May 8, 2012

Phase 2 Study of Kalydeco and VX-809 in Combination Shows Promising Interim Results

(Bethesda, Md.) – Vertex Pharmaceuticals Inc. today announced promising interim results from a Phase 2 clinical trial of its cystic fibrosis drug Kalydeco™ and VX-809, a CF drug in development.

The results showed a significant improvement in lung function in people with two copies of the most common CF mutation who received the two drugs in combination.

Both Kalydeco and VX-809 are designed to treat the underlying cause of CF. Complete results from the Phase 2 trial are expected this summer.

The ongoing Phase 2 study enrolled 108 people, ages 18 and older, who have one or two copies of the Delta F508 mutation. Today’s results are based on data from about half of the study participants after they had completed 56 days of treatment.

Vertex plans to begin a pivotal trial of Kalydeco and VX-809 in people with two copies of the Delta F508 mutation, pending final study results. Pivotal trials are typically designed to gather data that could be used by the U.S. Food and Drug Administration (FDA) to decide whether or not to approve a potential drug.

"We are eagerly awaiting the full results, and are pleased that Vertex is accelerating its plans for a pivotal study of the combination treatment in those with two copies of Delta F508," said Robert J. Beall, Ph.D., president and CEO of the CF Foundation.

The CF Foundation played a key role in the development of Kalydeco and VX-809, providing significant scientific, clinical and financial support.

About 50 percent of people with CF in the United States have two copies of the Delta F508 mutation. About 40 percent of people with CF in the United States have one copy.

Earlier this year, the FDA approved Kalydeco for people with the G551D mutation ages 6 and older. Kalydeco is the first drug that treats the underlying cause of CF — a defective gene and its protein product, known as CFTR.

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About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, go to

Media Contact

  • Laurie Fink, national director of media relations, Cystic Fibrosis Foundation: (301) 841-2602;