CAMBRIDGE, Mass.--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the publication of Phase 1 data on edasalonexent in adult subjects. Edasalonexent is a potential disease-modifying therapy being developed for Duchenne muscular dystrophy (DMD). The Phase 1 trials demonstrated that edasalonexent (CAT-1004), an oral inhibitor of NF-kB, was safe, well tolerated, and inhibited activated NF-kB in adult subjects and the data are presented in an article titled “A Novel NF-kB Inhibitor, Edasalonexent (CAT-1004), in Development as a Disease-Modifying Treatment for Patients with Duchenne Muscular Dystrophy: Phase 1 Safety, Pharmacokinetics, and Pharmacodynamics in Adult Subjects” in the Journal of Clinical Pharmacology (J Clin Pharmacol. 2017 Jan 11. doi: 10.1002/jcph.842.)
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 Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. “We look forward to the results from the edasalonexent Phase 2 clinical trial in boys affected by Duchenne, which are expected in the first half of Q1 2017.”
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 FDA has granted orphan drug, fast track and rare pediatric disease
designations and the European Commission has granted orphan medicinal
product designation to edasalonexent for the treatment of DMD. We have
previously reported safety, tolerability and reduction in NF-kB activity
in Phase 1 trials in adults. We are currently conducting the MoveDMD®
trial of edasalonexent in 4-7 year-old boys affected by Duchenne. From
Part A of the MoveDMD trial, we have reported that edasalonexent was
generally well tolerated with no safety signals observed and we observed
NF-kB target engagement. Pharmacokinetic results demonstrated
edasalonexent plasma exposure levels consistent with those previously
observed in adults, at which inhibition of NF-kB was observed.
About Catabasis
At Catabasis Pharmaceuticals, our mission is
to bring hope and life-changing therapies to patients and their
families. Our SMART (Safely Metabolized And Rationally Targeted) linker
drug discovery platform enables us to engineer molecules that
simultaneously modulate multiple targets in a disease. We are applying
our SMART linker platform to build an internal pipeline of product
candidates for rare diseases and plan to pursue partnerships to develop
additional product candidates. For more information on the Company's
drug discovery platform and pipeline of drug candidates, please visit www.catabasis.com.
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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 September
30, 2016, which is on file with the Securities and Exchange Commission,
and in other filings that the Company may make with the Securities and
Exchange Commission in the future. In addition, the forward-looking
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