Edgewise Therapeutics Reports Positive Results on Sevasemten Program for Becker and Duchenne Muscular Dystrophies

Rhea-AI Summary

Edgewise Therapeutics (NASDAQ:EWTX) reported positive results for sevasemten in treating Becker and Duchenne muscular dystrophies. The MESA open-label trial showed sustained disease stabilization for up to three years in Becker patients, with a 0.8-point improvement in NSAA scores over 18 months.

Following a successful FDA Type C meeting, the company received clarity on the registration path for sevasemten as the first-ever Becker therapy. The GRAND CANYON pivotal trial is progressing toward topline data in Q4 2026. Additionally, Phase 2 trials (LYNX and FOX) in Duchenne patients identified 10 mg as the optimal dose for Phase 3, with plans to initiate the pivotal study in 2026.

Positive

• First-ever potential therapy for Becker muscular dystrophy
• 99% enrollment rate (n=85) in MESA trial
• Sustained disease stabilization with 0.8-point NSAA improvement over 18 months
• Favorable safety profile after three years of treatment
• Clear FDA regulatory pathway established for registration
• Successful identification of 10 mg optimal dose for Duchenne Phase 3

Negative

• FDA deemed CANYON data alone insufficient for accelerated approval
• GRAND CANYON pivotal trial results not expected until Q4 2026
• Phase 3 trial for Duchenne yet to begin, pending FDA discussions

Insights

Neuromuscular Disease Specialist

Sevasemten shows sustained disease stabilization in Becker MD and promising results in Duchenne MD with clear regulatory pathway ahead.

The clinical data from Edgewise's sevasemten program represents a potentially significant advancement for muscular dystrophy patients. The MESA open-label extension trial demonstrated remarkable disease stabilization for up to three years in Becker muscular dystrophy (BMD) patients - a striking outcome for a progressive condition. CANYON participants showed a 0.8 point improvement in North Star Ambulatory Assessment (NSAA) scores over 18 months, contrasting sharply with the functional decline typically observed in natural history studies.

The sustained efficacy across multiple years is particularly noteworthy, as muscular dystrophies typically show relentless progression. For BMD patients, who currently have no FDA-approved therapies, these results could represent a paradigm shift in disease management if confirmed in the pivotal GRAND CANYON trial.

In the Duchenne muscular dystrophy (DMD) program, the identification of 10 mg as the optimal dose based on consistent observations across multiple functional measures (SV95C, NSAA, and 4-stair climb) provides a clear path forward. The inclusion of post-gene therapy patients in the FOX study addresses a critical emerging need, as many gene therapy recipients continue to experience functional decline years after treatment.

The FDA's confirmation that NSAA is a clinically meaningful endpoint validates the company's approach, though the requirement for the full GRAND CANYON dataset rather than accelerated approval means patients will need to wait until after the Q4 2026 data readout for potential access to this treatment.

Biotechnology Investment Analyst

Edgewise's positive clinical data and clear FDA pathway position sevasemten as potential first-ever therapy for Becker muscular dystrophy.

Edgewise Therapeutics has achieved multiple significant milestones with its sevasemten program, solidifying its position as a frontrunner in muscular dystrophy treatment development. The sustained disease stabilization observed in the MESA extension study over three years represents compelling evidence of durable efficacy - a critical factor for chronic therapies targeting progressive diseases.

The 99% enrollment rate of eligible participants (n=85) in the MESA extension study signals exceptional patient and physician engagement, which bodes well for future commercial adoption. This strong patient retention also strengthens the reliability of the long-term efficacy and safety observations.

The recent FDA Type C meeting provides regulatory clarity, with the agency supporting NSAA as a clinically meaningful endpoint and endorsing the ongoing GRAND CANYON pivotal trial as potentially sufficient for registration. While accelerated approval based solely on CANYON data was ruled out, the FDA's willingness to review MESA data and natural history modeling ahead of GRAND CANYON completion suggests regulatory flexibility.

The company's dual-indication strategy targeting both Becker and Duchenne muscular dystrophies maximizes sevasemten's commercial potential. The exploration of efficacy in post-gene therapy DMD patients is particularly strategic, as this represents an emerging patient population with significant unmet needs. With GRAND CANYON results expected in Q4 2026 and Phase 3 DMD studies starting in 2026, Edgewise has established a clear development timeline toward potential commercialization of the first-ever therapy for Becker muscular dystrophy.

