Next-gen cystic fibrosis treatment hits Phase III targets

Boston, Massachusetts-based Vertex Pharmaceuticals (Nasdaq: VRTX) has announced positive data from the Phase III 445-104 study of Trikafta (elexacaftor/tezacaftor/ivacaftor), lifting shares in the company 3%.

The cystic fibrosis (CF) specialist is evaluating the therapy in certain people over the age of 12, identified by the F508del genetic mutation.

The study met its primary efficacy endpoint, showing a statistically-significant 3.7% improvement compared to baseline. The study also met all secondary endpoints, and the regimen was generally well-tolerated.