Mar 27, 2008 - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced results from a planned interim analysis of an ongoing Phase 2a clinical trial in patients who carry the G551D mutation in the gene that causes cystic fibrosis (CF). The interim analysis showed that dosing of VX-770, an investigational CF potentiator, as an oral agent for 14 days resulted in improved lung function and in improved function of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein as measured by changes in sweat chloride levels and changes in nasal potential difference (NPD). Results from the interim analysis support the hypothesis that improving chloride ion transport in CF patients may correlate to improvements in lung function and provide benefit to patients. Results are being shared with regulatory authorities and leading CF investigators in order to identify the most rapid path forward for the compound. "While these are early data, it is unprecedented for an investigational oral compound for the treatment of CF to have such a marked effect on multiple measures of CF disease activity. We saw an average improvement in lung function of 10 percent in patients receiving the highest dose, compared to no observed improvement in patients who received placebo," said Frank Accurso, M.D., Director of the Cystic Fibrosis Center and Professor of Pediatrics at the University of Colorado School of Medicine in Denver. "These data suggest that VX-770 may be able to improve lung function by targeting an underlying defect in CFTR that causes the disease. In patients with CF, inadequately functioning or missing CFTR is believed to result in abnormal balance of fluid and salt in the airways. These are early clinical data in a subset of patients with malfunctioning CFTR, but an important proof-of-concept, and we look forward to evaluating the longer-term safety and efficacy of VX-770 in additional studies."... Vertex's Press Release -
