INO-4995 (1-O-octyl-2-O-butyryl-D-myo-inositol 3,4,5,6-tetrakisphosphate octakis(propionoxymethyl)ester) is an ion channel modulator that is slowly metabolized in cells. Studies have shown that it inhibits ENaC activity while promoting expression of the calcium-activated chloride channel, TMEM16a, in membranes. Thus it has potential therapeutic activity in the treatment of cystic fibrosis where it normalized nasal potential difference (npd) in a murine CF knock-out model and promoted fluid secretion in human CF airway epithelia.
Strong preclinical efficacy of INO-4995: a membrane-permeant Inositol-phosphate derivative has potential in treatment of Cystic Fibrosis (CF)
This graph is showing the difference in npd between mice before and after 4-day treatment with INO-4995 (0.0008mg/kg):
Am J Respir Cell Mol Biol 2010, 42(1), 105