What is the purpose of evaluating VX-770 and VX-809 as a combination therapy?
The clinical trial is testing the drugs in people who have two copies of the most common mutation of CF, known as Delta F508. The Delta F508 mutation leads to a CFTR protein that does not fold correctly. Because the defective CFTR is not the right shape, it does not make it to the surface of the cell. Research in the laboratory suggests that using more than one drug may work better on the defective CFTR protein than using one drug alone. VX-809 is designed to move the defective CFTR protein to the cell surface. VX-770 is designed to allow CFTR located at the cell surface to function better, and to improve the flow of salt and fluids in and out of the cell. The trial is designed to evaluate the safety and tolerability of VX-809 in combination with VX-770. The trial is also assessing the effect of VX-809 alone and in combination with VX-770 on CFTR function, as measured by sweat chloride levels.
