Vertex to Present Data Demonstrating Significant Benefits of Long-Term and Early Treatment With CFTR Modulators at the European Cystic Fibrosis Conference

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that five scientific abstracts on the company’s portfolio of cystic fibrosis (CF) medicines will be presented at the European Cystic Fibrosis Society's (ECFS) 45th European Cystic Fibrosis Conference held June 8-11, 2022, in Rotterdam, the Netherlands.

Vertex will present the first analysis of data collected in the U.S. CF Foundation Patient Registry (CFFPR) of over 16,000 people with CF treated with TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for an average of nine months. This first interim analysis of an ongoing five-year post-authorization study (abstract WS22.05) showed that real-world treatment with TRIKAFTA was associated with improved lung function and a 77% reduced risk of pulmonary exacerbations compared to pre-TRIKAFTA baseline, as well as an 87% lower risk of lung transplant and a 74% lower risk of death, compared to the historical 2019 U.S. CFFPR population. No new safety concerns were identified.

Vertex will also present data comparing the annual rate of lung function change in people with CF ages 12 years and older with two F508del mutations (F/F) or one F508del mutation and one minimal function mutation (F/MF) treated with TRIKAFTA in pivotal studies and an open-label extension study compared to propensity-score matched historical CFTR-modulator-untreated controls from the U.S. CFFPR (abstract WS22.04). Results show that TRIKAFTA demonstrated on average no decrease in ppFEV₁ over a two-year period in this population, in contrast to declines seen in the matched controls. The analysis indicates that treatment with TRIKAFTA has a significant impact on the trajectory of CF lung disease.

Additionally, Vertex will present data from a long-term real-world study demonstrating that initiating KALYDECO (ivacaftor) early in life (ages 6-10 years) preserves lung function to a greater extent than if KALYDECO is initiated at an older age (abstract WS17.03). These results show the importance of early initiation of KALYDECO for eligible patients.

“These long-term and real-world studies show the potentially transformative benefits of treatment with CFTR modulators and add to the substantial body of evidence supporting treatment as early in life as possible,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “We continue to make rapid progress in developing medicines that treat the underlying cause of CF, and today, we are closer to our goal of developing highly effective therapies for all patients with CF than ever before.”