VX-809 Research Update

This week, we gained important new ground in our work to advance the development of new cystic fibrosis treatments.

VX-809, a potential CF drug developed by Vertex Pharmaceuticals, showed encouraging results in a preliminary Phase 2a clinical trial. VX-809 is one of the first investigational drugs aimed at treating the underlying causes of CF.

The oral therapy was tested in patient volunteers who have the most common cystic fibrosis mutation, Delta F508.

Results from the trial showed VX-809 was well-tolerated and, in a subset of CF patients, reduced sweat chloride levels — a key indicator of CF.

VX-809 is the direct result of one of the Foundation’s largest investments in drug development. This gives us confidence that our innovative approach to science leads to progress.

The data from this trial pave the way for future studies of VX-809, including testing the therapy in combination with VX-770. Also developed by Vertex, VX-770 is an oral investigational drug that showed encouraging Phase 2 results in restoring the function of CFTR in patients with the G551D mutation of CF.

Research in the laboratory suggests that using two therapies in combination may increase CFTR function in cells with the Delta F508 mutation when compared to using a single therapy alone.

To read the full article (press release) or for more information on this study, click here.