Vertex Pharmaceuticals Inc. said a triple combination of its therapies improved lung function among patients with cystic fibrosis in two late-stage studies.
Cystic fibrosis is a rare, life-threatening genetic condition that results from the poor flow of salt and water into and out of the cells, causing mucus buildup in the lungs, pancreas and other organs. The disease results from changes in the CFTR gene, with F508del mutation being the most common gene defect.
Vertex markets ivacaftor as Kalydeco and the combination of tezacaftor and ivacaftor as Symdeko. Tezacaftor moves the defective CFTR protein to the proper place in the airway cell surface and ivacaftor helps facilitate the opening of the chloride channel on the cell surface to allow salt to move in and out of the cell.
The first study enrolled 385 patients with one F508del mutation and one minimal function mutation and tested the efficacy of a combination of VX-659, tezacaftor and ivacaftor compared to triple placebo among patients aged 12 years and older.
Meanwhile, the second study involved 111 patients with two F508del mutations and tested the efficacy of a combination of VX-659, tezacaftor and ivacaftor compared to placebo, tezacaftor and ivacaftor.
Both studies met their main goal with the VX-659 combination showing statistically significant improvements in the lung function compared to the placebo treatments after four weeks on the therapy.
The first study will continue to evaluate the patients for 24 weeks to test several secondary goals such as lung-related exacerbations and change in sweat chloride quantity within the body.
In addition, both studies showed that the VX-659 triple combination regimen was generally well-tolerated among the patients.
Boston-based Vertex Pharmaceuticals said it completed enrollment for two phase 3 studies to test a competing triple regimen of VX-445, tezacaftor and ivacaftor. It expects to report topline results from the studies in the first quarter of 2019, enabling it to choose the best of the two triple regimens in seeking regulatory approval.
In order to get a fair comparison, the company intends to reveal additional safety and efficacy data for the two VX-659 triple combination studies only after the completion of studies testing the VX-445 regimen.
Vertex said it expects to submit a new drug application for one of the triple combination regimens by the mid-2019.