Vertex announced the results from its Phase 2 study of VX-770 for the treatment of cystic fibrosis (CF).  This study was a two-part, randomized, placebo-controlled clinical study that enrolled 39 people with CF who had at least one copy of the G551D mutation. Approximately 4 to 5 % of people with CF carry the G551D mutation. In Part 1 of this study, 16 people received VX-770 (25, 75 or 150mg; cross-over design) and 4 people received placebo for two, 14-day periods. In Part 2, 15 people received VX-770 (150 or 250mg; parallel design) and 4 people received placebo for 28 days. VX-770 was dosed as two tablets taken by mouth every twelve hours. While in the study, patients continued to receive their standard medications for CF in addition to VX-770.

The primary objective of the study was to evaluate the safety and adverse event profile of VX-770 in people with CF. The frequency of adverse events was similar between the VX-770 and placebo groups and between Parts 1 and 2 of the study. In addition, secondary endpoints of the study evaluated the effect of VX-770 on lung function and markers of disease. These markers of disease included assessments of sweat chloride and nasal potential difference (NPD), which were used to determine whether VX-770 impacted the function of the defective cystic fibrosis transmembrane conductance regulator (CFTR) protein. In the study, improvements in lung function were observed among patients who received treatment with VX-770 for 14 days or 28 days as compared to their lung function when they enrolled in the study. Additionally, measurements of sweat chloride and NPD showed that treatment with VX-770 was associated with improvements in CFTR function in the sweat duct and airway epithelial cells.

VX-770 is an oral (tablet) that directly targets the defective CFTR protein known to cause CF.

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