Catabasis’ chief medical officer, Dr. Joanne Donovan, will present results from the first phase of the two-part MoveDMD Trial, exploring the company’s investigational drug edasalonexent (CAT-1004) in patients with Duchenne muscular dystrophy (DMD). The presentation will take place at the 2016 Parent Project Muscular Dystrophy (PPMD) Annual Connect Conference, set for June 26–29 in Orlando, Florida.
Both an oral presentation, “Edasalonexent (CAT-1004), an Oral Agent Targeting NF-kB: MoveDMD Trial in Duchenne Muscular Dystrophy (DMD),” as well as a poster presentation are planned.
Unlike many therapies under development for Duchenne today, edasalonexent is designed to treat all patients independent of their specific mutation. This is possible because the drug works by blocking a key inflammatory factor, NF-κB (nuclear factor kappa B), which acts as a so-called transcription factor (binding to genes to activate their transcription).
NF-κB is a known activator of a multitude of genes involved in inflammatory and fibrotic processes, and drives muscle degeneration in Duchenne. Data from animal studies showed that the drug effectively improved regeneration and function of skeletal, heart, and diaphragm muscle.
A previous Phase 1 study, investigating the safety and tolerability of the drug in adults, as well as data from the completed Part A of the MoveDMD trial, demonstrated the drug to be safe, and that it blocked NF-κB.
MoveDMD is a Phase1/2 trial in boys, ages 4 to 7, with Duchenne MD. Participants in Part A were invited to continue in the second part of the trial — a 12-week, placebo-controlled and multiple-dose study, now getting underway, focusing on the drug’s disease modifying effects as well as its safety, tolerability and pharmacokinetics. After 12 weeks of treatment, patients in the placebo group will have the option of receiving 12 weeks of edasalonexent treatment.
This second part will enroll about 30 patients at five sites in the U.S., who will be evaluated both by functional tests and magnetic resonance imaging of the leg muscles. Part B of the MoveDMD trial is also recruiting new patients. More information is available on its clinical trials.gov site (NCT02439216).
Edasalonexent has been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA). This status is reserved for potential therapies for rare diseases and unmet needs, and aim to speed the development and review of these drugs. The European Commission has likewise designated edasalonexent an orphan medicinal product for the treatment of DMD.