Vertex Potentiator Trial Underway; Corrector Slated to Begin Trials by Year's End

November 7, 2007

The Vertex compound known as VX-770 has entered Phase 2 clinical testing and is in the process of enrolling 36 adult volunteers at more than a dozen care centers across the country. To view a list of the care centers that are enrolling for this trial, click here and keyword search VX-770.

“This is incredibly exciting for us because it is the first oral drug to directly treat the abnormal CFTR ion transport defect in CF," said Foundation President and CEO Robert J. Beall, Ph.D. “We talk about 'shots on goal' in this disease and this is one of the most important."

The VX-770 trial studies the safety, tolerability and absorption rate of VX-770. The compound, which belongs to a category of compounds called CFTR potentiators, is believed to partially restore CFTR protein function and increase the probability that the CFTR channel is open. VX-770 may allow for increased chloride transport across the cell surface, alleviating a fundamental problem in cystic fibrosis. The previous Phase 1 trial, completed in 2006, showed that the potential drug could achieve the expected levels in the blood.

In addition, Vertex recently selected a second compound for development. The compound, known as VX-809, is expected to begin clinical development by the end of 2007. VX-809 is part of a class of compounds known as "correctors," which help the defective CF protein move to its proper place in the cell.

“These are tremendous milestones for us," said Beall. "These are the result of an historic collaboration with Vertex, which harnesses high-throughput screening for drug discovery.  These two compounds are the first by-products of our investment in high-throughput screening.  If these two compounds work, they may help treat up to 90% of CF patients."

Both VX-770 and VX-809 were developed by Vertex Pharmaceuticals as a result of early-stage development support from the CF Foundation.