Vertex Shows TRIKAFTA® Benefits at European Cystic Fibrosis Conference

Vertex Pharmaceuticals (Nasdaq: VRTX) has unveiled new data on their cystic fibrosis (CF) treatment, TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), known in Europe and the U.K. as KAFTRIO® in combination with ivacaftor, at the European Cystic Fibrosis Society’s (ECFS) 47th European Cystic Fibrosis Conference in Glasgow, Scotland. The findings, derived from a series of studies, highlight significant and clinically meaningful improvements in various health parameters for individuals with cystic fibrosis, particularly those with rare CFTR mutations and non-F508del CFTR mutations.

Data from a randomized, placebo-controlled, Phase 3 trial (abstract WS06.04) indicated that TRIKAFTA® produces significant clinical benefits for CF patients with rare, non-F508del CFTR mutations. Key improvements included a 9.2 percentage point increase in lung function (measured by ppFEV1), a reduction in sweat chloride concentration by 28.3 mmol/L, and a 72% decrease in annual pulmonary exacerbations. The safety and tolerability profiles were consistent with the known safety data for TRIKAFTA®.

An interim analysis of a large-scale, real-world study (abstract WS01.04) also provided compelling evidence of TRIKAFTA®'s impact. This five-year study, which includes over 19,000 CF patients from the U.S. and Germany, revealed significant reductions in severe health events. The study demonstrated a 76% and 70% reduction in pulmonary exacerbations in the U.S. and Germany, respectively, compared to the pre-treatment period. Furthermore, death rates dropped by 62% in the U.S. and 84% in Germany, while lung transplant rates fell by 86% in the U.S. and 96% in Germany. Notably, no new safety concerns emerged from this large cohort.

Dr. Carmen Bozic, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex, emphasized the transformative impact of TRIKAFTA®, stating, "These studies demonstrate that TRIKAFTA is changing the course of CF treatment and the lives of those living with CF."

Additional presentations at the conference included real-world effectiveness data and long-term observational studies on TRIKAFTA® in various subpopulations of CF patients, highlighting the broad and sustained benefits of the treatment across different demographics and geographies.

Cystic fibrosis is a life-shortening genetic disorder that affects multiple organs, including the lungs, liver, pancreas, and gastrointestinal tract. It results from mutations in the CFTR gene, leading to defective or insufficient CFTR protein, which causes the build-up of thick, sticky mucus in the organs. This mucus accumulation can lead to severe respiratory and digestive problems. The median age of death for CF patients is in the 30s, but advancements in treatment, such as those seen with TRIKAFTA®, are improving survival rates.

TRIKAFTA® works by enhancing the amount and function of CFTR protein at the cell surface, thereby improving salt and water flow in and out of cells. This action helps to reduce mucus build-up in the lungs and other organs. The medication is indicated for CF patients aged 2 years and older who have at least one F508del mutation or another responsive mutation.

Vertex Pharmaceuticals, the developer of TRIKAFTA®, continues to innovate in the field of genetic diseases. The company has a robust pipeline of investigational therapies targeting various serious conditions, including sickle cell disease, transfusion-dependent beta thalassemia, and multiple kidney diseases.

With a global presence and a commitment to scientific excellence, Vertex has established itself as a leader in biotechnology, dedicated to transforming the lives of patients with serious diseases through groundbreaking therapies like TRIKAFTA®. The company's consistent recognition as a top employer underscores its commitment to fostering a dynamic and innovative work environment.

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