Edgewise Therapeutics Provides Corporate Update and Highlights Priorities for 2025

Rhea-AI Summary

Edgewise Therapeutics (NASDAQ: EWTX) provided corporate updates and priorities for 2025, highlighting progress in their muscular dystrophy and cardiovascular programs. The company's lead drug sevasemten, a first-in-class fast skeletal myosin inhibitor for Becker and Duchenne muscular dystrophies, is nearing completion of pivotal cohort enrollment. The company plans to seek FDA feedback on Phase 2 results in first half 2025.

For their cardiovascular program, EDG-7500, a cardiac sarcomere modulator for hypertrophic cardiomyopathy (HCM), will report initial 28-day data in Q1 2025 and 12-week trial results in H2 2025. Key 2024 achievements include raising $232 million through a public offering, positive Phase 2 CANYON trial results for Becker muscular dystrophy, and positive Phase 1 results for EDG-7500 in HCM patients.

Positive

• Raised $232 million through public offering in January 2024
• Positive Phase 2 CANYON trial results for sevasemten in Becker muscular dystrophy
• 99% enrollment rate in MESA Phase 2 extension trial
• Obtained Fast Track designation from FDA and Orphan Drug Designations from EMA
• Positive Phase 1 results for EDG-7500 in HCM patients

Negative

• None.

Insights

Biotech Research Analyst

The corporate update reveals substantial progress in Edgewise's muscular dystrophy and cardiac programs. The completion of GRAND CANYON pivotal trial recruitment in Q1 2025 for Becker muscular dystrophy (BMD) represents a critical milestone, as positive data could lead to the first approved therapy for this indication. The $232 million capital raise significantly strengthens their financial position, providing runway for key clinical developments.

The sevasemten program's multiple positive readouts in 2024, including the CANYON Phase 2 trial and two-year ARCH data, build confidence in the drug's potential. The dual Fast Track (FDA) and Orphan Drug (EMA) designations accelerate the regulatory pathway. EDG-7500's positive early data in hypertrophic cardiomyopathy (HCM) suggests potential expansion into a $4+ billion market opportunity.

Clinical Development Expert

The robust clinical development strategy across multiple patient populations demonstrates exceptional pipeline execution. The parallel advancement of sevasemten in both Becker and Duchenne muscular dystrophy shows sophisticated trial design, particularly the FOX study examining post-gene therapy patients - a important unmet need. The 99% retention rate in the MESA extension study suggests strong patient benefit perception.

EDG-7500's rapid progression from Phase 1 to multiple Phase 2 arms indicates compelling early safety and efficacy signals. The expansion into both obstructive and non-obstructive HCM populations maximizes the therapeutic potential while gathering comprehensive safety data. The planned IND for a second-generation heart failure candidate reflects confidence in the platform and strategic portfolio expansion.

- Advancing robust clinical pipeline of novel therapeutics for serious muscular dystrophies and cardiac conditions -

- Presenting at the 43^rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025 at 1:30 p.m. PT (4:30 p.m. ET) -

BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today provided recent corporate updates and highlighted upcoming priorities for 2025. Edgewise Chief Executive Officer, Kevin Koch, Ph.D., will present these updates today at the Annual J.P. Morgan Healthcare Conference.

“We were pleased with our progress in 2024 as we advanced our drug pipeline and have great momentum heading into 2025. With positive Phase 2 results and enrollment nearing completion in our pivotal cohort for individuals with Becker muscular dystrophy, we are optimistic about the potential of sevasemten,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise Therapeutics. “Additionally, we are thrilled with initial results from EDG-7500 in patients with obstructive hypertrophic cardiomyopathy and look forward to sharing additional, longer-term data across HCM populations in 2025.”

2025 Priorities

Muscular Dystrophy Program

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. There are currently no approved therapies for individuals with Becker, a serious genetic, progressive neuromuscular disorder.

• Complete recruitment of the GRAND CANYON pivotal placebo-controlled cohort in the first quarter of 2025
• Seek end of Phase 2 feedback from the U.S. Food and Drug Administration (FDA) on CANYON results for sevasemten in Becker in the first half of 2025
• Report data from the Phase 2 LYNX and FOX trials in the first half of 2025 and outline potential Phase 3 plans in individuals with Duchenne

Cardiovascular and Cardiometabolic Programs

EDG-7500 is a novel oral, selective, cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with hypertrophic cardiomyopathy (HCM) and other diseases of diastolic dysfunction.

