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Edgewise Therapeutics Announces Positive 6-Month Interim Results from the ARCH Open Label Study of EDG-5506 in Adults with Becker Muscular Dystrophy (BMD)
– Continued positive trend in North Star Ambulatory Assessment (NSAA) scores relative to BMD natural history trajectories – – Highly significant decreases in
About this update from Edgewise Therapeutics, Inc.
[{"type":"text","content":"\n– Continued positive trend in North Star Ambulatory Assessment (NSAA) scores relative to BMD natural history trajectories –\n\n– Highly significant decreases in levels of serum creatine kinase (CK) and fast skeletal muscle troponin I (TNNI2), enzyme biomarkers strongly associated with muscle damage observed in BMD –\n\n– EDG-5506 continues to be well-tolerated at higher doses –\n\n– Edgewise leadership to discuss findings on Friday, October 14 at the Annual Congress of the World Muscle Society –\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 6-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 investigational orally administered small molecule myosin modulator designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies such as Duchenne muscular dystrophy (DMD) and BMD.\nThis press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20221013005743/en/Figure 1. NSAA and NSAD Durable Improvements Observed Over 6 months of EDG-5506 Relative to Natural History (Graphic: Business Wire)\nThe twelve adults with BMD enrolled in the ARCH study initially received a 10 mg dose of EDG-5506 during the first 2 months, followed by a 15 mg dose during the subsequent 4 months of the study. The Company is reporting data at the end of 6 months of treatment with EDG-5506. Plasma PK levels reached target exposures and EDG-5506 continues to be well-tolerated in all participants with no discontinuations or dose reductions. The most common adverse events observed to date were dizziness, drowsiness, and headache. All participants have now been dose escalated to daily 20 mg oral doses of EDG-5506.\n\nConsistent with prior observations, treatment with EDG-5506 led to a significant decrease in key biomarkers of muscle damage when assessed by laboratory assays. Importantly, CK and fast skeletal muscle troponin I were reduced by an average of 39% and 75%, respectively, after the participants most ...