Positive 12-month results reported in study of Becker muscular dystrophy therapeutic

Key takeaways:

• Treatment with EDG-5506 resulted in significant reduction in two biomarkers of muscle damage.
• Nine of 12 participants showed functional improvement or stable disease via North Star Ambulatory Assessment scores.

Edgewise Therapeutics Inc. announced positive 12-month topline results from the ARCH study evaluating the safety, tolerability and pharmacokinetics of EDG-5506 in adults with Becker muscular dystrophy.

According to a company release, EDG-5506 is an oral small molecule created to prevent contraction-induced muscle damage in dystrophic diseases, such as Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy.

The ongoing ARCH study is assessing varying doses of EDG-5506 given daily to 12 adults with BMD over 24 months. At 12 months, the company reported EDG-5506 was well-tolerated in all participants with no discontinuations or dose reductions due to adverse events.

In addition, treatment led to significant decreases in key biomarkers serum creatine kinase and fast skeletal muscle troponin 1 by an average of 37% and 79% from baseline, respectively, at 12 months, the release stated. Further, nine of 12 participants demonstrated functional improvement (n = 6) or stable disease (n = 3) via North Star Ambulatory Assessment compared with baseline.

“We are quite pleased by the robust and consistent results observed at 12 months with EDG 5506 treatment, which, together with the favorable safety/tolerability profile, are highly encouraging,” Joanne Donovan, MD, PhD, chief medical officer at Edgewise, said in the release. “We thank the patients, their families, caregivers and the broader Becker community, who are currently underserved with no available treatment options, for engaging with us on this promising therapy.”

According to the release, the FDA in August 2021 granted fast track designation to EDG-5506 for treatment of patients with BMD.