US-based biotechnology company Vertex Pharmaceuticals Incorporated is set to begin a Phase lll study of VX-659 and VX-445 under two different triple combination regimens for treating cystic fibrosis (CF).
The study will be conducted in two parts, with one expected to start by the first half of this year and be designed to investigate VX-659 in triple combination with tezacaftor and ivacaftor to treat F508del mutation and one minimal function mutation (F508del/Min) patients.
In the second part of the study, VX-445 will be evaluated in triple combination with tezacaftor and VX-561 in people who have two copies of F508del mutation (F508del/F508del).
The combination is also expected to be evaluated as a once-daily regimen in F508del/Min and F508del/F508del patients.
The study will be based on initial Phase ll data from an ongoing combination study of VX-445, tezacaftor, and VX-561.
New data from the randomised, double-blind, Phase ll study have demonstrated mean absolute improvements in a percent predicted forced expiratory volume in one second (ppFEV) of up to 13.3% and 13.8% from baseline through four weeks of treatment for the triple combination regimens with VX-659 or VX-445 respectively. This was in people with one F508del mutation and one minimal function mutation (F508del/Min).
Vertex executive vice-president and chief medical officer Jeffrey Chodakewitz said: “These results support the selection of both the VX-659 and VX-445 triple combination regimens and underscore the potential for these regimens to provide significant clinical benefits for up to 90% of people with CF.”
The company is currently in discussions with regulators to begin the Phase lll programme.