Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that enrollment is complete for the two Phase 3 studies of the next-generation corrector VX-659 in triple combination with tezacaftor and ivacaftor in people with cystic fibrosis (CF) with one F508del mutation and one minimal function mutation and in people with two F508del mutations. Based on the completion of enrollment, Vertex expects to report data from both Phase 3 studies of the VX-659 triple combination regimen in late 2018. Vertex expects to complete enrollment of the two Phase 3 studies of the next-generation corrector VX-445 in triple combination with tezacaftor and ivacaftor in the fourth quarter of 2018 and to report data from these studies in the first quarter of 2019. Vertex plans to evaluate data from both the VX-659 and VX-445 Phase 3 triple combination programs to choose the best regimen to submit for potential regulatory approval. The data expected in late 2018 for VX-659 and in the first quarter of 2019 for VX-445 are expected to provide the basis for submission of a New Drug Application (NDA) to the U.S. FDA for people with one F508del mutation and one minimal function mutation no later than mid-2019.
“The two Phase 3 studies of VX-659, tezacaftor and ivacaftor enrolled nearly 500 people with CF in less than five months, underscoring the significant interest within the CF community to advance triple combination regimens that have the potential to treat up to 90 percent of all people with the disease,” Reshma Kewalramani, M.D., Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “Our goal is to bring the best triple combination regimen to people with CF as quickly as possible, and we look forward to obtaining data later this year and in the first quarter of 2019 to support a submission for potential approval of a triple combination regimen.”
The Phase 3 study of the VX-659 triple combination regimen in patients with one F508del mutation and one minimal function mutation was designed with a pre-specified interim analysis to evaluate the primary endpoint of ppFEV1 at 4 weeks and safety through 12 weeks. This interim analysis will be conducted once all patients are through the primary efficacy endpoint at week 4. These efficacy and safety data from the interim analysis are expected in late 2018 and are intended to support a potential NDA submission for the VX-659, tezacaftor and ivacaftor triple combination regimen in people with one F508del mutation and one minimal function mutation.
Similar to the interim analysis for the VX-659 triple combination regimen, the Phase 3 study of the VX-445 triple combination regimen in patients with one F508del mutation and one minimal function mutation was designed with a pre-specified interim analysis to evaluate the primary endpoint of ppFEV1 at 4 weeks and safety through 12 weeks. This interim analysis will also be conducted once all patients are through the primary efficacy endpoint at week 4. Data from the VX-445 interim analysis are expected in the first quarter of 2019. The interim analyses for the VX-659 and VX-445 triple combination regimens will enable Vertex to evaluate comparable data from both Phase 3 triple combination programs and determine the best regimen to submit for potential regulatory approval.
To preserve the integrity of the Phase 3 studies of VX-659, tezacaftor and ivacaftor and the Phase 3 studies of VX-445, tezacaftor and ivacaftor that will be ongoing into mid-2019, Vertex expects to disclose in late 2018 and the first quarter of 2019 only the topline results for the primary 4-week efficacy endpoints of the VX-659 and VX-445 Phase 3 studies, respectively, and whether the safety and efficacy profiles observed support a potential NDA submission. In the second half of 2019, Vertex intends to disclose additional safety and efficacy data, including secondary endpoints, for each study following the completion of both the VX-659 and VX-445 Phase 3 triple combination programs.
