Catabasis Pharmaceuticals Phase 1 Data on Edasalonexent (CAT-1004), a Potential Disease-Modifying Therapy Being Developed for Duchenne Muscular Dystrophy, Published in the Journal of Clinical Pharmacology
-- Edasalonexent Was Safe, Well Tolerated and Generated Positive Biomarker Results in Adult Subjects --
-- Top-Line Phase 2 Results in Boys with Duchenne Muscular Dystrophy On Track: Expected in the First Half of Q1 2017 --
In Duchenne muscular dystrophy (DMD), NF-kB is activated in muscle from infancy regardless of the underlying dystrophin mutation and drives inflammation and muscle degeneration while inhibiting muscle regeneration. Edasalonexent (CAT-1004) is a bifunctional orally administered small molecule that covalently links two compounds known to inhibit NF-kB, salicylic acid and docosahexaenoic acid (DHA). The three placebo-controlled trials in adult subjects assessed the safety, pharmacokinetics and pharmacodynamics of single or multiple edasalonexent doses up to 6000 mg (approximately 100 mg/kg). Seventy-nine adult subjects received edasalonexent and 25 received placebo. The NF-kB pathway and proteosome gene expression profiles in peripheral mononuclear cells were significantly decreased after 2 weeks of edasalonexent treatment. NF-kB activity was inhibited following a single dose of edasalonexent but not by equimolar doses of its component bioactives salicylic acid and DHA dosed in combination. Edasalonexent was well tolerated, and the most common adverse events were mild diarrhea and headache.
“These Phase 1 safety, tolerability and positive NF-kB biomarker results
support edasalonexent development in Duchenne muscular dystrophy and
potentially other diseases. The Phase 1 results in adults informed on
the dose and dose schedule for the current MoveDMD trial in 4-7 year-old
boys affected by Duchenne, where similar Phase 1 results were seen,”
About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is an oral small molecule that has the potential to be a disease-modifying therapy for all patients affected by Duchenne muscular dystrophy (DMD or Duchenne), regardless of their underlying mutation. Edasalonexent inhibits NF-kB, a protein that is activated in Duchenne and drives inflammation and fibrosis, muscle degeneration and suppresses muscle regeneration. In animal models of DMD, edasalonexent produced beneficial effects in skeletal, diaphragm and cardiac muscle and improved function. 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 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; availability and timing of results from preclinical studies and clinical trials; 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 period ended
Catabasis Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971