Edgewise Therapeutics announced today positive 12-month topline 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 designed to prevent contraction-induced muscle damage in dystrophinopathies including BMD and Duchenne muscular dystrophy (DMD).
The ARCH study is evaluating varying doses of EDG-5506 administered daily over 24 months in 12 adults with Becker. Edgewise is reporting data collected at the end of 12 months of treatment with EDG-5506, their investigational therapy. Highlights of the release include:
- Sustained positive trend in North Star Ambulatory Assessment (NSAA) scores relative to BMD natural history.
- Significant and sustained decreases in levels of serum creatine kinase (CK) and fast skeletal muscle troponin I (TNNI2), enzyme biomarkers associated with skeletal muscle damage.
- EDG-5506 continues to be well-tolerated at all doses studied with no discontinuations or dose adjustments due to adverse events.
- Individuals with Becker continue to be enrolled in the CANYON Phase 2 trial.
Please read the full press release here
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