VX-770
(Cystic Fibrosis)
Cystic Fibrosis (CF) 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.
Vertex is conducting a Phase 2a trial to evaluate, in 39 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 who have a specific type of CFTR mutation known as G551D. In March 2008, Vertex announced interim results from Part 1 of this Phase 2a trial and in October 2008, Vertex announced interim results of Part 2 of this Phase 2a trial. Based on these results, Vertex intends to work with global regulatory authorities to finalize the design of a registration program for VX-770 targeted to begin in the first half of 2009. Click here for the October 20, 2008 press release updating the CF program.
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.
