Vertex Triple Combination CF Drugs Hit the Mark in Phase III Trials

A Vertex triple-combination treatment for cystic fibrosis hit the mark in two Phase III trials, setting the stage for the company to secure approval for another treatment for the life-shortening genetic disease that affects approximately 75,000 people in North America, Europe, and Australia. This morning, Boston-based Vertex said its triple-combination treatment VX-445, tezacaftor, and ivacaftor resulted in statistically significant improvements in lung function in two late-stage cystic fibrosis trials. The company said that data from a pre-specified interim analysis of the Phase 3 study in people with one F508del mutation and one minimal function mutation showed a mean absolute improvement in percent predicted forced expiratory volume in one second (ppFEV1). The improvement was 13.8 percentage points from baseline at week four of treatment compared to placebo, Vertex said. In the Phase III study of people with two F508del mutations, the addition of VX-445 in patients already receiving tezacaftor and ivacaftor resulted in a mean absolute improvement in ppFEV1 of 10 percentage points from baseline at week 4 of treatment compared to the control group in whom a placebo was added to tezacaftor and ivacaftor.