Catabasis Pharmaceuticals Presents Information on Edasalonexent, a Potential Foundational Therapy for Duchenne Muscular Dystrophy, at the Virtual 25th International Congress of the World Muscle Society
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today shared information on the edasalonexent program in Phase 3 development for Duchenne muscular dystrophy (DMD) in poster presentations at the Virtual 25th International Congress of the World Muscle Society. The posters include new preclinical research findings supporting potential positive effects on cardiac function and preservation of bone health with edasalonexent in mouse models of DMD. Information from clinical trials with edasalonexent was also presented with baseline assessments from the Phase 3 PolarisDMD trial and long-term safety results from the Phase 2 MoveDMD trial and open-label extension.
“As we approach top-line Phase 3 PolarisDMD results, which we expect to report in the fourth quarter of this year, we are pleased to share new findings from preclinical studies with edasalonexent that could support its potential as a foundational therapy for those affected by Duchenne,” said Andrew Nichols, Ph.D., Chief Scientific Officer of Catabasis. “Cardiac function and bone health are critical components of Duchenne care and we are excited to learn more about these key areas that are important to patients, their caregivers and physicians.”
Persistent activation of NF-kB in DMD can drive cardiac dysfunction, which is the leading cause of mortality in DMD. In a preclinical study performed in the laboratory of Pradeep Mammen, M.D., Medical Director of the Neuromuscular Cardiomyopathy Clinic and Director of Translational Research for the Advanced Heart Failure and Transplant Cardiology Program at UT Southwestern, edasalonexent prevented the development of DMD-associated cardiomyopathy in the mdx:Utrn+/- mouse model of DMD. Edasalonexent reduced the cardiac hypertrophy apparent in these mice, reduced myocardial fibrosis and prevented the development of DMD-associated cardiomyopathy.
In DMD, NF-kB activation also drives inflammation and fibrosis, leading to loss of skeletal muscle function and disease progression. Reduced skeletal muscle function results in reduced bone strength. Steroids, which activate the glucocorticoid receptor (GR), can further negatively impact bone health. In a preclinical study led by Frank Rauch, M.D., of Shriners Hospitals for Children - Canada, edasalonexent was seen to maintain bone density and bone strength in mdx mice. Consistent with these results, edasalonexent treatment in cells inhibited NF-kB and as expected did not impact the GR, while the steroid prednisolone strongly activated the GR.