BOULDER, Colo. / Jan 09, 2024 / Business Wire / Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, will present today at the 42nd Annual J.P. Morgan Healthcare Conference at 10:30 am PT (1:30 pm ET), and a live webcast will be available at www.edgewisetx.com. Ahead of the presentation, the Company highlighted its 2023 accomplishments and announced its anticipated key milestones for 2024.

“In 2023, we had tremendous progress in the clinic advancing our novel oral therapeutics that are designed to treat severe muscle conditions,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. “We are well positioned to execute on our 2024 milestones including important EDG-5506 data in muscular dystrophies, first-in-human studies in our EDG-7500 cardiac program, as well as preclinical exploration of novel cardiometabolic targets.”

2023 Accomplishments

Musculoskeletal Program – EDG-5506

Becker muscular dystrophy (Becker)

• Announced positive 12-month open label ARCH trial data. ARCH is an open label, single-center study assessing the safety, tolerability, impact on muscle damage biomarkers, function and pharmacokinetics (PK) of EDG-5506 in adults with Becker.
• Over-enrolled the Phase 2 CANYON placebo-controlled cohorts with 40 adults and 29 adolescents.
• Initiated GRAND CANYON, a global pivotal cohort in individuals with Becker. GRAND CANYON is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of EDG-5506 in adults with Becker.
• Initiated Phase 2 open-label extension MESA trial that will assess the long-term effect of EDG-5506 on safety, biomarkers and functional measures. MESA will provide continued access to EDG-5506 treatment to study participants who were previously enrolled in Edgewise studies.
• Advanced Phase 2 DUNE exercise challenge study to evaluate the effect of EDG-5506 on biomarkers of muscle damage following controlled exercise in adults with Becker, Limb Girdle muscular dystrophy2I/R9 or McArdle disease.
• U.S. Food & Drug Administration (FDA) granted EDG-5506 Orphan Drug Designation for the treatment of Becker.

Duchenne muscular dystrophy (Duchenne)

• Rapidly enrolled and expanded Phase 2 LYNX placebo-controlled trial in children aged 4 to 9 years with Duchenne. LYNX will assess the effect of multiple doses of EDG-5506 over 12 weeks on safety, PK and biomarkers of muscle damage. Participants will then continue in an open-label extension portion of the trial for a total of 24 months to gain further insights into safety and functional measures.
• Initiated Phase 2 FOX placebo-controlled trial that will assess the effect of EDG-5506 over 12 weeks on safety, PK and biomarkers of muscle damage in children and adolescents with Duchenne who have been previously treated with gene therapy. Participants will then continue in an open-label extension portion of the trial for a total of 12 months to gain further insights into safety, PK, function and biomarker measures.
• FDA granted EDG-5506 Rare Pediatric Disease Designation for the treatment of Duchenne.
• FDA granted EDG-5506 Orphan Drug Designation for the treatment of Duchenne.

Cardiac Program – EDG-7500

Hypertrophic Cardiomyopathy (HCM)

• Expanded the pipeline to include EDG-7500, a first-in-class cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with HCM and other diseases of diastolic dysfunction.
• Initiated first-in-human Phase 1 trial of EDG-7500, a randomized, double-blind, placebo-controlled, single and multiple ascending dose study evaluating safety, tolerability, PK and pharmacodynamics in healthy adults.

Anticipated 2024 Key Milestones

Musculoskeletal Program – EDG-5506

Becker muscular dystrophy

• Report 24-month data from the open label ARCH trial in the first half of 2024.
• Continue to advance the Phase 2 CANYON placebo-controlled study, announce 1-year data in the second half of 2024.
• Announce Phase 2 DUNE (Becker, LGMD, McArdle) exercise challenge data in the first half of 2024.

Duchenne muscular dystrophy

• Continue to advance Phase 2 LYNX trial, announce 3-month controlled dose-ranging data in the first half of 2024.
• Pending data from ongoing EDG-5506 studies, initiate Phase 3 trial in the second half of 2024.

Cardiac Program – EDG-7500

Hypertrophic Cardiomyopathy

• Announce Phase 1 data in healthy volunteers and individuals with obstructive hypertrophic cardiomyopathy in the second half of 2024.

Presentation at the 42nd Annual J.P. Morgan Healthcare Conference

Edgewise will webcast its presentation today from the J.P. Morgan 42nd Annual Healthcare Conference at 10:30 am PT (1:30 pm ET). To access the live webcast, please visit the Edgewise events page at https://investors.edgewisetx.com/. A replay of the webcast will be available on Edgewise’s website for a limited time following the conference.

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 first-in-class therapeutics. EDG-5506 is an orally administered skeletal myosin inhibitor in clinical trials in patients with Becker, Duchenne, and Limb-Girdle muscular dystrophies as well as McArdle Disease. EDG-7500, currently in a Phase 1 trial, is a novel cardiac sarcomere modulator for the treatment of HCM and other disorders of cardiac diastolic dysfunction. 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 (formerly Twitter), Facebook, Instagram and Threads.

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 EDG-5506 and EDG-7500; statements regarding Edgewise’s expectations relating to its clinical trials, including timing of reporting data (including the 24-month data from the open label ARCH trial, the 1-year data for the Phase 2 CANYON trial; the Phase 2 DUNE data; the 3-month data for the Phase 2 LYNX trial; and the Phase 1 data for the EDG-7500 cardiac program) and commencement of trials (including the Phase 3 trial for EDG-5506); 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 2024 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 the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early clinical stage company including the potential for Edgewise’s product candidates to cause serious adverse events; Edgewise’s ability to develop, initiate or complete clinical trials for, obtain approvals for and commercialize any of its product candidates; Edgewise’s ability to take advantage of potential benefits associated with designations granted by FDA and/or to maintain qualifications for applicable designations over time; the timing, progress and results of clinical trials for EDG-5506 and EDG-7500; Edgewise’s ability to enroll and maintain patients in clinical trials; Edgewise’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; the timing, scope and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; Edgewise’s ability to develop a proprietary drug discovery platform to build a pipeline of product candidates; Edgewise’s manufacturing, commercialization and marketing capabilities and strategy; the size of the market opportunity for Edgewise’s product candidates; the loss of key scientific or management personnel; competition in the industry in which Edgewise operates; Edgewise’s reliance on third parties; Edgewise’s ability to obtain and maintain intellectual property protection for its product candidates; 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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