In April, the CF Foundation announced an unprecedented milestone in its effort to treat the basic defect in cystic fibrosis. Historic results from an ongoing Phase 2 clinical trial of VX-770, a potential therapy being developed by Vertex Pharmaceuticals, Inc., suggest that the compound improves the function of the faulty CFTR protein. VX-770 is the result of a collaboration that began in 1998 with Vertex Pharmaceuticals and is the largest in the Foundation's history.
The interim data from the Phase 2a study performed in a subset of CF patients indicate that VX-770 improves lung function, sweat chloride levels and nasal potential difference. The first compound of its kind, VX-770 belongs to a category of compounds termed potentiators, which aim to improve the opening of the CFTR chloride channel that is defective in CF. It is being studied in patients who carry the G551D mutation. More »