VX-770 drug candidate improves lung function in people with cystic fibrosis

In a study published in this week's New England Journal of Medicine, treatment with a new drug candidate known as VX-770 resulted in improvements in lung function and markers of disease in a Phase 2 clinical trial of 39 people with cystic fibrosis (CF). There were no discontinuations due to adverse events in the study, and the frequency of adverse events was similar across the study groups. VX-770 is an oral (tablet) medicine that is being developed by Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) to directly target the defective protein known to cause CF. An accompanying editorial on this study was also published in this week's New England Journal of Medicine.

“Based on these results, we are now evaluating VX-770 as part of a Phase 3 development program and expect data early next year”

"This study marks a significant step in the development of innovative CF therapies that target the defective protein known to be the underlying cause of CF," said Frank Accurso, M.D., Lead Investigator for the VX-770 study and Director of the Cystic Fibrosis Center and Professor of Pediatrics at the University of Colorado Denver and Children's Hospital in Aurora. "The increase in lung function and improvements in other markers of disease observed in this trial support the continued evaluation of VX-770 in late-stage trials."

CF is a genetic disease that affects 30,000 people in the United States. The disease is caused by a mutated gene that produces defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) proteins. The absence of functional CFTR proteins results in poor flow of fluids across certain cell membranes, including in the lung, and leads to accumulation of abnormally thick, sticky mucus that contributes to chronic lung infections and progressive lung damage. The study published in this week's New England Journal of Medicine enrolled people with CF who have the G551D mutation in the CFTR gene, where the CFTR protein reaches the cell surface but does not function properly. VX-770, known as a CFTR potentiator, aims to increase the function of defective CFTR proteins by increasing the gating activity, or ability to transport chloride ions, across the cell membrane.

Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation stated, "Nearly a decade ago, the CF Foundation recognized the need to develop new therapies that address the underlying cause of CF and not just the symptoms of the disease. We are encouraged by the data from this Phase 2 trial and see the trial as a milestone in our efforts to discover and develop new treatment options for this disease."

VX-770 was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) to discover and develop novel CFTR modulators. CFFT is the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. Vertex retains worldwide rights to develop and commercialize VX-770.