Edgewise Therapeutics to Present on EDG-5506 for Becker and Duchenne Muscular Dystrophy (BMD, DMD) at the 2022 Annual MDA Clinical and Scientific Conference

March 8, 2022 13:00 UTC

• Podium presentations on March 15 and 16, 2022
• Company to host a conference symposium to discuss its new approach to protecting dystrophic muscle featuring a key opinion leader

BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the treatment of rare 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 Gaylord Opryland Resort & Convention Center in Nashville, TN, from March 13-16, 2022.

Details of the Edgewise presentations at MDA:

Platform Session: Small Molecule Approaches to Overcoming Muscle Wasting

Panelists: Helen Blau, Ph.D. (Co-Chair), Michael Rudnicki, OC, Ph.D., FRS, FRSC (Co-Chair) and Alan Russell, Ph.D., Chief Scientific Officer, Edgewise Therapeutics

Dr. Russell’s Presentation: Modulating Fast Skeletal Muscle Contraction as a Novel Therapeutic Strategy for Muscular Dystrophy

Date: Tuesday, March 15, 2022, 10:30 am-12 pm CT

Podium Presentation in Clinical Trial Outcomes Session

Title: EDG-5506 Targets Fast Skeletal Myosin to Protect Dystrophic Muscle and Reduce Muscle Damage Biomarkers in a Phase 1 Trial in Becker Muscular Dystrophy

Presenter: Joanne Donovan, M.D., Ph.D., Chief Medical Officer, Edgewise Therapeutics

Date: Wednesday, March 16, 2022, 10:10-10:25 am CT

Podium Presentation in Insights into Neuromuscular Disease Mechanisms Session

Title: Use of an Exercise Challenge System to Define a Universal Proteomic Signature of Muscle Injury in Adult Individuals with Diverse Inherited Myopathies

Presenter: Ben Barthel, Ph.D., Scientist II, Edgewise Therapeutics

Date: Wednesday, March 16, 2022, 11:45 am-12:00 pm CT

Scientific Posters

Companion poster presentations will be available for Dr. Donovan’s and Dr. Barthel’s presentations (posters 041 and 155). Posters will be viewable both in-person and virtually during the conference from March 13-March 15, 2022 and showcased in the Exhibit Halls from 6:00-8:00 pm CT. The full MDA 2022 Conference program is available here: https://mdaconference.org.

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

Edgewise Symposium with Key Opinion Leader

On March 14, 2022, at 7 am CT, Edgewise will sponsor a symposium, “A novel approach to protecting dystrophic muscle by targeting fast muscle myosin: EDG-5506.” The symposium will feature leading neuromuscular disease expert, Luca Bello, M.D., Ph.D, Assistant Professor, Department of Neurosciences, DNS, University of Padova, as well as Dr. Russell and Dr. Donovan. Only registered conference attendees can register for the symposium.

About EDG-5506 for DMD and BMD

EDG-5506 is an orally administered small molecule designed to address muscle damage induced by mechanical stress in dystrophinopathies including DMD and BMD. EDG-5506 presents a novel mechanism of action to selectively limit the exaggerated muscle damage caused by the absence of functional dystrophin. 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 a synergistic or additive effect in combination with available therapies and therapies currently in development. In August 2021, the U.S. Food and Drug Administration 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 PD of EDG-5506 in adult healthy volunteers (Phase 1a) and in adults with BMD (Phase 1b). Go to clinicaltrials.gov to learn more about this clinical trial (NCT04585464). A follow-on open-label, single-center study is assessing the long-term safety and PK of EDG-5506 in adults with BMD (NCT05160415). We anticipate initiation of Phase 2 trials in individuals with BMD in the first half of 2022 and DMD in the second half of 2022.

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. To learn more, go to: www.edgewisetx.com 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; statements regarding Edgewise’s expectations relating to its results from its preclinical studies and clinical trials; statements about the expected timing of Edgewise’s initiation of its preclinical studies and clinical trials including its Phase 2 clinical trials of EDG-5506 in BMD and DMD; 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; 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 Securities and Exchange Commission (the “SEC”). 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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