Positive Early Stage Study Results Announced for Next-Generation Modulator

Phase 2 clinical trial results were announced today for a potential next-generation modulator to treat people with two copies of the most common cystic fibrosis mutation, F508del.

March 1, 2018 | 2 min read

Phase 2 clinical trial results were announced today for the cystic fibrosis transmembrane conductance regulator (CFTR) modulator candidate VX-659 in combination with tezacaftor and ivacaftor for people with CF who have two copies of the F508del mutation. VX-659 is one of two drug candidates that Vertex Pharmaceuticals Inc. recently decided would be moving into Phase 3 clinical trials.

The early stage study showed a significant improvement in lung function (9.7 percent increase in FEV₁ above baseline) and an improvement in sweat chloride (-42.2 mmol/L) when VX-659 was added to the treatment in participants with two F508del mutations who already were taking tezacaftor and ivacaftor.

In January, Vertex announced its decision to advance VX-659 and its other triple modulator candidate, VX-445, into Phase 3 clinical trials. Enrollment for the Phase 3 trials of VX-659 is expected to begin in the next several weeks. In mid-2018, Vertex also plans to begin a Phase 3 clinical trial for VX-445 in combination with tezacaftor and the compound VX-561 as a once-daily therapy in people with one F508del mutation and one minimal function mutation, and in people with two F508del mutations.

The Cystic Fibrosis Foundation provided $150 million in research funding to Vertex to support its pursuit of treatments that address the underlying defect in CF, including $75 million to develop next-generation candidates currently in clinical development.

For additional information regarding the results of these clinical trials, including the safety and efficacy outcomes, please see the Vertex press release.

Share this article
Research | Drug Pipeline
Recent news
The CF Foundation’s 16th Annual Teen Advocacy Day Empowers Teens to Make Their Voice Heard
News | 4 min read
Cystic Fibrosis Community Perspectives Influence Future of CF Care Model
News | 4 min read
CF Foundation Adapts Registry Reporting to New Race-Neutral Standards
News | 5 min read
Stay up to date with The Foundation

Sign up for our newsletter to get all of the latest news from The Foundation right in your inbox.