Phase 2 clinical trial, CANYON, shows potential at reducing muscle damage in Becker muscular dystrophy

18 December 2024

Sevasemten, a potential treatment for Becker muscular dystrophy, reduced levels of a marker of muscle damage in the CANYON clinical trial.

The phase 2 CANYON clinical trial tested sevasemten in 40 adults and 29 adolescents with Becker muscular dystrophy (BMD). Sevasemten is designed to reduce the activity of myosin, a protein which plays a key role in muscle contraction. People living with BMD are missing fully functional dystrophin, which normally acts as a shock absorber. The loss of dystrophin means there is less shock absorber when muscles contract. This can then lead to muscle damage. By reducing the activity of myosin, the muscle contractions should be gentler and reduce damage to muscles.

When analysing the data from the adults, sevasemten was found to reduce levels of a marker of muscle damage compared to those who received the placebo (dummy drug). This suggests sevasemten is working as designed in the body. The drug was also found to be safe and showed hints that it may be improving function. However, more data is needed to confirm if the drug is beneficial for people with BMD.

A larger phase 2 trial is now ongoing, called GRAND CANYON. This trial will gather further data on how the drug works in people with BMD. The company has also said they will continue to analyse the results from the trial and will start discussions with regulatory agencies. We will continue to update you on the developments of sevasemten.

Read the press release for more information.