Two phase III trials of VX-659, tezacaftor + ivacaftor meet primary endpoint in cystic fibrosis.- Vertex Pharma.

Vertex Pharmaceuticals announced that treatment with the triple combination of the next-generation corrector VX-659, tezacaftor and ivacaftor resulted in statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in two Phase III studies in people with cystic fibrosis (CF). Data from a pre-specified interim analysis of the Phase III study in people with one F508del mutation and one minimal function mutation showed a mean absolute improvement in ppFEV1 of 14.0 percentage points from baseline at week 4 of treatment compared to placebo.

In the Phase III study in people with two F508del mutations, the addition of VX-659 in patients already receiving tezacaftor and ivacaftor resulted in a mean absolute improvement in ppFEV1 of 10.0 percentage points from baseline at week 4 of treatment compared to the control group in whom placebo was added to tezacaftor and ivacaftor. The VX-659 triple combination regimen was generally well tolerated, and the safety and efficacy profile from the results support the potential submission of a New Drug Application (NDA) for the VX-659 triple combination regimen.