– New open label data in Becker demonstrated sustained disease stabilization up to three years, reinforcing prior clinical findings –

– Ongoing pivotal trial and FDA Type C meeting provide clear path to potential sevasemten registration as the first ever therapy for Becker –

– Encouraging Phase 2 observations in Duchenne define the dose and inform design for
Phase 3 –

– Edgewise leadership to discuss these updates on Thursday, June 26 at 8:30 a.m. Eastern Time at a virtual investor event –

BOULDER, Colo., June 26, 2025 /PRNewswire/ -- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today unveiled positive results in its sevasemten program for Becker and Duchenne muscular dystrophies.

The Company announced positive data from MESA, an open label extension trial that is providing 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.

The MESA data demonstrated sustained disease stabilization, reinforcing prior ARCH and CANYON findings. Importantly, CANYON participants who rolled over to MESA showed increased North Star Ambulatory Assessment (NSAA) scores over 18 months (0.8 point improvement from baseline), with a trend toward improvement in placebo participants switching to sevasemten (0.2 point improvement since initiation of 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. 

"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," said Joanne Donovan, Ph.D., M.D., Chief Medical Officer. "We are well positioned to deliver the first ever therapy for individuals with Becker muscular dystrophy."

Edgewise recently completed a successful Type C meeting with the U.S. Food and Drug Administration (FDA), which provided a clear path to registration of sevasemten as the first ever therapy for Becker. While the FDA deemed the CANYON data alone insufficient for an accelerated approval, the Agency reiterated that NSAA is a clinically meaningful endpoint for traditional approval. The FDA encouraged Edgewise to continue to share MESA data and natural history prospective modeling ahead of GRAND CANYON completion. Further, the FDA emphasized their support for GRAND CANYON, the ongoing global pivotal placebo-controlled cohort, and its potential as a single adequate well-controlled study to support registration. GRAND CANYON is highly powered 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. 

The Company also announced encouraging topline data from its Phase 2 Duchenne trials, LYNX and FOX. The goals were to explore a range of doses to assess safety and identify a potentially beneficial dose for Phase 3. The trial's dose escalation paradigm provided a three-month placebo-controlled period to evaluate biomarkers for dose selection, followed by an open label period. Across both studies, at target doses, sevasemten was well-tolerated.

LYNX is an ongoing multi-center, placebo-controlled, dose-finding Phase 2 trial 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. Consistent observations across functional measures, including Stride Velocity 95th Centile (SV95C), NSAA and 4 stair-climb, identified a dose of 10 mg to evaluate in Phase 3. 

Similar to the LYNX design, FOX is an ongoing multi-center, placebo-controlled Phase 2 trial 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 indicate that sevasemten 10 mg has the potential to reduce the rate of functional decline.

"We have executed an important Phase 2 exploration of sevasemten in Duchenne," said Kevin Koch, Ph.D., President and Chief Executive Officer. "We are encouraged by the functional response seen at the 10 mg dose and look forward to our discussions with the Agency later this year."

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 the 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. 

Virtual Investor Event

Members of the Edgewise management team will hold a live webcast on Thursday, June 26, at 8:30 a.m. ET to discuss the sevasemten program. An accompanying slide presentation will also be available. To register for the live webcast and replay, please visit the Edgewise events page.

About Sevasemten

Sevasemten 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. Sevasemten presents a novel mechanism of action designed to selectively limit the exaggerated muscle damage caused by the absence or loss of functional dystrophin. Its unique mechanism of action provides the potential to establish sevasemten as a foundational therapy in dystrophinopathies, either as a single agent therapy or in combination with available therapies and those in development. Sevasemten has achieved notable regulatory milestones by securing FDA Orphan Drug Designation for the treatment of Becker and Duchenne, Rare Pediatric 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.

About Becker

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.

About Duchenne

Duchenne, 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 to treat the disease, there remains a high unmet need for additional therapies.

About Edgewise Therapeutics

Edgewise Therapeutics is a leading muscle disease biopharmaceutical company developing novel therapeutics for muscular dystrophies and serious cardiac conditions. The Company's deep expertise in muscle physiology is driving a new generation of novel therapeutics. Sevasemten is an orally administered skeletal myosin inhibitor in late-stage clinical trials in Becker and Duchenne muscular dystrophies. EDG-7500 is a novel cardiac sarcomere modulator for the treatment of hypertrophic cardiomyopathy and other diseases of diastolic dysfunction, currently in Phase 2 clinical development. The entire team at Edgewise is dedicated to our mission: changing the lives of patients and families affected by serious muscle diseases. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn, X, Facebook and Instagram.

