Vertex Pharma initiates Phase III study for VX 659 + tezacaftor + ivacaftor to treat F508del mutation cystic fibrosis.

Vertex Pharmaceuticals Incorporated announced that it is initiating the first Phase III study of VX 659, tezacaftor and ivacaftor as an investigational triple combination regimen for people with cystic fibrosis (CF) who have one F508del mutation and one minimal function mutation.

The study will enroll 360 patients, and the primary endpoint of the study is the mean absolute change from baseline in percent predicted forced expiratory volume in one second (ppFEV1) at week four of treatment. The study is designed to support the submission of a New Drug Application (NDA) in the U.S. using data from the 4-week primary efficacy endpoint together with safety data through 12 weeks of treatment.