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

May 7, 2012

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.


On May 29, 2012, Vertex Pharmaceuticals released corrected interim results from the Phase 2 combination study of Kalydeco™ and VX-809.

The corrected results show that fewer study participants had an improvement in lung function of 5 percent or more than Vertex had originally reported on May 7, 2012.

The improvements in lung function, as shown in the corrected results, seen in those who took Kalydeco and VX-809 in combination remained highly statistically significant. Vertex still plans to begin a pivotal trial of Kalydeco and VX-809, pending final Phase 2 study results expected later this summer. 

People with CF and their families who have questions about the interim Phase 2 results can contact Vertex Medical Information at 1-877-634-8789.

Learn More

  • Read the corrected news release from Vertex Pharmaceuticals Inc.

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