– GRAND CANYON is the first pivotal study of an investigational therapy for Becker– – CANYON, the initial Phase 2 cohorts, is fully enrolled –

BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced the start of enrollment of GRAND CANYON, a global pivotal study of EDG-5506 in individuals with Becker. GRAND CANYON is an expansion of the CANYON study. CANYON, which was over-enrolled, includes 39 adults and 24 adolescents. EDG-5506 is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Becker and Duchenne muscular dystrophy. There are currently no approved therapies for individuals with Becker, a serious genetic, progressive neuromuscular disorder with significant unmet need.

GRAND CANYON is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of EDG-5506 in adults with Becker. Data from GRAND CANYON, if positive, could support a marketing application. The primary endpoint of GRAND CANYON is North Star Ambulatory Assessment (NSAA). In addition, other functional assessments, biomarkers of muscle damage and safety will be assessed. GRAND CANYON is anticipated to recruit approximately 120 individuals with Becker, aged between 18 and 50 years old, at up to 50 sites in 10 countries. The treatment period for participants will be 18 months. To learn more, go to clinicaltrials.gov (NCT05291091) or the GRAND CANYONmicrosite: https://www.beckergcstudy.com.

“Based on the strength of safety, functional and biomarker results from our ARCH open label study, we have rapidly initiated this ground-breaking clinical study,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise. “This study is critical for the Becker community, as there currently are no approved treatments for Becker, a condition that for far too long has been neglected.”

“It is exciting to move to the next stage of testing of the clinical hypothesis that a reduction in contraction-induced muscle damage has the potential to benefit people living with muscular dystrophies,” said Alan Russell, Ph.D., Co-Founder and Chief Scientific Officer of Edgewise. “Having seen promising preclinical results with EDG-5506 translate clinically, I am thrilled we are entering into a pivotal study.”

Edgewise will host a community webinar with Parent Project Muscular Dystrophy on October 18, 2023, at 1:00 pm ET to share details about the trial and answer questions from the community. The registration page will be available on the PPMD website in early October.

Positive 12-Month Results from the ARCH Open Label Study of EDG-5506 in Adults with Becker

The ongoing ARCH study is an open label, single-center study assessing the safety, tolerability, impact on muscle damage biomarkers, function and pharmacokinetics (PK) of EDG-5506 in adults with Becker. The ARCH study is evaluating varying doses of EDG-5506 administered daily over 24 months in 12 adults with Becker. The Company reported data at the end of 12 months of treatment with EDG-5506. EDG-5506 was well-tolerated in all participants with no dose reductions or discontinuations due to adverse events.

Consistent with prior observations, significant decreases in key biomarkers of muscle damage were seen with treatment with EDG-5506. Importantly, creatine kinase (CK) and fast skeletal muscle troponin I were reduced by an average of 37% (p=0.001) and 79% (p