Edgewise Therapeutics today announced topline 12-month data from the ARCH study of EDG-5506, an orally administered small molecule designed to prevent contraction-induced muscle damage in patients with Becker muscular dystrophy.
The ARCH study is evaluating varying doses of EDG-5506 administered daily over 24 months in 12 adults with Becker. Highlights of the 12-month data 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
Edgewise reported that the positive results from the 12-month ARCH study support the hypothesis that a reduction in contraction-induced muscle damage in muscular dystrophies, associated with EDG-5506 administration, has the potential to preserve and improve muscle function while preventing disease progression in dystrophinopathies.
Edgewise will be participating in PPMD’s Annual Conference in Dallas, Texas on Friday, June 30 during the Research Row: Impacting Downstream sessions. Click here for more information on PPMD’s Annual Conference and registration.