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Edgewise Therapeutics to Present on EDG-5506 for Becker and Duchenne Muscular Dystrophy (BMD, DMD) at the 2023 Annual MDA Clinical and Scientific Conference

Podium presentation on March 22, 2023 –

Company to host an Industry Forum to discuss its new approach to protecting dystrophic muscle featuring a key opinion leader

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, targeted, small molecule therapies for the treatment of devastating muscle disorders, today announced that the company will present on EDG-5506, an investigational therapy designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies, at the Muscular Dystrophy Association (MDA) Annual Clinical and Scientific Conference. The conference will take place at the Hilton Anatole in Dallas, TX from March 19-22, 2023.

Details of the Edgewise presentations at MDA:

Podium Presentation in Clinical Trial Updates Session

Title: Effects of EDG-5506, a Fast Myosin Modulator, on Biomarkers of Muscle Damage and Function in Adults with Becker Muscular Dystrophy (BMD) (Poster #73)

Presenter: Han C. Phan, M.D., Head of Research & Principal Investigator, Pediatric Neurologist, Rare Disease Research, LLC

Date: Wednesday, March 22, 2023, 2:30 – 2:45 pm CT

Scientific Posters

Title: Plasma Metabolic Profiling Suggests Enrichment of Glycolytic Pathways Over Fatty Acid Oxidation in Becker Muscular Dystrophy (Poster #71)

Title: Fast Skeletal Myosin Modulator EDG-5506 Confers Additional Protection to MDX Mice Receiving Glucocorticoid (Poster #72)

Title: Characterization of Short- and Long-Term Proteomic Response to the Fast Skeletal Myosin Inhibitor, EDG-5506, in Becker Muscular Dystrophy (Poster #119)

A companion poster presentation will be available for Dr. Phan’s presentation (poster 73). Posters will be viewable both in-person during the conference from March 19-21, 2023 and showcased in the Exhibit Halls from 6:00-8:00 pm CT. The full MDA 2022 Conference program is available here:

The Edgewise presentation and posters will be available on the Edgewise website when they are presented.

Edgewise Industry Forum with Key Opinion Leader

On March 21, 2023, at 12 pm CT, Edgewise is sponsoring an Industry Forum, “Targeting Fast Muscle Myosin: A Novel Approach to Protecting Muscle in the Dystrophinopathies.” The forum will feature leading neuromuscular disease expert, Barry J. Byrne, M.D., Ph.D., Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida, as well as Edgewise leadership including Alan Russell, Ph.D., Chief Scientific Officer, Joanne Donovan, M.D., Ph.D., Chief Medical Officer, and Sam Collins, MBBS, Ph.D., Vice President of Clinical Development. Only registered conference attendees can register for the symposium.

About EDG-5506

EDG-5506 is an orally administered small molecule designed to prevent muscle damage induced by mechanical stress in dystrophinopathies including DMD and BMD. EDG-5506 presents a novel mechanism of action designed to selectively limit the exaggerated muscle damage caused by the absence or loss of functional dystrophin. By impacting the progressive muscle damage that leads to functional impairment, EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It is anticipated to be used as a single agent therapy, but it may also provide an additional benefit in combination with available therapies and therapies currently in development. In August 2021, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to EDG-5506 for the treatment of individuals with BMD.

The Company has completed a Phase 1 clinical trial of EDG-5506 designed to evaluate safety, tolerability, PK and pharmacodynamics of EDG-5506 in adult healthy volunteers (Phase 1a) and in adults with BMD (Phase 1b) (NCT04585464). In ARCH, an open-label, single-center trial (NCT05160415) assessing long-term safety and PK, decreases in biomarkers of muscle damage and trends toward improvement in NSAA have been observed. CANYON, a Phase 2 trial (NCT05291091) is assessing safety, PK, biomarkers and functional measures in participants with BMD. LYNX, a Phase 2 trial (NCT05540860) is assessing safety, PK and biomarkers of muscle damage in participants with DMD.

About Edgewise Therapeutics

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of innovative treatments for severe, rare neuromuscular and cardiac disorders for which there is significant unmet medical need. Guided by its holistic drug discovery approach to targeting the muscle as an organ, Edgewise has combined its foundational expertise in muscle biology and small molecule engineering to build its proprietary, muscle-focused drug discovery platform. Edgewise’s platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue. The Company’s lead candidate, EDG-5506, an investigational orally administered small molecule designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies, is advancing in multiple clinical trials in individuals with Duchenne, Becker and Limb Girdle 2I/R9 muscular dystrophies, and McArdle’s disease. The Company is also advancing EDG-7500, a novel sarcomere modulator for hypertrophic cardiomyopathy, into IND-enabling preclinical development. To learn more, go to: or follow us on LinkedIn, Twitter and Facebook.

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 drug discovery platform, product candidates and programs, including EDG-5506 and EDG-7500; and statements regarding Edgewise’s pipeline of product candidates and programs. 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 preclinical studies and clinical trials for, obtain approvals for and commercialize any of its product candidates for muscular dystrophy patients or other patient populations; the timing, progress and results of preclinical studies and clinical trials for EDG-5506 and the EDG-7500; Edgewise’s ability to obtain IND clearance for EDG-7500; Edgewise’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; negative impacts of the COVID-19 pandemic on Edgewise’s operations, including preclinical and clinical trials; 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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