Vertex Pharma Offers Data Presentations at ECFS Conference: TRAFFIC, TRANSPORT Extension Study of Lumacaftor Im Combo with Ivacaftor Showed Improvements

Vertex Pharmaceuticals Incorporated

today announced data from PROGRESS, the long-term extension study of the investigational regimen ORKAMBI™ (lumacaftor/ivacaftor). The ongoing extension study enrolled people with cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation and completed 24 weeks of treatment in the Phase 3 TRAFFIC and TRANSPORT studies (lumacaftor/ivacaftor combination and placebo treatment groups) and met certain other eligibility criteria. These data are included in one of nine presentations at the 38th European Cystic Fibrosis Society (ECFS) Conference, June 10-12, 2015, in Brussels, Belgium. Lumacaftor in combination with ivacaftor in patients with cystic fibrosis who are homozygous for the F508del-CFTR mutation (ECFS Abstract WS01.3, oral presentation during Workshop 1 - Strategies to correct CFTR defects.) One thousand thirty-one people who completed 24 weeks of treatment in either of the Phase 3 studies, TRAFFIC or TRANSPORT, entered the 96-week PROGRESS Phase 3 extension study in which everyone received one of two lumacaftor/ivacaftor combination regimens. An interim analysis was conducted once all patients completed 24 weeks in PROGRESS for a total of 48 total weeks of treatment (48 weeks of treatment with a combination regimen for patients who received a combination regimen in TRAFFIC and TRANSPORT; 24 weeks of treatment with a combination regimen for patients who received placebo in TRAFFIC and TRANSPORT). These data showed that the initial improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) observed in the 24-week TRAFFIC and TRANSPORT studies among those treated with a lumacaftor/ivacaftor combination were sustained through 48 weeks of treatment across all patients. Reduced rates of pulmonary exacerbations and improvements in body mass index (BMI) and patient-reported respiratory symptoms as measured by the respiratory domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) were also maintained over 48 weeks. In addition, the pattern and magnitude of response observed after the initiation of combination treatment across all patients who received placebo in TRAFFIC and TRANSPORT and subsequently received a combination regimen in PROGRESS were similar to those seen among patients who received a combination regimen in TRAFFIC and TRANSPORT. At the time of this analysis, the safety and tolerability results, including the type and frequency of adverse events and serious adverse events, were consistent with those observed in TRAFFIC and TRANSPORT, and no new safety concerns were identified. Over 48 weeks, the most common adverse events were infective pulmonary exacerbation, cough and increased sputum. The incidence of serious adverse events during PROGRESS was generally similar to TRAFFIC and TRANSPORT. Other data presented at the Conference include: "Lumacaftor/ivacaftor combination therapy in CF patients homozygous for F508del-CFTR with severe lung dysfunction." Poster 143. "VX-661 in combination with ivacaftor in patients with cystic fibrosis and the F508del-CFTR mutation." ECFS Abstract WS01.4, oral presentation during Workshop 1 - Strategies to correct CFTR defects. "R117H-CFTR has a defect in channel gating activity that can be potentiated by ivacaftor." ECFS Abstract WS06.2, oral presentation during Workshop 6 – Fixing ion transport. "An open-label study of the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2 to 5 years with cystic fibrosis and a CFTR gating mutation: the KIWI study." ECFS Abstract WS01.5, oral presentation during Workshop 1 - Strategies to correct CFTR defects. "Prevalence of cataracts in a population of cystic fibrosis patients homozygous for the F508del mutation." Poster 196. "Manifestation and progression of illness in young children with cystic fibrosis: a targeted literature review." Poster 186. "Frequency and costs of pulmonary exacerbations and association with % predicted FEV1 in patients with cystic fibrosis." Poster 190. "Lung function and health care resource utilization in patients with cystic fibrosis." Poster 191.