(RTTNews) – Vertex Pharmaceuticals Inc. (VRTX) said Tuesday that two Phase 3 studies of the triple combination of the next-generation corrector VX-659, Tezacaftor and Ivacaftor met the primary endpoint of improvement in lung function in people with cystic fibrosis or CF.
According to Vertex Pharma, the treatment with triple combination resulted in statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in the two Phase 3 studies in people with CF.
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 ppFEV1 of 14.0 percentage points from baseline at week 4 in people with one F508del mutation and one minimal function mutation compared to placebo.
In the Phase 3 study in people with two F508del mutations, the addition of VX-659 in patients already receiving tezacaftor and ivacaftor resulted in a mean absolute improvement in ppFEV1 of 10.0 percentage points from baseline at week 4, when VX-659 was added in people with two F508del mutations already receiving tezacaftor and ivacaftor, compared to control group of placebo added to tezacaftor and ivacaftor.
Vertex noted that the safety and efficacy profile from the results released today supports potential submission of a New Drug Application for the VX-659 triple combination regimen.
Vertex also said that enrollment is complete for the two Phase 3 studies of the triple combination of the next-generation corrector VX-445, tezacaftor and ivacaftor in people with CF with one F508del mutation and one minimal function mutation and in people with two F508del mutations.
Vertex added that it remains on track to report topline data from both Phase 3 studies of the VX-445 triple combination regimen in the first quarter of 2019.
The company noted that the data expected in the first quarter of 2019 for VX-445 and the data reported today for VX-659 will enable it to choose the best regimen to submit for potential regulatory approvals globally and also provide the basis for potential submission of an NDA for a triple combination regimen to the U.S. FDA no later than mid-2019.
Nov. 27, 2018