Vertex Phase 3 Data: Absolute Improvement +12.5% Vs. Placebo, No Discountinuations (VRTX)

Vertex Pharmaceuticals Incorporated VRTX today announced positive results from a 24-week analysis of the ongoing Phase 3 ENVISION study of VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis (CF). ENVISION (n=52) was designed to evaluate VX-770 among children ages 6 to 11 with the G551D mutation in the CFTR gene. Approximately 4 percent of people with CF in the United States have at least one copy of the G551D mutation. The study met its primary endpoint of mean absolute change from baseline in percent predicted FEV1 (forced expiratory volume in one second, or lung function) through week 24. A difference in mean absolute improvement from baseline in lung function of 12.5 percent and a difference in mean relative improvement from baseline in lung function of 17.4 percent compared to placebo (p<0.0001) were observed through week 24. Significant improvements in other measures of disease, including weight gain and a reduction in sweat chloride, were also observed through week 24 among those who received VX-770. At the time of this analysis, the most commonly reported adverse events were respiratory in nature and comparable across treatment groups. The study is ongoing, and additional assessments of key endpoints will be conducted at the end of the 48-week study. Vertex is on track to submit regulatory applications for approval in the United States and Europe in the second half of 2011. The submissions will be based on data from the Phase 3 STRIVE and ENVISION studies as well as the Phase 2 DISCOVER study. Vertex plans to submit data from all studies in the VX-770 Phase 3 registration program for presentation at upcoming medical meetings. “In this study, children with CF treated with VX-770 showed the same profound improvements in lung function seen in the recently-announced STRIVE study among an older group of people with the G551D mutation,” said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer for Vertex. “We are committed to making VX-770 available as soon as possible and are moving ahead quickly with our U.S. and European applications for regulatory approval that we plan to submit in the second half of this year.” “The data announced today are highly encouraging because children with CF tend to be healthier than adults and significant improvements in lung function may be harder to demonstrate in a clinical study,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “Data from Phase 3 studies of VX-770 in people with the G551D mutation provide strong support for the concept that addressing the defective protein that leads to CF may significantly improve outcomes for patients and give hope for the future of other CF treatments that repair the basic defect of the disease.” Vertex's medicines in development for CF were discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) to discover and develop novel CF Transmembrane Conductance Regulator (CFTR) modulators. CFFT is the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. Vertex retains worldwide rights to develop and commercialize these potential medicines. Summary of Key Data from ENVISION In the ENVISION study, 52 children were enrolled and received VX-770 or placebo as a single 150 mg tablet every 12 hours. At the time of this analysis, the last patients had completed their week 40 visit. All children who remain on this study will be followed through 48 weeks. All children who complete 48 weeks of treatment in ENVISION, including those in the placebo group, and who meet eligibility criteria, may choose to receive VX-770 for up to an additional 96 weeks, or until VX-770 is approved, as part of an extension study called PERSIST. ENVISION was designed to evaluate VX-770 in children between the ages of 6 and 11 with at least one copy of the G551D CFTR mutation. The primary endpoint of the study was mean absolute change from baseline in percent predicted FEV1 through week 24. Lung function was assessed using a standard test that measures the amount of air a person can exhale in one second (forced expiratory volume in one second, or FEV1).