elexacaftor

Elexacaftor (VX-445) is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector. It promotes the processing and trafficking of CFTR, increases the amount of CFTR on the cell surface, and improves the processing and trafficking of Phe508del CFTR protein.

(R)-Elexacaftor (compound 37) is the enantiomer of Elexacaftor (compound 1). (R)-Elexacaftor is a CFTR modulator with an EC50 of 0.29 μM for CFTR dF508.

I1421 is an activator of the cystic fibrosis transmembrane conductance regulator (CFTR), with an EC50 of 64 nM for WTCFTR currents. It also allosterically activates various CF-causing mutants, demonstrating good in vivo efficacy with an oral bioavailability of 60% in mice and a corresponding half-life of 75 minutes. I1421 works synergistically with Elexacaftor to enhance CFTR currents.