Catabasis announced their phase 1/2 trial testing safety and efficacy of edasalonexent (CAT-1004) for the treatment of Duchenne muscular dystrophy (DMD) failed to reach its primary endpoint.
During a conference call, the company showed that while the study did not meet its primary endpoint [magnetic resonance imaging (MRI) T2 composite measure of lower leg muscles after 12 weeks of treatment] the patients in the higher dose of edasalonext did show non-significant improvements in several secondary outcome measures. Data presented during the call are shown below.
At this time, the company plans to continue assessing the data and also plans to continue with its open label extension of the study.
In a press release, Jill C. Milne, Chief Executive Officer of Catabasis said, “Although we did not meet the MRI T2 composite end point, the continued safety, tolerability and plasma exposure data in Part B of the MoveDMD trial are reassuring. We observed potential treatment-associated effects at 12 weeks in the 100 mg/kg/day treatment group, which we believe warrant further evaluation to see if the signals strengthen in the longer-term data from the ongoing open-label extension. Following additional data analysis from the open-label extension, we will determine the next steps for edasalonexent in DMD.”
Richard Finkel, M.D., Division Chief, Division of Neurology, Department of Pediatrics at Nemours Children’s Health System and a Principal Investigator for the study added,“The top-line data results from Part B of the MoveDMD trial provide us with an early snapshot of the effects of edasalonexent in boys with DMD over a 12-week period. Continuing the open-label extension of the MoveDMD trial will allow us to further evaluate the potential for edasalonexent to provide benefit in DMD.”
About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is an investigational oral small molecule that is being developed as a potential disease-modifying therapy for all patients affected by Duchenne muscular dystrophy (DMD or Duchenne), regardless of their underlying mutation.
Edasalonexent inhibits NF-kB, a protein that is activated in Duchenne and drives inflammation and fibrosis, muscle degeneration and suppresses muscle regeneration.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a progressive muscle disorder caused by the lack of functional dystrophin protein. Patients with Duchenne muscular dystrophy lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties.
There are an estimated 35,000 patients with Duchenne in the United States and Europe but the population has many subsets based on mutations of the dystrophin gene.