The Cystic Fibrosis Foundation issued the following statement in response to an announcement by Vertex Pharmaceuticals Inc. regarding promising results from a Phase 2 clinical trial of its cystic fibrosis drug Kalydeco™ in combination with another potential CF therapy, VX-661. The results showed a significant improvement in lung function in people with two copies of the most common CF mutation, Delta F508, who took the combination treatment, compared with those who received a placebo. About 50 percent of people with CF in the United States have two copies of the Delta F508 mutation.
Both Kalydeco and VX-661 are designed to treat the underlying cause of CF, a faulty gene and its protein product, known as CFTR.
"This announcement is more terrific news for the CF community," said Robert J. Beall , Ph.D., president and CEO of the Cystic Fibrosis Foundation. "While these results are still very early, they show that another potential therapy from our ongoing collaboration with Vertex can be combined with Kalydeco to achieve promising improvements in health. The data further validate our strategy of using small molecule compounds to address the basic genetic defect in cystic fibrosis."
The CF Foundation played a key role in the development of Kalydeco and VX-661, as well as other experimental compounds such as VX-809, providing significant scientific, clinical and financial support. Vertex is studying Kalydeco with VX-809 in Phase 3 trials that will enroll about 1,000 people at approximately 200 clinical trial sites in North America, Europe and Australia. "The CF Foundation is deeply grateful to the people with CF, families and medical professionals who have played a role in these studies, and to the Vertex scientists who remain dedicated to advancing CF drug development," said Beall.
Cystic Fibrosis Foundation