Vertex Pharmaceuticals Announces Interim Phase 2 Data Showed a Combination of VX-770 and VX-809 Improved Function of the Defective Protein that Causes Cystic Fibrosis in People

Vertex Pharmaceuticals Incorporated VRTX today announced interim results from the first part of a Phase 2 study designed to evaluate multiple combination regimens of VX-770 and VX-809, Vertex's lead medicines in development that aim to treat the defective protein that causes cystic fibrosis. That protein, known as the cystic fibrosis transmembrane conductance regulator (CFTR), is responsible for regulating the flow of chloride across the cell surface to help hydrate and clear mucus from the airways. The first part of the study enrolled and randomized 62 people with two copies of the most common mutation in the CF gene, known as the F508del mutation. In people with the F508del mutation, the CFTR protein does not reach the cell surface in normal amounts, resulting in a buildup of mucus and other complications that can lead to lung damage. VX-809, known as a CFTR corrector, is designed to help the protein reach the cell surface, while VX-770, known as a CFTR potentiator, aims to help the protein function more normally once it reaches the cell surface. VX-770 and VX-809 were advanced into development with support from Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation. Vertex retains worldwide rights to develop and commercialize these potential medicines.