ETX001

ETX001 is an inhaled, low–molecular-weight small molecule potentiator of the calcium-activated chloride channel TMEM16A (also known as anoctamin-1), developed by Enterprise Therapeutics for the treatment of cystic fibrosis and other muco-obstructive lung diseases.[1][3][6] By enhancing TMEM16A channel opening at physiological intracellular calcium levels without altering calcium signaling, ETX001 increases epithelial anion (chloride) secretion, elevates airway surface liquid height, and improves mucus clearance independently of CFTR function, offering a potential therapy for all CF genotypes and for non-CF mucoobstructive diseases.[2][3][5] In preclinical human bronchial epithelial cell systems and ovine models, inhaled ETX001 produced robust, concentration-dependent increases in airway fluid secretion and dose-dependent acceleration of mucociliary clearance, supporting its development as an inhaled therapy targeting mucus obstruction in the lung.[1][3][6]