Final results from Vertex KALYDECO second of two Phase 3 studies on CF

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced final results from the ENVISION study, the second of two pivotal Phase 3 studies of KALYDECO™ (ivacaftor, VX-770), a medicine in development that targets the defective protein that causes cystic fibrosis (CF). ENVISION>CFTR) gene. These results, as well as the first data from PERSIST, the long-term follow-up study of adolescents and adults treated in the Phase 3 STRIVE study, are being presented at the 25th Annual North American CF Conference (NACFC), November 3-5, 2011 in Anaheim, Calif.

Results from ENVISION showed that children who received KALYDECO (kuh-LYE-deh-koh) experienced rapid and sustained improvements in lung function (forced expiratory volume in one second, FEV1) and other key measures of disease, including weight gain and a reduction in sweat chloride, throughout the 48-week study compared to those treated with a placebo. Patients in the study who received KALYDECO experienced a mean absolute improvement from baseline in lung function of 12.5 percentage points through week 24 (primary study endpoint) and a mean relative improvement from baseline in lung function of 17.4 percent compared to placebo. Through 48 weeks, the mean absolute improvement in lung function for children treated with KALYDECO was 10 percentage points compared to placebo and the relative mean improvement was 15.1 percent from baseline compared to placebo. Phase 3 results and product labeling for currently available CF medicines generally describe relative improvements in lung function. The most commonly reported adverse events were respiratory in nature and comparable across treatment groups.

"The longer people live with CF, the greater the risk of damage and complications from the disease," said Richard Ahrens, M.D., Co-Director, Cystic Fibrosis Center University of Iowa Children's Hospital and lead investigator of the ENVISION study. "Given this progressive nature of CF, the earlier we can treat patients, the better. These data are encouraging because they confirmed the improvements in lung function, weight gain and sweat chloride reduction we saw among adults who were treated with KALYDECO in the STRIVE study."

"The rapid, significant and sustained response to treatment with KALYDECO offers hope of improved outcomes for people with CF who have the G551D mutation," said Chris Wright, M.D., Ph.D., Senior Vice President and Head of Global Medicines Development and Affairs for Vertex. "We're moving quickly to evaluate other targeted approaches to CFTR modulation for people with the most common form of CF."

Adverse events that were more common (≥5 percent) among those treated with KALYDECO compared to placebo were throat pain, headache, upper respiratory tract infections, ear infections, diarrhea and an increase in eosinophil count. Adverse events that were more common among children in the placebo group were cough, vomiting, productive cough, rales (noisy breathing) and a decrease in pulmonary function. Pulmonary exacerbations were uncommon in ENVISION regardless of treatment arm. There were no discontinuations due to adverse events in the KALYDECO treatment group and one discontinuation in the placebo group through 48 weeks.

Source: Vertex Pharmaceuticals Incorporated

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