Catabasis Pharmaceuticals Inc, a clinical-stage biopharmaceutical company, has collaborated with Duchenne UK, a charity that seeks to fund and accelerate treatments and a cure for Duchenne muscular dystrophy (DMD), for a phase two trial of edasalonexent, a novel NF-kB inhibitor, in non-ambulatory DMD patients, it was reported yesterday.

The exploratory phase two trial is subject to the receipt of adequate funding and is designed to evaluate safety, pharmacokinetics and exploratory measures of function including cardiac, skeletal muscle and pulmonary function in non-ambulatory DMD patients.

The Phase 2 trial is proposed to be a one-year, randomised, double-blind, placebo-controlled trial assessing safety, pharmacokinetics and exploratory measures of function with edasalonexent in non-ambulatory boys and men affected by DMD. The trial is likely to enrol around 16 non-ambulatory patients ages 10 and older regardless of mutation type who have not been on steroids for around six months at clinical trial sites in the United Kingdom. The exploratory functional endpoints are expected to include evaluations of cardiac function, upper limb skeletal muscle function and pulmonary function. The trial is also likely to explore patient reported results. It is intended that upon completing this trial, patients will have the option to transition to the GalaxyDMD open-label extension trial and receive edasalonexent.