The opportunity to redefine the treatment of major diseases is what drives everyone at Vertex. Our development portfolio represents the fruit of these efforts. Spend some time here and look around. Click on the pipeline chart to get information on our development programs and links to selected published articles. Go further and see how, just maybe, disease challenges may be overcome with innovation.

VX-770
(Cystic Fibrosis)

Cystic Fibrosis affects about 30,000 people in the United States and approximately 70,000 people worldwide. Cystic fibrosis is caused by genetic mutations that result in a malfunctioning or missing CFTR protein on cell surfaces, that results in an imbalance of salt and water in the airways. This fluid imbalance in the lungs causes a cascade of mucus plugging, infection and inflammation that characterizes CF, and accounts for a large portion of the morbidity and mortality seen with CF.

VX-770, an investigational oral potentiator, is designed to act directly on the malfunctioning CFTR protein to help restore the balance of salt and water. VX-770 was advanced into preclinical development based on successful collaborative research with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) that incorporated capabilities and proprietary ion channel research from Vertex's San Diego research site.

In May 2007, a Phase 2 trial was initiated to evaluate, in approximately 38 patients, the safety and pharmacokinetics of VX-770, and how VX-770 affects biomarkers of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in CF patients with genotype G551D. In March 2008, Vertex announced interim results from part one of this Phase 2 trial. In addition, patient enrollment is complete in Part 2 of the Phase 2a trial of VX-770. Part 2 of the trial will dose VX-770 for up to twenty-eight days in 18 patients. Based on discussions to date with U.S. and EU regulatory authorities, the Company believes that it will be in a position to reach agreement on the initiation of a registration program for VX-770 in 2009, pending results of the 28-day study.

Clinical development of VX-770 is currently focused on a subset of CF patients who have a specific type of CFTR mutation known as G551D that constitutes approximately 4 percent of the CF patient population in the U.S. Vertex also plans to evaluate VX-770 in patients with other mutations that result in malfunctioning CFTR on the cell surface. Click here for the March 27, 2008 press release updating the CF program.