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-809
(Cystic Fibrosis)

VX-809 is the second investigational oral drug candidate for the treatment of cystic fibrosis (CF) to be advanced into development as part of a successful collaboration with the Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT).

VX-809 may act to restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, the defective cell membrane protein responsible for the progression of CF. Defects in the CFTR protein affect the transport of chloride and other ions across cells, and lead to the accumulation of thick, sticky mucus in the lungs of patients with CF. This mucus fosters chronic infection and inflammation, and results in irreversible lung damage.

Corrector compounds such as VX-809 are designed to increase the amount of F508del CFTR protein on the surface of cells lining the airway, which may result in an increase in chloride transport across the cell surface in patients with the F508del CFTR mutation.

Based on Phase 2a data for VX-809, Vertex expects to initiate a Phase 2a clinical trial that will evaluate combination regimens of VX-809 and VX-770 in the second half of 2010. The trial will enroll patients who are homozygous for the F508del mutation.