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Edgewise Therapeutics Announces Positive 2-Month Interim Results from the ARCH Open Label Study of EDG-5506 in Adults with Becker Muscular Dystrophy (BMD)
– EDG-5506 treatment significantly lowered muscle damage biomarkers in adults with BMD – – BMD patients were more active during 2 months of dosing with
About this update from Edgewise Therapeutics, Inc.
[{"type":"text","content":"\n– EDG-5506 treatment significantly lowered muscle damage biomarkers in adults with BMD –\n\n– BMD patients were more active during 2 months of dosing with EDG-5506 relative to activity measured in BMD patients in the Phase 1 study –\n\n– EDG-5506 was well tolerated with no serious adverse events observed; safety profile supported dose escalation to 15 mg daily for all patients –\n\n– Management hosting webcast to discuss findings on June 21 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 2-month interim results from the ongoing ARCH study, an open label, single-center study assessing the safety, tolerability, impact on muscle damage biomarkers, and pharmacokinetics (PK) of EDG-5506 in adults with BMD. EDG-5506 is 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.\n\nThe twelve adults with BMD enrolled in the ARCH study, including all seven participants from the Phase 1b study, were administered daily 10 mg oral doses of EDG-5506 at night. EDG-5506 was well-tolerated with no discontinuations or dose reductions. The most common adverse events observed at the 10 mg dose were dizziness (17%) and somnolence (17%). These were generally transient and typically observed in the first few days of dosing. All patients have subsequently been dose escalated to 15 mg daily as per protocol.\n\nThe 2-month plasma PK data for the 10 mg once-daily dose of EDG-5506 showed exposure levels that were approximately 61% of what was observed in the Phase 1b study where BMD participants were dosed with 20 mg EDG-5506 once-daily for two weeks. The reduced exposure for BMD participants in the ARCH study is consistent with the lower dose of 10 mg and a shorter half-life of EDG-5506 in BMD patients, likely associated with a decreased overall muscle mass.\n\nTreatment with EDG-5506 led to a significant decrease in key biomarkers of muscle damage when assessed by laboratory assays. Importantly, creatine kinase (CK) and fast skeletal muscle troponin I (TNNI2) were reduced by an ...