Promising News from a Clinical Study in Becker Muscular Dystrophy

Jan 9

Becker is a rare genetic condition that weakens and damages muscles over time. Like Duchenne’s, it mostly affects males and causes big challenges for the diagnosed person and their family, including physical, emotional, and financial struggles. With Becker, muscles get damaged when they move, leading to muscle loss and difficulty with movement. Muscle loss can start at any age, and, once it happens, it cannot be fixed and worsens over time.

Recognizing the need for an effective way to treat Becker, researchers have been studying the cause of muscle loss, and ways to stop it from happening. Know Rare has been active in advocating for this type of research and helping people with Becker, who are interested in participating, to connect to researchers.

Craig M. McDonald, M.D., Distinguished Professor and Chair at the UC Davis Health Department of Physical Medicine and Rehabilitation, the principal researcher in the CANYON and GRAND CANYON studies, is hopeful of the possibility of an effective new treatment for Becker, sevastemten.

Here’s a quick summary of the study’s early results:

The study’s measures of muscle loss showed a decrease among those people who were taking the active study medication as compared to those taking placebo (a similar-looking sugar pill without an active ingredient).
Besides a reduction in levels of creatine kinase (CK), the measure of muscle damage, the study showed the sevastemten-treated participants had maintained their ability to move, measured by the North Star Ambulatory Assessment (NSAA) and showed signs of improvement after 12 months compared to those who received a placebo.
Those taking the sevastemten did not have serious safety concerns.

Overview of CANYON and GRAND CANYON Studies

Forty adults and twenty-nine adolescents with Becker muscular dystrophy were enrolled in CANYON, the largest interventional Becker trial. CANYON is a Phase 2 clinical study that investigated the effect of sevasemten on the safety and functional measures of participants (NCT05291091).

Around 120 individuals with Becker were enrolled in GRAND CANYON, an expansion of the CANYON, which will continue to assess the safety and efficacy of sevasemten in adults with Becker. The main goal of the GRAND CANYON study is to demonstrate an improved ability to move (as measured by the NSAA) after 18 months.

If the data is positive from the larger GRAND CANYON study, it could pave the way to being approved by the FDA and European authorities as a new treatment for Becker.

About Sevastemten, the Studied Medication

Sevasemten is a unique new oral drug designed to protect muscle against contraction-induced muscle damage in muscular dystrophies including Becker and Duchenne. Besides being studied in the Phase 2 CANYON and the GRAND CANYON studies in adults and adolescents with Becker muscular dystrophy, sevasemten is also being studied in the ongoing Phase 2 trials, LYNX and FOX, in children and adolescents with Duchenne muscular dystrophy.

Know Rare was able to help connect a large number of people with Becker to multiple study centers around the country to find out about participating in the GRAND CANYON Study. It is gratifying to see that the study has shown positive results.

There is no cure for Becker right now, so more research to discover effective treatments is urgently needed, and cheers to anyone who reached out to Know Rare to find out about the study, and may have been one of the participants.

About Edgewise Therapeutics, the Study’s Sponsors

Edgewise Therapeutics is a leading muscle disease biopharmaceutical company developing novel therapeutics for muscular dystrophies and serious cardiac conditions. The Company’s deep expertise in muscle physiology is driving a new generation of novel therapeutics. Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor in late-stage clinical trials in Becker and Duchenne muscular dystrophies. EDG-7500 is a novel cardiac sarcomere modulator for the treatment of hypertrophic cardiomyopathy and other diseases of diastolic dysfunction, currently in Phase 2 clinical development. The entire team at Edgewise is dedicated to our mission: changing the lives of patients and families affected by serious muscle diseases. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn, X, Facebook, and Instagram.

Stay in touch for the latest research and resources: