ETD001

ETD001 is a novel, inhaled small molecule drug that acts as a potent and long-acting inhibitor of the epithelial sodium channel (ENaC) in the airway epithelium. By blocking ENaC, ETD001 increases airway surface hydration, thereby enhancing mucociliary clearance and reducing mucus congestion in the lungs. This mechanism is particularly relevant for people with cystic fibrosis (CF), where thick, sticky mucus leads to recurrent infections and progressive lung damage. Unlike CFTR modulators, ENaC inhibition by ETD001 is independent of CFTR mutation status, making it potentially beneficial for all individuals with CF regardless of genotype. The drug has demonstrated an extended duration of action (≥16 hours) at well-tolerated doses in Phase 1 studies and is currently being evaluated in Phase 2 clinical trials for its efficacy and safety in people with cystic fibrosis[2][3][4][5][6][8].

Other names

3-amino-n-{[5-[4-[bis[(2S,3R,4R,5R)-2,3,4,5,6-pentahydroxyhexyl]amino]piperidine-1-carbonyl]-1,3-diethyl-benzimidazol-1-ium-2-yl]methyl}-5h-pyrrolo[2,3-b]pyrazine-2-carboxamide dihydrochloride chloride