Vertex Pharmaceuticals plans to conclude the enrollment of patients in its Phase 3 programs involving its triple combo therapies to treat cystic fibrosis (CF) in the second half of 2018, the company recently announced in a corporate update.
This is earlier than expected for the four Phase 3 trials, which are testing triple combinations of VX-661 (tezacaftor) and Kalydeco (ivacaftor) plus either VX-659 or VX-445, both of which are investigational treatments.
Based on this anticipated completion of enrollment, Vertex intends to submit a new drug application to the U.S. Food and Drug Administration for these triple combo therapies by mid-2019.
Earlier this year, the company announced the start of a randomized, double-blind Phase 3 trial (NCT03447249) testing the VX-659 combo in CF patients with one F508del mutation and one minimal function mutation in the CFTR gene — the gene that causes the disease. This trial is supported by early positive data from a recently completed Phase 2 trial (NCT03224351).
The VX-659 combo will also be tested in a Phase 3 trial (NCT03460990) in CF patients with two copies of the F508del mutation. These trials are part of the company’s ECLIPSE program.
In May, Vertex also reported the launch of two randomized, double-blind Phase 3 trials to evaluate the safety and effectiveness of the VX-445 triple combo therapy in CF, as part of its AURORA program.
CF patients, ages 12 and older, with one F508del mutation and one minimal function mutation, or two copies of the F508del mutation, are eligible for enrollment.
Vertex also reported that, after the recent U.S. approval of Symdeko (tezacaftor/ivacaftor and ivacaftor) for cystic fibrosis, the company has seen a strong demand for the medication, and it has received broad access and coverage from commercial and government payers.
“We have made tremendous progress across our business in the first half of 2018 marked by the successful launch of Symdeko, the fast enrollment of two Phase 3 programs for our triple combination regimens, and continued success in bringing our CF medicines to more people outside the U.S.,” Jeffrey Leiden, MD, PhD, Vertex’s CEO, said in the press release.
“As we enter the second half of the year, we are well positioned to continue our progress toward developing new medicines for all people with CF as well as advancing other transformative medicines outside of CF from research into development,” he said.
Vertex is focusing on developing therapies able to treat CF patients at younger ages, earlier in the disease course.
The company expects data from Phase 3 trials in infants and children to be available in the second half of 2018, namely results in infants ages 6 months to less than 12 months taking part in a trial testing Kalydeco (NCT02725567), and children 6 to 11 years old receiving tezacaftor and Kalydeco (NCT03559062).
In addition, the company plans to launch a Phase 3 trial testing Orkambi (lumacaftor/ivacaftor) in children 12 months to less than 24 months old in the second half of 2018.
Vertex also has pending approvals for Kalydeco to treat children ages 12 months to less than 24 months, with a Prescription Drug User Fee Act (PDUFA) action date of Aug. 15; and for Orkambi in children ages 2 to 5 years old, with a PDUFA date of Aug. 7. PDUFA dates are deadlines for the FDA to approve new medications or indications.