Positive results were announced today from late-stage studies of a potential triple-combination CFTR modulator therapy for people with cystic fibrosis. Vertex Pharmaceuticals Inc. released Phase 3 clinical trial data for two studies of the next-generation modulator VX-659 in combination with ivacaftor and tezacaftor.

The first study tested this triple-combination drug on people with CF ages 12 years and older who have one copy of the most common cystic fibrosis mutation, F508del, and one minimal function mutation. Results stated that those who received VX-659 combined with ivacaftor and tezacaftor had a 14 percent increase in lung function compared to participants taking a placebo. Researchers believe that anyone with at least one copy of the F508del mutation -- regardless of their second mutation -- could benefit from next-generation modulators.

The second study included people with CF ages 12 years and older who have two copies of the F508del mutation. The trial compared the effectiveness of Symdeko® (tezacaftor/ivacaftor) to VX-659 combined with tezacaftor and ivacaftor. Participants who were given VX-659 combined with tezacaftor and ivacaftor had a 10 percent improvement in lung function over those who were only given Symdeko® (tezacaftor/ivacaftor).

VX-659 is one of two next-generation modulators currently being tested by Vertex. The results for the second modulator (VX-445) are expected in early 2019. It is anticipated that after these top-line clinical trial results are announced next year, Vertex will decide which of the two next-generation modulators they will submit to the U.S. Food and Drug Administration (FDA) for potential approval as a new drug. If approved, it is estimated that triple-combination CFTR modulators could bring the benefits of therapies that treat the underlying cause of the disease to more than 90 percent of people with CF.

“It is an exciting time for the CF community, as we approach a milestone that seemed impossible even just a few years ago. Today's announcement represents an important step in our journey to developing treatments for the underlying cause of this disease for all people with cystic fibrosis,” said Michael P. Boyle, M.D., senior vice president of therapeutics development for the Cystic Fibrosis Foundation. “We are enthusiastic about the clinical benefit VX-659 demonstrated in these studies and look forward to seeing how these compare to the other next-generation modulator in clinical trials, VX-445.”

For additional information regarding the results of these clinical trials, please see the Vertex press release.

The Foundation is steadfast in its commitment to finding treatments for the underlying cause of the disease for people with nonsense and rare mutations who will not benefit solely from CFTR modulators, and to the continued development of treatments for complications of the disease. To learn more about the research the Foundation is supporting in these areas, please visit the Research We Fund section of cff.org.