Edgewise-Funded Natural History Trial Of Becker Muscular Dystrophy (BMD) Now Enrolling
By: Colorado BioScience Association Date: 04/15/2022
Edgewise Therapeutics, Inc. (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the treatment of rare muscle disorders, today announced the initiation of an Edgewise-funded observational trial in individuals with BMD. The trial is designed to understand the disease progression of individuals with BMD as assessed by functional measures and imaging endpoints. This global, multi-center trial is led by the GRASP (General Resolution and Assessments Solving Phenotypes) consortium and Virginia Commonwealth University (VCU), in collaboration with ImagingDMD University of Florida (UF).
The trial is expected to recruit approximately 150 individuals with BMD to collect information over a two-year period that may provide a more comprehensive understanding of the disease and help advance potential future therapies, like Edgewise’s lead clinical candidate, EDG-5506. This observational study (NCT05257473) will enroll individuals, ages 8 and older, at multiple locations across the United States and Europe. As a natural history study, it will not test any investigational drugs, but rather monitor participants over time to examine their disease course.
“The severity and progression of symptoms common in BMD, like progressive muscle weakness, vary from person to person and have posed a challenge in clinical trial design,” said Joanne Donovan, Ph.D., M.D., Chief Medical Officer, Edgewise Therapeutics. “With this study, we hope to evolve assessments of disease progression as we advance our lead clinical candidate, EDG-5506, into Phase 2 clinical trials for adolescents and adults with BMD.”
Find out more in the Edgewise Therapeutics press release.