Lead Clinical Candidate
Our lead product candidate, cavosonstat (N91115), is an investigational small molecule that has the potential to address a defect in CFTR, resulting from mutations in the CFTR gene, the underlying cause of CF. We believe cavosonstat is a first-in-class CFTR stabilizer that modulates CFTR activity through a novel mechanism of action that we expect to be complementary to existing and future CFTR modulators.
Cavosonstat is the only clinical stage product candidate we know of designed to stabilize CFTR inside the cell and at the cell surface. We have shown in preclinical studies that the stabilizing effect of cavosonstat increases and prolongs CFTR activity when added to other CFTR modulators. Currently, we are evaluating the safety and efficacy of cavosonstat in a Phase 2 clinical trial with cavosonstat used with Kalydeco in patients heterozygous for F508del and a gating mutation.