The company said it plans to cease the Phase III trial of VX-661 in combination with Kalydeco (ivacaftor) in people with one copy of the F508del mutation and one copy of a mutation that results in minimal CFTR protein function.
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The trial was reviewed during a planned interim analysis by a Data Safety Monitoring Board, which recommended the trial end.
The terminated trial was part of a Phase 3 program which comprises three other late-stage studies testing the combination regimen in various groups of CF patients with at least one copy of the F508del mutation.
“Vertex is still not disclosing the bar for this interim analysis, so it is hard to know if any small signals activity were observed, though we believe if there were any lung function benefits worth following up on the study would likely have proceeded to part 2”. It has multiple development programs in the field of CF, including VX-661, VX-371, and VX-152 and VX-440. Data from this study were presented at the 39th European Cystic Fibrosis Society (ECFS) conference on June 10, 2016. The report stated: “We are positive about Vx-661, and expect 3 of 4 ongoing phase III trials with Vx-661 to succeed, and show superiority to their comparator arms and to the prior precedents with Kalydeco and Orkambi in their respective indications”. It is approved for treating CF patients who have two F509del mutations of CFTR.
Vertex expects to file VX-661 plus ivacaftor for approval in the second half of 2017. CF, a rare life-shortening disease, affects almost 75,000 people across North America, Europe and Australia with a median age of death in the mid-20s. The median predicted age of survival for a person with cystic fibrosis is between 34 and 47 years, but the median age of death remains in the mid-20s, Vertex said.
One copy of the F508del mutation and a second mutation that results in residual CFTR function. CF is caused by loss-of-function mutations in the CFTR protein. Children must inherit two defective CFTR genes – one from each parent – to have CF. The trial was meant to test the combination compound in patients suffering from a specific mutation of cystic fibrosis (CF). All of these changes prevent the channel from functioning properly and cells that line the passageways of the lungs, pancreas, and other organs produce mucus that is abnormally thick and sticky. The Company uses precision medicine approaches to create transformative drugs for patients with serious diseases in specialty markets.
Founded in 1989 in Cambridge, Mass., Vertex today has research and development sites and commercial offices in the United States, Europe, Canada and Australia.
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This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Dr. Chodakewitz’s statements in the second paragraph of the press release and statements regarding (i) the timing of completing enrollment of Phase 3 studies of VX-661 in combination with ivacaftor and (ii) plans to submit an NDA to the FDA for VX-661 in combination with ivacaftor.
