FDA backs Edgewise’s sevasemten after strong Becker study data
Rhea-AI Filing Summary
Edgewise Therapeutics (Nasdaq:EWTX) filed an 8-K furnishing upbeat interim data on sevasemten for Becker and Duchenne muscular dystrophies and summarizing recent FDA guidance.
- MESA extension (n = 85): 0.8-point NSAA improvement over 18 months, 3-year disease stabilization and 99 % rollover from prior studies.
- Placebo switchers gained 0.2 NSAA points.
- Safety remains favorable after up to three years.
- FDA confirmed NSAA as a meaningful endpoint and indicated the ongoing GRAND CANYON study (topline Q4 2026) could be a single pivotal trial for Becker registration.
- Duchenne Phase 2 LYNX & FOX: Well-tolerated across doses; 10 mg selected for Phase 3 after functional and biomarker benefits.
Management plans a Phase 3 design meeting with FDA in Q4 2025 and expects pivotal Duchenne enrollment in 2026. No financial metrics were disclosed.
Positive
- MESA extension showed a sustained 0.8-point NSAA improvement over 18 months, indicating meaningful functional benefit in Becker patients.
- FDA Type C meeting provided a clear path to traditional approval with GRAND CANYON positioned as a single pivotal study.
- Sevasemten maintained a favorable safety profile for up to three years, supporting long-term tolerability.
- Phase 2 LYNX and FOX data identified a well-tolerated 10 mg dose, enabling Phase 3 Duchenne initiation in 2026.
Negative
- FDA judged CANYON data insufficient for accelerated approval, pushing earliest potential market entry to post-2026.
Insights
TL;DR: Solid efficacy signals and FDA alignment materially de-risk Becker program.
The 0.8-point NSAA gain and durable stabilization across three years corroborate earlier ARCH/CANYON findings and counter the natural 1–2-point annual decline cited in natural-history studies. High 99 % rollover strengthens dataset integrity. Crucially, FDA validation of NSAA and openness to a single adequate study mean registration hinges mainly on GRAND CANYON’s Q4-26 read-out, providing a clear catalyst path. While no revenue is imminent, the probability-adjusted NPV of sevasemten rises given lower regulatory uncertainty and the prospect of first-in-class status for Becker. Duchenne dose selection de-risks the next program leg and broadens addressable market. Overall, today’s disclosure meaningfully improves the long-term risk-reward profile.
TL;DR: FDA support narrows risk, but lack of accelerated path delays launch.
The Type C minutes clarify that CANYON data alone will not justify accelerated approval, removing a near-term approval scenario. However, FDA endorsement of NSAA as a traditional endpoint and encouragement to continue sharing MESA/natural-history modeling reduce qualitative regulatory risk. GRAND CANYON’s design appears aligned with agency expectations—large, placebo-controlled, 18-month primary analysis—suggesting minimal protocol amendments. Timelines shift commercial opportunity to 2027+ and increase burn-rate exposure, warranting scrutiny of funding runway. Overall impact is mixed: greater clarity but extended timeline.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
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| Item 7.01 | Regulation FD Disclosure. |
On June 26, 2025, Edgewise Therapeutics, Inc. (the “Company” or “Edgewise”) issued a press release announcing positive results in its sevasemten program for Becker and Duchenne muscular dystrophies. The press release is attached hereto as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.
The information furnished by this Current Report on Form 8-K under Item 7.01, including Exhibit 99.1 attached hereto shall be deemed furnished and not “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, and shall not be incorporated by reference in any filing under the Securities Act of 1933, as amended or the Exchange Act, except as shall be expressly set forth by specific reference in such filing.
| Item 8.01 | Other Information. |
On June 26, 2025, the Company announced 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.
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.
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.
Cautionary Note Regarding Forward Looking Statements
This Current Report on Form 8-K and press release attached hereto 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 Current Report on Form 8-K and 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 Current Report 8-K 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.
| Item 9.01 | Financial Statements and Exhibits. |
(d) Exhibits
| Exhibit Number |
Description | |
| 99.1 | Press Release, dated June 26, 2025. | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document). | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| EDGEWISE THERAPEUTICS, INC. | ||
| By: | /s/ R. Michael Carruthers | |
| R. Michael Carruthers | ||
| Chief Financial Officer | ||
Date: June 26, 2025
FAQ
What did Edgewise Therapeutics (EWTX) announce in its June 26 2025 8-K?
How much did NSAA scores improve for Becker patients in the MESA trial?
Did the FDA support a fast-track approval for sevasemten?
When will topline data from the GRAND CANYON pivotal study be available?
What dose of sevasemten will be used in Phase 3 Duchenne trials?
What is the current safety profile of sevasemten?
