News & Articles 
Vertex CF Drug Shows Promise in Trial
February 3 2010
source Reuters
Vertex Pharmaceuticals Inc. said today that a mid-stage trial of its experimental cystic fibrosis drug showed promise, and the company will study it further.
Cambridge, Massachusetts-based Vertex said preliminary analysis of data from a 28-day trial of its drug, known as VX-809, in patients with a certain genetic mutation, showed patients experienced a statistically significant decline in sweat chloride.
Sweat chloride becomes elevated in patients with cystic fibrosis and is caused by a defective protein in certain cells in the sweat duct.
Most patients with cystic fibrosis have a genetic mutation known as F508del which causes a protein, known as Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), to be missing from the cell surface.
VX-809 is designed to direct the protein to its proper location.
The company is also developing a drug known as VX-770 which is designed to treat patients who have the protein but find it does not work properly.
Vertex said it plans to begin a combination trial of VX-809 and VX-770 in the second half of 2010 for patients with the F508del mutation. The idea is that VX-809 would direct the CFTR protein to the cell surface, while VX-770 would optimize its effectiveness.
Cystic fibrosis is a life-threatening genetic disease affecting about 30,000 people in the United States and 70,000 worldwide. Defects in the CFTR gene result in poor chloride flow across cell membranes, causing the body to produce abnormally thick, sticky mucus that leads to chronic, life-threatening lung infections.
Note: To view the Vertex Pharmaceuticals press release on this story, please click here.
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