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Edgewise Therapeutics Announces Positive Topline Results from the EDG-5506 Phase 1 Multiple Ascending Dose (MAD) Study in Healthy Volunteers (HVs) and Doses First Becker Muscular Dystrophy (BMD) Patients
– EDG-5506 was generally well tolerated with no serious adverse events observed to date – – Pharmacokinetic (PK) data demonstrated robust target engagement
About this update from Edgewise Therapeutics, Inc.
[{"type":"text","content":"\n– EDG-5506 was generally well tolerated with no serious adverse events observed to date –\n\n– Pharmacokinetic (PK) data demonstrated robust target engagement with achievement of muscle concentrations well above efficacious levels observed in preclinical disease models –\n\n– Pharmacodynamic (PD) data demonstrated dose proportionate effect on involuntary twitch inhibition in HVs –\n\n– Phase 1a single ascending dose/MAD package supports initiation of Phase 2 trials in BMD and Duchenne muscular dystrophy (DMD) in 2022 –\n\n– Management hosting webcast to discuss findings today at 9:00 a.m. Eastern Time –\n\n BOULDER, Colo.--(BUSINESS WIRE)--\nEdgewise 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 MAD portion of a first-in-human Phase 1 clinical trial assessing the safety, tolerability, PK and PD of escalating doses of EDG-5506, an orally administered small molecule myosin inhibitor designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies such as DMD and BMD.\n\n“We are extremely encouraged by these multiple ascending dose data, which clearly demonstrate high levels of target engagement with a well-tolerated dosing regimen. We are excited by the potential that EDG-5506 may serve as an oral treatment to decrease muscle damage, the root cause of DMD and BMD, and we eagerly look forward to better understanding its full potential in future clinical studies,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise.\n\nIn addition to safety, the goal in the MAD study was to derive a relationship between dose, muscle exposure and involuntary twitch force inhibition, a PD surrogate for EDG-5506’s novel mechanism of action. The 40 healthy volunteers enrolled in the MAD study were administered oral doses of EDG-5506 (5 mg to 40 mg) or placebo. EDG-5506 was shown to be generally well tolerated at all doses studied with no adverse events seen in laboratory values, electrocardiograms, or other vital signs. The most common adverse events were dizziness and somnolence, which were mild, transient and self-resolving. At projected therapeutic levels, as exposure increased over time, the incidence of adverse event...