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Research Update -
Vertex Announces Data from Study of VX-661 in Combination with Ivacaftor

Written by: Andrea N. Flynn, Ph.D.

Vertex recently announced results from a 12-week Phase 2 study of the CFTR corrector VX-661 in combination with ivacaftor, a CFTR potentiator in individuals ages 18 years or older with two copies of F508del-CFTR. Corrector therapies are designed to help the F508del-CFTR protein reach the cell surface, while potentiator therapies act at near the cell surface to keep the CFTR channel open. The study evaluated the effect of the combination therapy on lung function (forced expiratory volume in one second; FEV1) and found a rapid improvement in lung function within 4 weeks of treatment. The study also found lung function returned to the pre- treatment levels after participants completed the 12-week study.

"The safety and efficacy data from this study are consistent with prior Phase 2 studies of VX-661 in combination with ivacaftor and provide further support for our ongoing Phase 3 program in people with one or two copies of the F508del mutation," said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex.

A summary of the phase 3 study is as follows and additional details can be found at www.vrtx.com:

  • Two Copies of the F508del Mutation: Enrollment is ongoing in people ages 12 and older who have two copies of the F508del mutation and aims to enroll approximately 500 patients in North America and Europe.

  • One Copy of the F508del Mutation and a Second Mutation That Results in Residual CFTR Function: Vertex plans to begin a Phase 3 study in April in people ages 12 and older who have one copy of the F508del mutation and a second mutation that results in residual CFTR function. This study will also evaluate ivacaftor dosed without VX-661 and aims to enroll approximately 300 patients.

  • One Copy of the F508del Mutation and a Second Mutation that Results in a Gating Defect in the CFTR Protein: Vertex plans to begin a Phase 3 in May in people ages 12 and older who have one copy of the F508del mutation and a second mutation that results in a gating defect in the CFTR protein. The study will enroll approximately 200 patients.

  • One Copy of the F508del Mutation and A Second Mutation That Results in Minimal CFTR Function: In mid-2015, Vertex plans to begin a Phase 3 study in people ages 12 and older who have one copy of the F508del mutation and a second mutation that results in minimal CFTR function. The study will initially enroll approximately 120 patients but depending on an interim analysis, enrollment may be expanded.
Cystic Fibrosis Foundation Teams Up with Anthera Pharmaceuticals To Develop Sollpura

The CF Foundation Therapeutics recently awarded Anthera Pharmaceuticals a $3 million grant to support both the manufacturing and clinical development of SollpuraÔ (liprotamase), a novel pancreatic enzyme replacement therapy. Anthera plans to begin a Phase 3 clinical trial, SOLUTION, to assess

liprotamase later this year. If the trial yields positive results, Anthera will complete all of the regulatory and clinical activities needed for a New Drug Application to the United States Food and Drug Administration. More information is available at www.anthera.com.