Edgewise Therapeutics Touts Positive Data From Investigational Drug In Form Of Muscular Dystrophy

Edgewise Therapeutics Inc EWTX released 12-month topline results from the ARCH study, an open-label, single-center study assessing EDG-5506 in adults with Becker Muscular Dystrophy (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 BMD.

Related: Flexing On Muscular Dystrophy, Analyst Expects Almost 200% Upside For Edgewise Therapeutics.

After 12 months of treatment, EDG-5506 was well-tolerated in all participants, with no discontinuations or dose reductions due to adverse events.

Treatment with EDG-5506 led to significant decreases in key biomarkers of muscle damage, including CK and TNNI2, reduced by an average of 37% (p=0.001) and 79% (p<0.0001) from baseline, respectively.

Nine of the twelve participants showed either a functional improvement (n=6) or exhibited stable disease (n=3) on North Star Ambulatory Assessment (NSAA) relative to their baselines after 12 months of EDG-5506 dosing.

The positive results from the 12-month ARCH study support the hypothesis that EDG-5506 administration reduces contraction-induced muscle damage in muscular dystrophies and has the potential to preserve and improve muscle function while preventing disease progression in dystrophinopathies.

Based on the ARCH study, a pivotal cohort, GRAND CANYON, has been added to the CANYON study and is anticipated to begin enrollment in Q3 of 2023.

Price Action: EWTX shares closed at $6.86 on Monday.