Cystic fibrosis (CF) is the most common lethal genetic disease in the Caucasian population and is caused by the loss-of-function in the CF transmembrane conductance regulator (CFTR) chloride channel. Recent clinical trials have demonstrated the efficacy of combinations of a CFTR-potentiator with one or more CFTR-correctors. We will present the hit to lead story of a pyrazole series of compounds that are structurally distinct from current correctors. This novel CFTR-corrector has demonstrated additive potency and efficacy on top of standard of care in an <i>in-vitro</i> setting, ideal physicochemical properties, favorable <i>in-vivo</i> exposure, and displays a clean off-target profile.

Discovery of a novel second-site corrector for delF508 CFTR mutations

2019 Spring National Meeting

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