Catabasis Pharmaceuticals Announces Collaboration with the University of Texas Southwestern to Explore the Potential Benefits of Edasalonexent on Cardiac Function in Duchenne and Becker Muscular Dystrophies
-- One-Year Preclinical Study of Edasalonexent, a Novel Inhibitor of NF-kB, in Animal Models of Duchenne to Investigate Potential Improved Cardiac Function --
Cardiomyopathy is the leading cause of mortality in Duchenne and Becker muscular dystrophies. Young boys with Duchenne typically have an elevated heart rate that exceeds the normal resting rate for age, which is the first cardiac manifestation in boys with DMD. Preclinical and clinical biomarker data support the potential for cardiac benefits with edasalonexent in Duchenne and Becker muscular dystrophies.
The one-year collaboration between Catabasis and Dr. Mammen will utilize the muscular dystrophy mdx mouse model with reduced utrophin as these animals display an early and prominent cardiomyopathy. The study will evaluate both functional and echocardiographic assessments of the heart as well as histological, biochemical and molecular assessments to study the potential benefits of edasalonexent treatment. Results are expected in the second half of 2019.
“Inhibiting NF-kB with edasalonexent offers a unique mechanism with the
advantage of potentially impacting both skeletal and cardiac muscle
disease in those living with Duchenne and Becker muscular dystrophies,”
Dr. Mammen is an expert in advanced heart failure, with a focus on
neuromuscular cardiomyopathy, and is board certified in advanced heart
failure/VAD/transplant cardiology. As a treating physician at the
“There is significant unmet medical need for therapies that could treat
both the skeletal and cardiac muscle disease in Duchenne and Becker
muscular dystrophies,” said
The collaboration builds upon preclinical and clinical biomarker data supporting the potential for cardiac benefits with edasalonexent. In the Catabasis Phase 2 MoveDMD clinical trial and open-label extension, significantly decreased heart rate towards age-normative values was observed in boys with DMD. Preclinical data in mdx mice and GRMD dogs, animal models of Duchenne, have shown substantially decreased cardiac fibrosis with NF-kB inhibition.
Edasalonexent is currently being studied as a potential treatment for Duchenne muscular dystrophy in the Phase 3 PolarisDMD clinical trial, a one-year, randomized, double-blind, placebo-controlled trial. Catabasis plans to enroll approximately 125 patients in the trial ages 4 to 7 (up to 8th birthday) regardless of mutation type who have not been on steroids for at least 6 months. In the clinic, edasalonexent has been shown to preserve muscle function and substantially slow DMD disease progression across all four assessments of muscle function (the North Star Ambulatory Assessment, time to stand, 4-stair climb, and 10-meter walk/run) compared to control. Edasalonexent has been well tolerated through more than 50 patient-years of treatment with no safety signals observed.
About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is an investigational oral small molecule that is being developed as a potential new standard of care for all patients affected by DMD, regardless of their underlying mutation. Edasalonexent inhibits NF-kB, which is a key link between loss of dystrophin and disease progression in DMD. NF-kB has a fundamental role in skeletal and cardiac muscle disease in DMD. Catabasis is currently enrolling the single global Phase 3 PolarisDMD trial to evaluate the efficacy and safety of edasalonexent for registration purposes. In the clinic, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. Edasalonexent continues to be dosed in the open-label extension of the MoveDMD Phase 2 clinical trial. The
Forward Looking Statements
Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company’s global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, and other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “may” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company’s product candidates; and general economic and market conditions and other factors discussed in the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the quarter ended
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