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Edgewise Therapeutics

Edgewise Therapeutics: DMD/BMD Clinical Trial Updates

August 10, 2023

Edgewise Therapeutics has provided updates on their Duchenne and Becker muscular dystrophy clinical trials:

Positive 12-month topline results from the ARCH open-label study of EDG-5506 in adults with Becker muscular dystrophy

The Company announced positive 12-month topline results, 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, treatment with EDG-5506 from the ongoing ARCH study, led to significant decreases in key biomarkers of muscle damage.. The positive results from the 12-month ARCH study support the hypothesis that a reduction in contraction-induced muscle damage in muscular dystrophies, has the potential to preserve and improve muscle function while preventing disease progression in dystrophinopathies. Go to clinicaltrials.gov to learn more about this study (NCT05160415).

Advancing Phase 2 trial of EDG-5506 (CANYON study) in Becker muscular dystrophy, which now includes a pivotal cohort called GRAND CANYON

GRAND CANYON is anticipated to recruit approximately 120 Becker patients who will receive study drug for 18 months. GRAND CANYON is anticipated to begin enrollment in the second half of 2023. Go to clinicaltrials.gov to learn more (NCT05291091).

Advancing Phase 2 trial of EDG-5506 (LYNX study) in Duchenne muscular dystrophy

The Company is recruiting the LYNX Phase 2 clinical trial of EDG-5506 in children with Duchenne. LYNX is a placebo-controlled trial to assess the effect of multiple doses of EDG-5506 over 12 weeks on safety, PK and biomarkers of muscle damage. Approximately 27 children with Duchenne aged 4 to 9 years on stable corticosteroids and/or exon skippers are expected to be enrolled at up to 14 sites across the United States. Participants will then continue in an open-label extension portion of the trial for a total of 12 months to gain further insights into safety and functional measures. Importantly, this trial is designed to identify the optimal dose of EDG-5506 that reduces biomarkers of muscle damage and has the potential to provide functional benefit to patients in a Phase 3 trial. The Company expects to report Phase 2 interim data in the fourth quarter of 2023. Go to clinicaltrials.gov to learn more about this trial (NCT05540860).

Advancing DUNE Phase 2 Trial of EDG-5506 in Adults with LGMD2I/R9, Becker and McArdle Disease

The Company is continuing to recruit the DUNE Phase 2 exercise challenge study, to evaluate the effect of EDG-5506 on biomarkers of muscle damage following exercise in adults with LGMD2I/R9, Becker or McArdle disease at a single site in Denmark. Participants in the placebo-controlled study are enrolled for 16 weeks, then continue to an open label extension through 52 weeks. The goal of this trial is to assess safety and efficacy in individuals with myopathy distinct from Duchenne/Becker where muscle contraction is associated with exaggerated injury.

“Edgewise has made significant progress in the first half of the year most notably with the positive ARCH data that have allowed us to quickly pivot our CANYON study to include a potentially registration-enabling cohort in individuals with Becker, a disorder for which there are no approved treatments.

The team continues to advance our LYNX, CANYON and DUNE clinical trials of EDG-5506 in addition to advancing EDG-7500, our novel sarcomere modulator for HCM, towards the clinic. Over the coming months, we expect to start enrolling GRAND CANYON and a Phase 1 trial of EDG-7500 and share interim Duchenne data from our Phase 2 LYNX trial.”

Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise