BACKGROUND: Edasalonexent (CAT-1004) is an orally-administered novel small 
molecule drug designed to inhibit NF-κB and potentially reduce inflammation and 
fibrosis to improve muscle function and thereby slow disease progression and 
muscle decline in Duchenne muscular dystrophy (DMD).
OBJECTIVE: This international, randomized 2 : 1, placebo-controlled, phase 3 
study in patients ≥4 - < 8 years old with DMD due to any dystrophin mutation 
examined the effect of edasalonexent (100 mg/kg/day) compared to placebo over 52 
weeks.
METHODS: Endpoints were changes in the North Star Ambulatory Assessment (NSAA; 
primary) and timed function tests (TFTs; secondary). Assessment of 
health-related function used the Pediatric Outcomes Data Collection tool 
(PODCI).
RESULTS: One hundred thirty one patients received edasalonexent (n = 88) and 
placebo (n = 43). At week 52, differences between edasalonexent and placebo for 
NSAA total score and TFTs were not statistically significant, although there 
were consistently less functional declines in the edasalonexent group. A 
pre-specified analysis by age demonstrated that younger patients (≤6.0 years) 
showed more robust and statistically significant differences between 
edasalonexent and placebo for some assessments. Treatment was well-tolerated and 
the majority of adverse events were mild, and most commonly involved the 
gastrointestinal system (primarily diarrhea).
CONCLUSIONS: Edasalonexent was generally well-tolerated with a manageable safety 
profile at the dose of 100 mg/kg/day. Although edasalonexent did not achieve 
statistical significance for improvement in primary and secondary functional 
endpoints for assessment of DMD, subgroup analysis suggested that edasalonexent 
may slow disease progression if initiated before 6 years of age. (NCT03703882).