New England Journal of Medicine Publishes Study about VX-770 — A Promising Drug That Targets Underlying Defect in CF

The New England Journal of Medicine, the world's most influential and widely read medical periodical, features a study this week about a Phase 2 clinical trial of VX-770, an oral drug in development that targets the root cause of cystic fibrosis.

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VX-770 was discovered in a collaboration between Vertex Pharmaceuticals and the Cystic Fibrosis Foundation, which invested more than $75 million in the project. Vertex is developing the drug.

The Phase 2 trial showed improvements for patients who carry the G551D mutation, including lung function and sweat chloride levels - key indicators of the disease. The trial enrolled 39 patients with the G551D mutation. The findings suggest that VX-770 may improve function of a faulty CF protein known as CFTR, which causes CF.

“This research represents a milestone along the pathway of discovery leading to better preventions, treatments and cures," says Michael Welsh, M.D., the author of an independent editorial that accompanied the Journal study.

VX-770 is among the first in a new class of potential drugs known as CFTR modulators.

“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,” said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation.

The primary objective of the study was to evaluate the safety and tolerability of VX-770 in people with 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 now being tested in Phase 3 clinical trials. Results from this program are expected in the first half of 2011. Pending data from the Phase 3 program, Vertex anticipates it will submit a New Drug Application for VX-770 to the U.S. Food and Drug Administration in the second half of 2011.

The Foundation funds more cystic fibrosis research than any other organization in the world, and virtually every approved CF drug available today was made possible in part because of Foundation support. Since the 1980s, the Foundation has played an integral role in the development of Pulmozyme®, TOBI®, azithromycin, hypertonic saline and Cayston®, essential drugs for the treatment of CF.

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