Edgewise Therapeutics has announced 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 Becker muscular dystrophy.
The ARCH study evaluates varying doses of EDG-5506 administered daily over 24 months in 12 adults with Becker. This reports data collected at the end of 12 months of treatment with EDG-5506, an 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.
The positive results support the hypothesis that a reduction in contraction-induced muscle damage in muscular dystrophies associated with EDG-5506 administration can potentially preserve and improve muscle function while preventing disease progression. Observations from ARCH identified key factors, including the optimal dosing strategy of EDG-5506, for the design of a potentially registrational trial. A pivotal cohort, GRAND CANYON, has been added to the CANYON study and is anticipated to begin enrollment in the third quarter of 2023.
The Phase 2 dose-finding LYNX study in Duchenne is also making steady progress, and taken together with the ARCH data, we will continue to provide updates as they are available.