UPDATE 06/07/2023 - Edgewise Therapeutics has announced the results from a study evaluating their EDG-5506 drug in adults with Becker muscular dystrophy (BMD). The Phase 1b study called ARCH assessed the safety, tolerability, and impact on muscle damage
The use of EDG-5506 showed a significant reduction in key biomarkers of muscle damage in the research participants. Functional improvements were also observed in nine out of twelve participants. These results support the potential for EDG-5506 to preserve muscle function and slow progression of BMD. The results have also informed the design of a potential registrational trial – a pivotal trial that aims to provide data and evidence to support the registration and approval of a new treatment by regulatory authorities.
EDG-5506 is an investigational drug designed to protect the muscle. It works to inhibit (or stop) a particular form of a protein called myosin found in skeletal muscle fibres. Myosin produces energy for muscle contraction – a little bit like a motor. In people living with Becker muscular dystrophy this muscle contraction could eventually lead to muscle damage as they are missing fully functional dystrophin, which acts as a kind of shock absorber. The idea is that EDG-5506 stops myosin from producing the energy required to contract the muscle, therefore reducing the stress and damage to the muscle.
Enrolment onto the trial has started at clinical sites across the United States, United Kingdom and the Netherlands. In the UK, the trial is taking place in London (University College London Hospital) and Newcastle (Newcastle Freeman Hospital). The aim is to include 48 adult and 18 adolescent participants with Becker muscular dystrophy across these countries.
People with Becker muscular dystrophy interested in participating in the Canyon clinical trial should discuss it with their neuromuscular consultant.
Further information about the Canyon trial, including eligibility, inclusion and exclusion criteria, can be found here. For any additional information, people are encouraged to contact the study coordinators at studies@edgewisetx.com