• Report initial CIRRUS-HCM 28-day data in the first quarter of 2025
• Release data from the 12-week CIRRUS-HCM trial in individuals with obstructive HCM and non-obstructive HCM by the second half of 2025
• File an investigational new drug application for a second-generation heart failure candidate
• Select a proprietary cardiometabolic drug candidate from preclinical proof of concept data

2024 Accomplishments

Financial

• Strengthened balance sheet with net proceeds of approximately $232 million from January 2024 public follow-on offering supporting our muscular dystrophy and cardiovascular programs​

Muscular Dystrophy Program / Sevasemten

• Reported positive topline data from the CANYON Phase 2 placebo-controlled trial in adults with Becker
• Substantially enrolled the GRAND CANYON global pivotal cohort of sevasemten in adults with Becker; data from GRAND CANYON, if positive, could support a marketing application
• Advanced the MESA Phase 2 open label extension trial in adults with Becker, which to date has enrolled 99% of eligible participants completing prior Edgewise Becker trials
• Reported positive two-year topline results from the ARCH open label trial of sevasemten in adults with Becker
• Advanced the LYNX Phase 2 trial, a 2-part, dose-finding trial to evaluate the effect of sevasemten in children aged 4 to 9 years with Duchenne
• Advanced the FOX Phase 2 trial, a Phase 2 placebo-controlled trial to assess the effect of sevasemten in children and adolescents aged 6 to 17 years with Duchenne who have been previously treated with gene therapy
• Obtained Fast Track designation for sevasemten for the treatment of Duchenne from the FDA and Orphan Drug Designations for sevasemten for the treatment of Becker and for the treatment of Duchenne from the European Medicines Agency

Cardiovascular Program / EDG-7500

• Reported positive topline data of EDG-7500 in the Phase 1 trial in healthy subjects
• Reported positive topline data of EDG-7500 from the single-dose arm of the Phase 2 CIRRUS-HCM trial in patients with obstructive HCM
• Opened and began enrolling the 28-day arms of CIRRUS-HCM in patients with obstructive HCM and non-obstructive HCM

J.P. Morgan Healthcare Conference Presentation and Webcast

Edgewise management will highlight these updates in a corporate presentation today at the 43^rd Annual J.P. Morgan Healthcare Conference at 1:30 pm PT (4:30 pm ET). The presentation will be webcast live; a link for the webcast can be found on the Edgewise Events & Presentations page and will be accessible for replay, for a limited time, following the conference. It is recommended that users connect to the live webcast several minutes prior to the start to ensure a timely connection.

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 first-in-class fast 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.

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, Edgewise’s product candidates and programs, including sevasemten and EDG-7500; statements regarding Edgewise’s expectations relating to its clinical trials, including timing of reporting data (including the LYNX and FOX Phase 2 trials, CIRRUS-HCM 28-day and 12-week data); the timing of the completion of recruitment of the GRAND CANYON trial; statements regarding the advancement of Edgewise’s research and development programs; the timing of the initiation of a Phase 3 trial of sevasemten in Duchenne; the timing of receiving Phase 2 feedback from the FDA on sevasemten in Becker; the timing of filing an investigational new drug application for a second-generation heart failure candidate; the timing of selecting a proprietary cardiometabolic drug candidate; Edgewise’s 2025 priorities; the possibility of data from GRAND CANYON to support a marketing application; statements regarding Edgewise’s pipeline of product candidates and programs; statements regarding Edgewise’s anticipated milestones; and statements by Edgewise’s President and Chief Executive 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 EDG-7500 and discover, develop and commercialize product candidates in future programs, including risks relating to recruiting patients for its trials; 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 being lengthy, time consuming and inherently unpredictable; 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.

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Edgewise Contacts

Investors:

Behrad Derakhshan, Ph.D., Chief Business Officer

ir@edgewisetx.com

Media:

Maureen Franco, VP Corporate Communications

media@edgewisetx.com

Source: Edgewise Therapeutics

FAQ

When will Edgewise Therapeutics (EWTX) report CIRRUS-HCM trial data for EDG-7500?

Edgewise will report initial 28-day CIRRUS-HCM data in Q1 2025, followed by 12-week trial results in both obstructive and non-obstructive HCM patients in H2 2025.

How much capital did EWTX raise in their January 2024 public offering?

Edgewise Therapeutics raised approximately $232 million in net proceeds through their January 2024 public follow-on offering.

What are the key regulatory milestones for EWTX's sevasemten in 2025?

EWTX plans to seek FDA end of Phase 2 feedback on CANYON results for sevasemten in Becker muscular dystrophy in the first half of 2025.

What is the enrollment status of EWTX's GRAND CANYON pivotal trial?

The GRAND CANYON pivotal placebo-controlled cohort is expected to complete recruitment in the first quarter of 2025.

What regulatory designations has sevasemten received from FDA and EMA?

Sevasemten received Fast Track designation from FDA for Duchenne treatment and Orphan Drug Designations from EMA for both Becker and Duchenne treatments.