ICENTICAFTOR

Icenticaftor is a probe of excellent quality for both in vitro and in vivo work. As described in its original publication, it was documented to be selective for the potentiation of CFTR against a broad panel of 46 G-protein coupled receptor, enzyme, transporter, and ion channels (including hERG) as well as 20 kinase targets with no observed inhibition >50% at 10 μM concentration. Additionally, it displays good cellular activity with IC50 values well below 1 μM using media containing up to 10% FBS.

Icenticaftor is a high quality potentiator of both wild-type and delF508 CFTR activity. Based on profiling experiments, it appears to have high selectivity for CFTR over different GPCRs, transporters, ion channels as well as a number of kinases. Icenticaftor is well tolerated in vivo in rat, dog and monkey experiments and has shown target engagement and clinical improvement in cystic fibrosis and COPD trials.