Edgewise Therapeutics, Inc. has announced an expansion of their clinical development program of EDG-5506. EDG-5506, which comes in the form of a small pill, is an investigational product that aims to protect muscles, specifically fast-twitch skeletal muscles, from injuries that can happen during muscle contractions and relaxations.
Edgewise has announced a new trial called FOX, which will be a Phase 2 study focused on children and adolescent boys aged 6-14 years who have Duchenne and have previously received gene therapy. In this 12-week placebo-controlled trial, they will be checking to make sure that EDG-5506 is safe to use, how it moves through the body (pharmacokinetics), and how it affects certain markers related to muscle health. The study will also look at how well the treatment improves physical abilities, like the North Star Ambulatory Assessment (NSAA), and how it makes patients and caregivers feel.
After the 12-week study, all the participants will get the chance to use EDG-5506 in an open-label program. This means all participants will have access to EDG-5506 even if they were on placebo during the initial 12 weeks.
Edgewise is also trying to understand the impact of EDG-5506 on individuals with Duchenne who haven’t used corticosteroids. They’re doing this in their Phase 2 placebo-controlled LYNX trial for children aged 4-9 years. The goal of the LYNX trial is to figure out the right amount of EDG-5506 that can reduce muscle damage markers and help patients feel better in a Phase 3 trial.
In the near future, you can expect to hear some interim results from the LYNX Phase 2 trial, likely in the first half of 2024. Edgewise also intends to start a Phase 3 trial in Duchenne with the dose they’ve found to be most effective in 2024.
PPMD appreciates this opportunity to open studies of EDG-5506 to broader groups of individuals with Duchenne and we look forward to hearing more from Edgewise about these trials and their progress.
Read Edgewise’s press release here.