References

[1] Bello L, et al. Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies. Sci Rep. 2016;6:32439. doi:10.1038/srep32439

[2] van de Velde NM, et al. Selection approach to identify the optimal biomarker using quantitative muscle MRI and functional assessments in Becker muscular dystrophy. Neurology. 2021;97(5):e513-e522. doi: 10.1212/WNL.0000000000012233.

[3] De Wel B, et al. Lessons for future clinical trials in adults with Becker muscular dystrophy: disease progression detected by muscle magnetic resonance imaging, clinical and patient-reported outcome measures. Eur J Neurol. 2024:e16282. doi:10.1111/ene.16282. Online ahead of print.

[4] Muntoni F, et. al., Neuromuscul Disord., 2022 Apr;32(4):271-283. doi: 10.1016/j.nmd.2022.02.009

Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Such forward-looking statements include, among other things, statements regarding the potential of, and expectations regarding sevasemten, statements regarding the potential market opportunity for sevasemten, statements regarding Edgewise's expectations and milestones relating to its clinical trials and clinical development of sevasemten, including the timing of topline data for the GRAND CANYON trial and the timing of the initiation of a pivotal study of sevasemten, statements regarding the potential outcome of the GRAND CANYON trial, statements regarding the timing and outcome of Edgewise's discussions with the FDA, statements regarding the clear path to potential sevasemten registration as the first ever therapy for Becker, and statements by Edgewise's President and Chief Executive Officer and Chief Medical Officer. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. The forward-looking statements contained herein are based upon Edgewise's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those projected in any forward-looking statements due to numerous risks and uncertainties, including but not limited to: risks associated with Edgewise's limited operating history, its products being early in development and not having products approved for commercial sale; risks associated with Edgewise not having generated any revenue to date; Edgewise's ability to achieve objectives relating to the discovery, development and commercialization of its product candidates, if approved; Edgewise's need for substantial additional capital to finance its operations; Edgewise's substantial dependence on the success of sevasemten; Edgewise's ability to develop and commercialize sevasemten and discover, develop and commercialize product candidates in future programs; risks related to Edgewise's clinical trials of its product candidates not demonstrating safety and efficacy; risks related to Edgewise's product candidates causing serious adverse events, toxicities or other undesirable side effects; the outcome of preclinical testing and early clinical trials not being predictive of the success of later clinical trials and the risks related to the results of Edgewise's clinical trials not satisfying the requirements of regulatory authorities; delays or difficulties in the enrollment and/or maintenance of patients in clinical trials; risks related to failure to capitalize on other indications or product candidates; risks related to competition; risks relating to interim, topline and preliminary data from Edgewise's clinical trials changing as more patient data becomes available; risks related to the regulatory approval processes of domestic and foreign authorities being lengthy, time consuming and inherently unpredictable; risks related to production of drugs by Edgewise's third-party manufacturers; risks related to changes in methods of product candidate manufacturing or formulation; risks related to not achieving adequate market acceptance; risks related to the patient population for our product candidates having a small patient population; risks related to regulatory authorities not accepting data from trials conducted in locations outside of their jurisdiction; risks relating to Edgewise's ability to attract and retain highly skilled executive officers and employees; Edgewise's ability to obtain and maintain intellectual property protection for its product candidates; Edgewise's reliance on third parties; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled "Risk Factors" in documents that Edgewise files from time to time with the U.S. Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Edgewise assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law.

This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release.

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SOURCE Edgewise Therapeutics

FAQ

What are the latest clinical results for Edgewise Therapeutics' (EWTX) sevasemten in Becker muscular dystrophy?

The MESA trial showed sustained disease stabilization for up to three years, with a 0.8-point improvement in NSAA scores over 18 months and a favorable safety profile.

When will Edgewise Therapeutics (EWTX) complete the GRAND CANYON pivotal trial for sevasemten?

The GRAND CANYON pivotal trial is expected to deliver topline data in the fourth quarter of 2026.

What dose of sevasemten did Edgewise Therapeutics identify for Duchenne muscular dystrophy Phase 3?

The Phase 2 LYNX and FOX trials identified 10 mg as the optimal dose for the Phase 3 trial in Duchenne patients.

What is the FDA's stance on Edgewise Therapeutics' sevasemten for Becker muscular dystrophy?

The FDA provided a clear path to registration through the GRAND CANYON trial, though deemed CANYON data alone insufficient for accelerated approval. They confirmed NSAA as a clinically meaningful endpoint for traditional approval.

What is the enrollment rate in Edgewise Therapeutics' MESA trial for sevasemten?

The MESA trial has achieved 99% enrollment of eligible participants (n=85) from previous trials including ARCH, CANYON, GRAND CANYON, and DUNE.