A Direct Approach to CF Therapy

Medical advances today allow cystic fibrosis (CF) patients to live longer, healthier and more productive lives than ever before. But it´s not easy. CF patients often require more than three hours of treatment every day. And with the median predicted lifespan of a CF patient at just 37 years, more effective therapies are greatly needed.

CF is a genetic disease affecting approximately 30,000 people in the U.S. and 70,000 worldwide. Patients with CF have defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) proteins at their cell surfaces, causing poor chloride ion flow across cell membranes. The result? A buildup of mucus in the lungs causing repeated cycles of infection and inflammation. CF ultimately robs patients of their lung function and leads to a shortened lifespan for many. Although the CF gene was discovered in 1989, no currently approved treatment addresses the underlying defect that is responsible for the disease. Vertex hopes to change that equation.

Advancing Two Novel Approaches to CF Therapy

VX-770 is Vertex´s lead investigational compound for the treatment of CF. Known as a CFTR potentiator, VX-770 is designed to increase fluid transport across the cell surface by restoring the activity of defective CFTR proteins. And as an oral therapy, VX-770 may provide systemic benefit beyond solely the lungs of CF patients.

In 2008, the first clinical data for VX-770 showed that after 28 days of treatment, patients with the G551D CFTR mutation had a rapid and sustained improvement in lung function. Further, VX-770 showed a measurable change in the sweat chloride levels of CF patients in the trial compared to placebo, indicating the compound may have partially restored the function of the CFTR protein — a medical first. In 2009, VX-770 is expected to enter a registration program focused on CF patients with the G551D mutation.

Vertex is also developing VX-809, an investigational CFTR corrector for the treatment of CF patients with the most common CFTR mutation, known as F508del. VX-809 is designed to normalize fluid transport across the cell surface by increasing the concentration of CFTR proteins at the cell surface. Vertex completed Phase 1 studies of VX-809 in healthy volunteers and CF patients in 2008 and expects to initiate a Phase 2a clinical trial in CF patients with the F508del mutation in 2009.

Cystic Fibrosis Foundation Collaboration

Vertex initiated its CF research program in 1998 as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, a non-profit, donor-supported drug discovery and development affiliate of the Cystic Fibrosis Foundation. This unique collaboration resulted in the advancement of both VX-770 and VX-809 from research into clinical development.