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Edgewise Therapeutics to Present on EDG-5506 for the Treatment of Becker Muscular Dystrophy at the 2024 MDA Clinical and Scientific Conference

Company to host an Industry Forum to discuss its approach to protecting and preserving dystrophic muscle featuring key opinion leaders –

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on EDG-5506, an investigational therapy designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies, at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. The conference will take place at the Hilton Orlando in Orlando, FL from March 3-6, 2024.

Details of the Edgewise presentations at MDA:

Podium Presentation in Clinical Trial Updates Session

Title: Functional and Muscle Damage Biomarker Changes Following Treatment with EDG-5506, a Fast Myosin Modulator, in Adults with Becker Muscular Dystrophy

Dr. Phan’s presentation will include 12-month data from the ARCH trial of EDG-5506.

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

Date: Wednesday, March 6, 2024, 11:15 – 11:30 am ET

Scientific Posters

Title: Comparison of Short- and Long-Term Proteomic Response to the Fast Skeletal Myosin Inhibitor, EDG-5506, in Becker Muscular Dystrophy (BMD) (Poster # M145)

Title: North Star (NSAA and NSAD) Functional Assessments in Individuals with Becker Muscular Dystrophy (poster # M147)

A companion poster presentation will be available for Dr. Phan’s presentation (poster # M146). Posters will be showcased during the Networking & Poster Reception in the Exhibit Hall on Monday, March 4, 2024, from 6 – 8 PM ET. The full MDA 2024 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 Leaders

On March 4, 2024, at 12 pm ET, Edgewise is sponsoring an Industry Forum, “Protecting and preserving dystrophic muscle: The balance between exercise and contraction-induced muscle injury.” The forum will feature presentations by leading neuromuscular disease experts, John Vissing, M.D., Ph.D., Professor of Neurology, Director Neuromuscular Clinic and Research Unit, Department of Neurology, University of Copenhagen, and Tanja Taivassalo, Ph.D., Associate Professor, Department of Physiology and Aging, University of Florida, as well as Edgewise Chief Medical Officer, Joanne Donovan, M.D., Ph.D. Only registered conference attendees can register for the symposium.

About EDG-5506 for Duchenne and Becker Muscular Dystrophies

EDG-5506 is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Duchenne and Becker. 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 minimizing the progressive muscle damage that leads to functional impairment, EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating neuromuscular disorders. Its unique mechanism of action provides the potential to establish EDG-5506 as a foundational therapy in dystrophinopathies, either as a single agent therapy or in combination with available therapies and those in development. The Company is advancing EDG-5506 through the clinic including completing enrollment of a Phase 2 trial cohort, called CANYON, evaluating safety and effects on function and biomarkers of muscle damage in adult males with Becker, which has been expanded to include an additional 120 adult participants in a pivotal cohort called GRAND CANYON, which is currently enrolling. In Duchenne, the Company is advancing its Phase 2 clinical trials, LYNX, assessing safety, pharmacokinetics and biomarkers of muscle damage, and FOX, which includes children and adolescents previously treated with gene therapy. For more information on Edgewise’s clinical trials

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: or follow us on LinkedIn, X (formerly Twitter), Facebook, Instagram and Threads.


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