Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today shared information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) in poster presentations at the 2020 Muscular Dystrophy Association (MDA) Virtual Poster Session. The three posters include an analysis of the baseline characteristics of patients enrolled in the Phase 3 PolarisDMD trial, age-normative growth and normal adrenal function seen in the Phase 2 MoveDMD trial and open-label extension, and experience with capsule swallowing in both clinical trials for edasalonexent in boys with DMD. Top-line results from the Phase 3 trial are expected in Q4 2020.
“As we advance our Phase 3 PolarisDMD trial, we gain valuable insights into the full potential of edasalonexent,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. “We are pleased to share additional information supporting the design of the Phase 3 PolarisDMD trial and showing long-term safety of edasalonexent. In addition to being well-tolerated in over 100 cumulative years of patient exposure, edasalonexent has shown potential to both preserve muscle function as well as positive cardiac effects. Those living with Duchenne have limited treatment options, and our goal is for edasalonexent to be a new foundational therapy.”
An analysis of the baseline characteristics of the patients enrolled in the Phase 3 PolarisDMD trial was performed compared to the patients enrolled in the previous Phase 2 MoveDMD trial and found overall similar baseline characteristics in the patient populations in the two trials. Both the Phase 3 PolarisDMD trial and the Phase 2 MoveDMD trial enrolled boys affected by DMD ages 4 to 7 (up to 8th birthday) with any mutation type who had not been on steroids for the previous 6 months. There were no significant differences between the two trials in baseline age, North Star Ambulatory Assessment (NSAA) score and timed function test values (time to stand, 4-stair climb, and 10-meter walk/run). These findings are believed to support the assumptions on which the Phase 3 trial was powered.
Catabasis also shared long-term safety and tolerability data from the MoveDMD trial and open-label extension. Edasalonexent was associated with age-normative growth, without negative impact on bone health, and normal adrenal function in boys with DMD as it does not impact the glucocorticoid receptor. There was no evidence of adrenal insufficiency for up to 150 weeks of edasalonexent treatment, and no clinically significant changes in cortisol or ACTH. Additionally, edasalonexent treatment in a mdx mouse model of DMD preserved bone length and bone density. In more than 100 years of cumulative patient exposure, edasalonexent has been well-tolerated, with no serious adverse events on treatment, and without the adverse effects associated with high-dose steroids. In both the Phase 2 MoveDMD trial and the ongoing Phase 3 PolarisDMD trial, 97% of 4 to 7 year-old boys with DMD screened for the studies were able to swallow soft-gel study drug capsules. There have been no discontinuations due to capsule burden. This demonstrates that edasalonexent has the potential to be broadly adopted by those affected by DMD and supports the long-term safety and tolerability of edasalonexent.
Posters from the MDA Virtual Poster Session are available under “Our Science” at www.catabasis.com.
About Edasalonexent (CAT-1004)