Vertex to Initiate Phase 3 Development Program for New Once-Daily Triple Combination Regimen in People With Cystic Fibrosis

- Phase 2 study met primary endpoint and all secondary endpoints -

- Phase 2 data demonstrated that a once-daily triple combination of VX-121/ tezacaftor/VX-561 has potential for enhanced clinical benefit compared to TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) -

- Initiation of Phase 3 program expected in the second half of 2021 -

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the company will initiate a Phase 3 development program for the new once-daily investigational triple combination of VX-121/tezacaftor/VX-561 (deutivacaftor) in the second half of 2021. The combination of VX-121/tezacaftor/VX-561 was first identified as having potential for increased efficacy based on its ability to drive higher levels of chloride transport compared to TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in human bronchial epithelial cells in vitro. The combination of VX-121/tezacaftor/VX-561 was evaluated in a Phase 2 study in people with cystic fibrosis (CF) ages 18 and older with one F508del mutation and one minimal function mutation (F/MF) and in people with CF ages 18 and older with two F508del mutations (F/F). The regimen was generally well-tolerated, and the study met the primary efficacy endpoint of absolute change from baseline in ppFEV1 and all secondary efficacy endpoints including absolute change from baseline in sweat chloride concentration in both patient populations. Taken together, these data suggest the triple combination holds the potential to restore cystic fibrosis transmembrane conductance regulator (CFTR) function in people with CF to even higher levels than seen with other Vertex CFTR modulators and thereby provide enhanced clinical benefit.

“TRIKAFTA has demonstrated high levels of efficacy in people with CF who have at least one F508del mutation. However, we remain committed to continuing our efforts to maximize the benefit and convenience we can deliver for these patients,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “With this once-daily, next-in-class, triple combination regimen, our goal is to develop a more effective treatment regimen with the potential to restore CFTR function in people with CF to even higher levels than currently achievable.”

Phase 2 Study Results: