BioCentury
ARTICLE | Clinical News

VX-661: Phase III discontinued

August 22, 2016 7:00 AM UTC

Vertex said it will discontinue a 2-part, double-blind, international Phase III trial in about 150 CF patients with 1 copy of the delta F508 CFTR mutation and a second mutation that results in minimal CFTR function in Part A of the trial after an interim futility analysis by an independent DSMB showed that once-daily 100 mg oral VX-661 plus Kalydeco ivacaftor did not lead to a prespecified improvement in lung function. The company said there were no safety concerns in the DSMB’s review.

The Phase III program of VX-661 plus Kalydeco consists of 3 additional trials in patients with 1 or 2 copies of the delta F508 CFTR mutation. Next half, Vertex expects data from a Phase III trial of VX-661 plus Kalydeco in CF patients with 2 copies of the delta F508 CFTR mutation and another trial in CF patients with 1 copy of the delta F508 CFTR mutation and a second mutation that results in residual CFTR function. By early 2017, the company also expects to complete enrollment in a Phase III trial of the combination in CF patients with 1 copy of the delta F508 CFTR mutation and a second mutation that results in a gating defect in the CFTR protein. In 2H17, Vertex plans to submit an NDA to FDA for the combination. ...