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,”
said
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
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Source:
Catabasis Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com