C-10355

C-10355 is a deuterated analog of ivacaftor, a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) potentiator, developed by Concert Pharmaceuticals. Utilizing Concert's proprietary deuterium chemical entity (DCE) platform, C-10355 was designed to enhance the pharmacokinetic properties of ivacaftor, specifically to reduce metabolic clearance and potentially allow for once-daily oral dosing. In a Phase 1 clinical study (NCT02445443), C-10355 was evaluated in healthy volunteers alongside another deuterated candidate, C-10358, and compared to the non-deuterated parent drug, Kalydeco (ivacaftor). Although C-10355 demonstrated the intended mechanism of action by increasing the open-channel probability of CFTR, Concert Pharmaceuticals ultimately prioritized a different deuterated ivacaftor analog, CTP-656 (also known as VX-561 or posenacaftor), for further clinical development. The cystic fibrosis program was subsequently acquired by Vertex Pharmaceuticals in 2017.

Other names

deuterated ivacaftor