Phase 1 study of edasalonexent (CAT-1004), an oral NF-κ B inhibitor, in pediatric patients with duchenne muscular dystrophy

Erika Finanger, Krista Vandenborne, Richard S. Finkel, H. Lee Sweeney, Gihan Tennekoon, Sabrina Yum, Maria Mancini, Pradeep Bista, Andrew Nichols, Hanlan Liu, Angelika Fretzen, Joanne M. Donovan

Research output: Contribution to journalArticlepeer-review

37 Scopus citations


Background: Edasalonexent is an orally administered small molecule designed to inhibit NF-κB, which is activated from infancy in Duchenne muscular dystrophy and is central to causing muscle damage and preventing muscle regeneration. Objective: Evaluate the safety, tolerability, pharmacokinetics and exploratory pharmacodynamics of three doses of edasalonexent in ambulatory males ≥4 to <8 years of age with genetically confirmed Duchenne muscular dystrophy. Methods: This was a 1-week, open-label, multiple-dose study with 3 sequential ascending doses (33, 67 and 100mg/kg/day) of edasalonexent administered under different dietary conditions to 17 males with a mean age of 5.5 years. Results: All doses of edasalonexent were well tolerated, with no serious adverse events, no drug discontinuations and no dose reductions. The majority of adverse events were mild, and the most common adverse events were gastrointestinal (primarily diarrhea). Edasalonexent was rapidly absorbed with peak levels observed 2-6 hours after dosing and exposures appeared to increase nearly proportionally to dose for the 2 lower and all 3 doses under low-fat and high-fat meal conditions, respectively. Only minor plasma accumulation of edasalonexent was observed with 7 days of dosing. After treatment with edasalonexent for 7 days, levels of NF-κB-regulated genes and serum proteins were decreased. Conclusions: This first report of edasalonexent oral administration for one week in male pediatric patients with Duchenne muscular dystrophy showed that treatment was well tolerated and inhibited NF-kB pathways.

Original languageEnglish (US)
Pages (from-to)43-54
Number of pages12
JournalJournal of Neuromuscular Diseases
Issue number1
StatePublished - 2019


  • CAT-1004
  • Duchenne muscular dystrophy
  • NF-κ B
  • edasalonexent

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology


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