Vertex Pharma Begins Phase 3 Study Of VX-659 - Quick Facts

(RTTNews) - Vertex Pharmaceuticals Incorporated (VRTX) announced the company is initiating a Phase 3 study of VX-659, tezacaftor and ivacaftor as an investigational triple combination regimen for people with cystic fibrosis who have two copies of the F508del mutation, the most common genetic form of the disease. The study will enroll approximately 100 patients, and the primary endpoint of the study is the mean absolute change from baseline in percent predicted forced expiratory volume in one second at week four of treatment.

The company said the study is designed to support the submission of an application for approval in patients with two copies of the F508del mutation in the U.S. using data from the 4-week primary efficacy endpoint together with 24-week safety data generated from the recently initiated Phase 3 study in patients with one F508del mutation and one minimal function mutation.