Edgewise: Positive Topline Results in Phase 1b Trial in Becker Muscular Dystrophy

 DG-5506 was well tolerated with no serious adverse events observed –

– Pharmacokinetic (PK) data demonstrated robust target engagement with achievement of muscle concentrations beyond levels predicted to provide meaningful clinical benefit –

– EDG-5506 treatment resulted in significant lowering of muscle damage biomarkers in adults with BMD after only two weeks of dosing –

– Management hosting webcast to discuss findings today at 9:00 a.m. Eastern Time –

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, announced today positive topline results from the BMD, or Phase 1b, portion of a first-in-human Phase 1 clinical trial assessing the safety, tolerability, PK and pharmacodynamics (PD) of EDG-5506, an orally administered small molecule myosin inhibitor designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies such as Duchenne muscular dystrophy (DMD) and BMD.

"BMD is a rare, serious, progressive, permanently debilitating, and potentially fatal inherited neuromuscular disorder, which causes great hardship to individuals with BMD and their families and caregivers," said Craig McDonald, M.D., Professor and Chair of the Department of Physical Medicine & Rehabilitation at UC Davis. "I am encouraged by the EDG-5506 Phase 1b clinical data in individuals with BMD, for which there are no approved treatments. I look forward to working with fellow investigators and the BMD and DMD communities in the upcoming Phase 2 trials of EDG-5506."

The seven adults with BMD enrolled in the Phase 1b clinical trial were administered 20 mg oral doses of EDG-5506 (n=5) or placebo (n=2). EDG-5506 was shown to be well-tolerated with no discontinuations or dose reductions. The most common adverse event, observed in all Phase 1b participants, was dizziness which was mild, transient and self-resolving.

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