Catabasis Pharmaceuticals Inc. has announced plans for a Phase 3 clinical trial called Polaris DMD. It will investigate the experimental drug edasalonexent for the treatment of patients with Duchenne muscular dystrophy.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is one of the most common types of muscular dystrophy, a group of conditions that cause muscle weakness.
DMD is a life-shortening condition that usually affects boys, and people who have it typically begin to show symptoms between the ages of one to three years old. The early symptoms may include difficulty with certain movements, such as running, jumping, standing up, or climbing the stairs. Children may also show learning difficulties, behavioural difficulties, or delayed speech. As DMD progresses, children are likely to need mobility assistance, such as a wheelchair, and can develop other symptoms such as heart conditions and breathing problems.
According to Muscular Dystrophy UK, around one in every 3,500 to 5,000 male newborns are born with DMD.
About Edasalonexent (CAT-1004)
Edasalonexent is an investigational drug being developed as a potential treatment for DMD. It is designed to be effective for DMD caused by any genetic alteration. The drug works by inhibiting the NF-kB protein, which is involved in inflammation, muscle degeneration, and fibrosis.
So far, it has been granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations by the FDA, and Orphan Medicine Product designation by the European Commission.
It is being tested in a Phase 2 clinical trial, and Catabasis is planning to further evaluate the drug as a potential treatment for DMD in a Phase 3 clinical trial later this year.
The Phase 3 Clinical Trial (Polaris DMD)
The trial is expected to begin in the second half of 2018, and top-line results are predicted to be released in the second quarter of 2020.
The study was designed with input from the FDA, physicians, and families affected by DMD. It will be a randomised and double-blind trial that compares the effects of edasalonexent to those of a placebo. The researchers are aiming to enroll around 125 patients aged between 4 to 7.
For more information about the study, what it will evaluate, and who will be eligible, click here to read the original article.
