Vertex Pharmaceuticals’ triple combination, which added the corrector VX-659 to the tezacaftor and ivacaftor combination, resulted in statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in two Phase 3 studies in people with cystic fibrosis (CF).
Data from Vertex’s (VRTX) Phase 3 study in people with one F508del mutation and one minimal function mutation resulted in a mean absolute improvement in ppFEV1 of 14.0 percentage points from baseline at week 4 compared to placebo.
The Phase 3 study in people with two F508del mutations, with the addition of VX-659 to those who are already receiving tezacaftor and ivacaftor, resulted in a mean absolute improvement in ppFEV1 of 10.0 percentage points from baseline at week 4 compared to the control group in whom a placebo was added to tezacaftor and ivacaftor.
The VX-659 triple combination regimen was generally well tolerated and the safety and efficacy profile from the results released supported the potential submission of a New Drug Application (NDA) for the VX-659 triple combination regimen.
We consider this to be great news for Vertex at this time. We were expecting its coming to allow Vertex to reclaim its leadership in treating CF patients. Recently some investors have prematurely rushed to doubt Vertex’s leadership in treating the debilitating and life-threatening disease at its root origin when other small firms demonstrated promising results but at very early clinical trials.
This is not all
Vertex also announced that enrollment was completed 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 as well as in people with two F508del mutations. Vertex 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.
According to Vertex the data that have already been reported and of those expected to be reported in early 2019 will enable Vertex to select the best regimen to submit for potential regulatory approvals globally. These data are expected to provide the basis for potential submission of an NDA for a triple combination regimen to the Food and Drug Administration (FDA) no later than mid-2019.
The results from the trials with different products on CF patients having different mutations are extremely important. They enable pinpointing the best, most effective combinations with the least adverse effects for CF patients who have at least one F508del mutation.
According to Reshma Kewalramani, M.D., Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex, the firm plans to evaluate data for the VX-445 and VX-659 triple combination regimens in the first quarter of next year and to choose the best regimen to submit for potential approval. The goal is bringing forward a new treatment option for patients having one F508del mutation and one minimal function mutation as well as to those with two F508del mutations as rapidly as possible.
For details about the VX-659 Phase 3 Study in people with one F508del mutation and one minimal function mutation, the open-label extension study, the VX-659 Phase 3 study in people with two F508del mutations and the topline data, please read the press release by clicking on the following link.
Data from the studies of VX-659 and VX-445 will also be used for regulatory submissions outside the U.S. planned for late 2019.
A Final Word
This is great news. It guarantees the growth of Vertex’s CF products’ sales’ revenues, in addition to the firm’s capability of financing its other investigational products in its pipeline, alone and with other partners, including Crispr Therapeutics (CRSP).
This news is, indeed, impressive.