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Edgewise Therapeutics Announces Positive 12-Month Topline Results from the ARCH Open Label Study of EDG-5506 in Adults with Becker Muscular Dystrophy (BMD)

Published: 2023-06-26 20:01:00 ET
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– Sustained positive trend in North Star Ambulatory Assessment (NSAA) scores relative to BMD natural history –

– Significant decreases in levels of serum creatine kinase (CK) and fast skeletal muscle troponin I (TNNI2), enzyme biomarkers associated with skeletal muscle damage–

– EDG-5506 was well-tolerated at all doses –

– Company continuing to enroll patients with BMD in the CANYON Phase 2 trial which now includes a pivotal cohort called GRAND CANYON –

– Edgewise leadership to discuss ARCH findings on Tuesday, June 27 at 8:30 a.m. Eastern Time at virtual investor event –

BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, targeted, small molecule therapies for the treatment of devastating muscle disorders, 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).

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20230626317214/en/

Edgewise Therapeutics Figure 1. NSAA shows stabilization and trend toward improvement after 12 months of dosing with EDG-5506 (Graphic: Business Wire)

Edgewise Therapeutics Figure 1. NSAA shows stabilization and trend toward improvement after 12 months of dosing with EDG-5506 (Graphic: Business Wire)

The ARCH study is evaluating varying doses of EDG-5506 administered daily over 24 months in 12 adults with BMD. The Company is reporting data at the end of 12 months of treatment with EDG-5506. EDG-5506 was well-tolerated in all participants with no discontinuations or dose reductions due to adverse events.

Consistent with prior observations, treatment with EDG-5506 led to significant decreases in key biomarkers of muscle damage. Importantly, CK and TNNI2 were reduced by an average of 37% (p=0.001) and 79% (p