– Company to host conference symposium to discuss its approach to protecting dystrophic muscle featuring key opinion leaders and 6-Month Interim Results from the ARCH Open Label Study of EDG-5506 in Adults with BMD –

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 27th International Hybrid Annual Congress of the World Muscle Society (WMS). The conference will take place at the Halifax Convention Centre in Halifax, Nova Scotia, from October 11-15, 2022.

Details of the Edgewise symposium and scientific posters at WMS:

Edgewise Symposium with Key Opinion Leaders

On Friday, October 14, 2022, at 7 am ADT, Edgewise will sponsor a symposium, “Targeting Fast Muscle Myosin: a Novel Approach to Protecting Muscle for the Dystrophinopathies.” As part of the symposium, Edgewise Chief Medical Officer, Joanne Donovan, M.D., Ph.D., will present 6-month interim results from the ongoing ARCH study, an open label, single-center study assessing the safety, tolerability, impact on muscle damage biomarkers, pharmacokinetics (PK), functional assessments and patient reported outcomes of EDG-5506 in adults with BMD. The symposium will also feature presentations by leading neuromuscular disease experts:

• Craig McDonald, M.D., Professor and Chair, Department of Physical Medicine & Rehabilitation, University of California Davis
  • Presentation: Clinical Course of Dystrophinopathies
• John Vissing, M.D., Ph.D., Professor of Neurology, University of Copenhagen, and Director, Copenhagen Neuromuscular Center at the National Hospital, Rigshospitalet, Copenhagen
  • Presentation: Mechanical Stress Induced Injury: Changes in Biomarkers of Dystrophic Muscle During Exercise in LGMD and BMD
• Alan Russell, Ph.D., Chief Scientific Officer, Edgewise Therapeutics
  • Presentation: Preferential Fast Fiber Injury in Dystrophinopathies, presented on behalf of Lee Sweeney, Ph.D., University of Florida, Department of Pharmacology and Therapeutics
  • Presentation: Targeting Fast Muscle Myosin to Decouple Injury from Muscle Contraction in DMD and BMD

Only registered conference attendees can register for the symposium. The symposium will be streamed to the virtual element of the event and available for replay via the conference platform.

Scientific Posters

Title: Patient Reported Aspects of Becker Muscular Dystrophy from The Duchenne Registry a Comprehensive Registry for Duchenne and Becker Muscular Dystrophy (BMD) (P.25)

Date: Wednesday, October 12, 2022, 2:30 – 4:00 pm ADT

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 (BMD) (P.124)

Date: Friday, October 14, 2022, 2:30 – 4:00 pm ADT

The full WMS Congress program is available here: https://www.wms2022.com/page/programme.

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

About EDG-5506

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 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 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 (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, a follow-on open-label, single-center trial (NCT05160415) assessing long-term safety and PK, decreases in biomarkers of muscle damage and improvements in NSAA have been observed. CANYON, a Phase 2 trial (NCT05291091) is assessing safety, PK, biomarkers and functional measures in participants with BMD. Recently, the FDA authorized LYNX, a Phase 2 trial (NCT05540860) of EDG-5506 for the treatment of DMD that will assess safety, PK and biomarkers of muscle damage.

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.

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