Vertex takes next step in testing medicine to treat the underlying cause of  cystic fibrosis

Vertex takes next step in testing medicine to treat the underlying cause of cystic fibrosis

February 1, 2018 Off By Dino Mustafić
Vertex Pharmaceuticals will move its cystic fibrosis treatment VX-659 and VX-445 into Phase 3, the company said Wednesday, after the initial Phase 2 data.

Vertex plans to kick off a Phase 3 program in the first half of 2018 to see if VX-659 is effective in triple combination with tezacaftor and ivacaftor. In addition, the company said it wants to start a Phase 3 program in mid-2018 to test VX-445 in triple combination with tezacaftor and VX-561 as a once-daily regimen. This can be done after getting additional data in the first half of 2018, including the Phase 2 data on the combination of VX-445, tezacaftor and VX-561.

The company said the triple combination regimens were generally well tolerated across both studies, and the majority of adverse events were mild to moderate in severity. Across the studies, the discontinuation rate due to adverse events was low.
“These results support the selection of both the VX-659 and VX-445 triple combination regimens and underscore the potential for these regimens to provide significant clinical benefits for up to 90 percent of people with CF,” said Jeffrey Chodakewitz, Executive Vice President and Chief Medical Officer at Vertex.
Jennifer Taylor-Cousar, co-chair of Vertex’s Triple Combination Steering Committee, said: “Together, all the Phase 2 data to date provide further evidence that the addition of a next-generation corrector to tezacaftor and ivacaftor has the potential to provide substantial clinical benefits to patients with one F508del and one minimal function mutation who don’t currently have a medicine to treat the underlying cause of their CF, as well as to provide additional benefits to patients with at least one F508del mutation who are already eligible for CFTR modulator therapies.”