This exploratory Phase 2 trial, which is subject to the receipt of adequate funding, is designed to assess safety, pharmacokinetics and exploratory measures of function including cardiac, skeletal muscle and pulmonary function in non-ambulatory DMD patients.
“We are thrilled to announce plans to expand our knowledge of edasalonexent to non-ambulatory boys and men affected by Duchenne. We recognize the urgent need for a well-tolerated treatment like edasalonexent with the potential to slow disease progression and preserve muscle function by benefitting both skeletal muscle as well as cardiac function,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. “We are incredibly fortunate to have the opportunity to partner with Duchenne UK for this important work and appreciate their deep commitment as we work together to bring treatment options to all patients.”
“We first approached Catabasis last year to ask if we could encourage them to advance a trial to look at the non-ambulant patient population and we are delighted to be able to announce this collaboration today,” said Emily Crossley and Alex Johnson, Duchenne UK cofounders. “Duchenne UK is committed to developing medicines for all boys and men with DMD, regardless of their physical stage, mutation or age. This trial will represent an important step in that direction.”
The Phase 2 trial is designed to be a one-year, randomized, double-blind, placebo-controlled trial evaluating safety, pharmacokinetics and exploratory measures of function with edasalonexent in non-ambulatory boys and men affected by DMD. The trial expects to enroll approximately 16 non-ambulatory patients ages 10 and older regardless of mutation type who have not been on steroids for at least 6 months at clinical trial sites in the United Kingdom. The exploratory functional endpoints are anticipated to include assessments of cardiac function, upper limb skeletal muscle function and pulmonary function. In addition, the trial is also expected to explore patient reported outcomes. The intention is that upon completing this trial, patients will have the option to transition to the GalaxyDMD open-label extension trial and receive edasalonexent.
Source: Company Press Release