Results of the Phase 2 MoveDMD trial showed that Catabasis Pharmaceuticals’ candidate drug edasalonexent (CAT-1004) can reduce the rate of functional decline in boys ages 4–7 with Duchenne muscular dystrophy (DMD).
The recent findings will be subject of a presentation titled, “MoveDMD: Phase 2 Trial of Edasalonexent, an NF-kB Inhibitor, in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy,” delivered by Joanne Donovan, MD, PhD, chief medical officer at Catabasis, during the 2017 Parent Project Muscular Dystrophy (PPMD) Annual Connect Conference, June 29–July 2 in Chicago.
Edasalonexent is a potential disease–modifying therapy that was specifically designed to have a broader effect instead of targeting mutation–specific mechanisms that can only be used in a subset of patients. This investigational drug has the potential to treat patients with DMD independently of the genetic variation responsible for their condition.
It is an inhibitor of a protein called NF-kB that is activated in DMD. Preclinical studies demonstrated that edasalonexent could reduce muscle degeneration and promote muscle regeneration and function.
Currently, the investigational therapy is being evaluated in the three–part Phase 1/2 MoveDMD trial (NCT02439216) to assess its efficacy and safety in boys with DMD, ages 4 to 7. The study has included 31 boys not treated with corticosteroids who received edasalonexent orally or a placebo control.
In Part B of the trial, treatment with edasalonexent was found to improve by approximately 80% the results of a 10–meter walk/run speed test compared to the placebo group. It also improved the speed it took the children to climb a flight of stairs, and improved the Pediatric Outcomes Data Collection Instrument (PODCI) score.
Evaluation of a composite functional score showed that those treated with the candidate drug had improved function compared to the placebo group. Overall, edasalonexent was found to slow the rate of functional decline of these patients, independently of the genetic background.
Researchers also compared the effects of edasalonexent treatment with the symptoms of patients during the off–treatment period (crossover analysis) in 12 boys who participated in Part A and Part B of the trial. Results of this evaluation were also positive, demonstrating a global benefit of the treatment by improving patients’ functional activity in all tested parameters.
The treatment was found to be well–tolerated and safe.
Researchers currently are conducting Part C of the trial to assess long–term effects of the treatment. Results of this open–label extension of the MoveDMD study are expected by the end of 2017.
These positive results will support further clinical development of edasalonexent with a Phase 3 trial, which the company expects to announce in the second half of 2017.
Previous results had shown that Part A and Part B of the trial led to no statistical improvement in lower leg muscle function by magnetic resonance imaging (MRI) after 12 weeks. That was the trial’s primary efficacy endpoint. In an exclusive interview, we asked Jill C. Milne, PhD, co-founder and chief executive officer of Catabasis Pharmaceuticals, how these results affected study goals. That interview can be read ici.
Catabasis’ candidate drug has been granted orphan drug, fast track, et rare pediatric disease designations from the U.S. Food and Drug Administration, as well as orphan medicinal product designation by the European Commission.