6 November 2017 - - The New England Journal of Medicine (NEJM) has published two articles with results from Boston, Massachusetts-based serious disease therapeutics developer Vertex Pharmaceuticals Inc.'s (NASDAQ: VRTX) two Phase 3 studies of the tezacaftor/ivacaftor combination treatment, a medicine in development to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have certain mutations in the cystic fibrosis transmembrane conductance regulator gene, the company said.
In both studies, the tezacaftor/ivacaftor combination demonstrated statistically significant and clinically meaningful improvements in lung function (ppFEV1) and other measures of disease. Data also revealed a favorable safety profile across multiple patient groups.
The 24-week EVOLVE study evaluated tezacaftor/ivacaftor in more than 500 people with CF ages 12 and older who have two copies of the F508del mutation.
Improvements across multiple disease measures, including lung function, were demonstrated among patients treated with tezacaftor/ivacaftor compared to those who received placebo. There was also a reduction in pulmonary exacerbations among those treated with tezacaftor/ivacaftor.
The EXPAND study evaluated eight weeks of treatment with the tezacaftor/ivacaftor combination, ivacaftor monotherapy or placebo in approximately 250 people with CF ages 12 and older who have one F508del mutation and one mutation that results in residual CFTR function.
Improvements across multiple disease measures, including lung function, were demonstrated among patients treated with tezacaftor/ivacaftor and those treated with ivacaftor alone in the study compared to those who received placebo.
Kalydeco (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene.
Known as a CFTR potentiator, Kalydeco is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.
Vertex invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.
The company has R and D sites and commercial offices in the United States, Europe, Canada, and Australia.