Edgewise Therapeutics, Inc. announced an expansion of their clinical development program of EDG-5506, an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne. The Company is initiating FOX, a new Phase 2 placebo-controlled trial in children and adolescent boys with Duchenne who have been previously treated with gene therapy. Further, the Company is continuing dose escalation and expanding enrollment in their Phase 2 placebo-controlled LYNX trial; one of the new LYNX cohorts will study EDG-5506 in boys with Duchenne not currently treated with corticosteroids.

With strong interest from the Duchenne community and supportive initial safety data from LYNX, the Company initiated the FOX trial, a new Phase 2 placebo -controlled trial in children and adolescents with Duchenne who have be previously treated with gene therapy. LYNX is designed to identify a dose of EDG-5506 that will reduce biomarkers of muscle damage and has the potential to provide functional benefit to patients in a Phase 3 trial. The Company expects to report Phase 2 interim data in the first half of 2024, with the Phase 3 dose identified.

Additionally, the Company is adding a new cohort to LYNX to include children aged 4 to 9 years with Duchenne who are not currently treated with corticostoids. 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. In August 2021, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to EDG-5506 for the treatment of individuals with Becker.

The Company has advanced EDG-5506 through the clinic including completed a Phase 1 trial evaluating safety, tolerability, PK and pharmacodynamics of EDG-5506 in adult healthy volunteers (Phase 1a) and in adults with Becker (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 following 12 months of treatment with EDG-5506. 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. Such forward-looking statements include, among other things, statements regarding the potential of, and expectations regarding EDG-5506; statements regarding Edgewise's expectations relating to its clinical trials, including the timing of reporting data (including the Phase 2 interim data from the LYNX clinical trial of EDG-5506) and commencement of trials (including the FOX clinical trial and Phase 3 clinical trial of EDG- 5506 Duchenne); statements regarding Edgewise's pipeline of product candidates and programs; and statements by Edgewise's Chief Medical Officer.

Edgewise's ability to develop, initiate or complete clinical trials for, obtain approvals for and commercialize any of its product candidates; the timing, progress and results of clinical trials forEDG-5506; 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 continue to continue to continue the Phase 2 placebo-controlled trial.