Vertex's cystic fibrosis therapy meets goal of late-stage studies

(Reuters) – Vertex Pharmaceuticals Inc said on Wednesday its triple-combination therapy showed improvement in lung function for people with cystic fibrosis (CF) in two late-stage studies.

The combination therapy – consisting its experimental agent VX-445 and its other CF drugs Kalydeco and tezacaftor – met the main goal of improved lung function in CF patients.

CF is a rare disease that causes thick, sticky mucus to buildup in lungs, resulting in serious infections and declining organ function.

Vertex’s currently approved CF treatments, Kalydeco and Orkambi, address about 45 percent of patients worldwide. It is expected that a triple-combination therapy could treat about 90 percent of people with CF.

In one study, patients with one F508del mutation and one minimal function mutation showed a 13.8 percent point improvement in lung function, while in the other, patients with two of the F508del mutations showed 10 percentage point improvement.

Vertex’s Orkambi treats patients with two copies of the F508del mutation.

In November, the company’s other triple-combination therapy comprising its new compound VX-659, showed a 10 percentage point improvement in lung function.