneDuchenne, a severe degenerative muscle disorder, is the most common type of muscular dystrophy with a median life expectancy of around 30 years. Genetic mutations in the dystrophin gene result in contraction-induced muscle damage, which is the primary driver of irreversible muscle loss and impaired motor function. While there are approved therapies on the market aimed at treating the disease, there remains a high unmet need for additional therapies.
LYNX Phase 2 placebo-controlled, dose-finding trial in boys with Duchenne: LYNX is designed to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in four- to nine-year-old participants with Duchenne treated with sevasemten in a three-month placebo-controlled dose ranging study, followed by an open-label extension period. In June 2025, the Company announced encouraging observations across functional measures, including Stride Velocity 95th Centile (SV95C), NSAA and 4 stair-climb, while identifying a dose of 10 mg to evaluate in Phase 3.
FOX Phase 2 placebo-controlled trial in boys with Duchenne: FOX is designed to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in six- to 14-year-old participants with Duchenne who have been previously treated with gene therapy. FOX participants are on average over 10 years old and four years out from receiving gene therapy. Despite the lack of extensive natural history in Duchenne gene therapy treated boys, initial results from the FOX study support that sevasemten 10 mg has the potential to reduce the rate of functional decline.
The Company plans to meet with the FDA in the fourth quarter of 2025 to discuss a Phase 3 design including input on the patient population and endpoints, with plans to initiate a pivotal study in 2026. In addition, the Company plans to continue to collect longer-term open label extension data, which will provide further access to the drug to trial participants.
For more information, go to clinicaltrials.gov to learn more about LYNX (NCT05540860) and FOX (NCT06100887).
Cardiovascular and Cardiometabolic Programs
EDG-7500 and HCMEDG-7500 is a novel oral, selective, cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with HCM and other diseases of diastolic dysfunction. HCM is the most common form of genetic heart disease, affecting approximately one in 500 people, and is associated with reduced quality of life and an elevated risk ...
