Vertex stumbles in Phase III cystic fibrosis trials
pharmafile | August 16, 2016 | News story | Manufacturing and Production, Research and Development | Vertex, cystic fibrosis, phase III
Vertex Pharmaceuticals has provided an update on its ongoing Phase III trials investigating VX-661 in combination with Kalydeco (ivacaftor) in cystic fibrosis, with one study in patients with specific mutations set to be cancelled.
The company is calling off trialling its combination therapy in patients with one copy of the F508del mutation and one copy of a mutation that results in minimal CFTR protein function. In part A of the study, VX-661 plus Kalydeco failed to improve lung function in this cohort of patients.
To cushion the blow of this cancellation, Vertex has also announced that enrolment is now complete for its study of VX-661 and Kalydeco in patients with two copies of the F508del mutation. Having two copies of this mutation, inherited from both parents, is the leading cause of cystic fibrosis.
This study is one of four the company plans to undertake in specific mutations which cause cystic fibrosis using this combination treatment. The results of these trials will form the basis of a new drug application that Vertex plans to file with the US Food and Drug Administration in the second half of 2017.
Jeffrey Chodakewitz, chief medical officer at Vertex, says: “While we recognise that people with CF with minimal function mutations have a form of the disease that is particularly difficult to treat, we believe it was important to evaluate a dual combination of VX-661 and ivacaftor could provide some benefit to these patients given they do not today have a medicine to treat the cause of their disease.
“These results suggest that a triple combination regimen may provide this group of people with CF the best chance at obtaining a meaningful benefit and we look forward to beginning the first study of a next-generation corrector together with VX-661 and ivacaftor in this group of patients later this year.”
Shares at the company fell 3% at the time of writing.
Sean Murray
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