CFFT, Vertex collaborate to develop new medicines for cystic fibrosis

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) today announced they will collaborate on the continued discovery and development of new medicines known as correctors that aim to treat the underlying cause of cystic fibrosis (CF) in people with the most common form of the disease. The expanded collaboration will support development activities for VX-661, Vertex's second corrector to enter clinical development, and the accelerated discovery and development of next-generation correctors. Vertex plans to begin the first study of VX-661 by the end of 2011 in people with CF who have the F508del mutation.

Vertex and CFFT, a nonprofit drug discovery and development affiliate of the CF Foundation, began their collaborative research and development efforts in 1998, and to date, three potential new medicines, known as CFTR modulators, have resulted from the collaboration - the potentiator VX-770 and the correctors VX-809 and VX-661. Correctors and potentiators are medicines in development that aim to treat the underlying cause of CF by improving the function of the defective protein known to cause the disease. Vertex retains worldwide rights to develop and commercialize these potential medicines.

"The CF Foundation is widely recognized by doctors, nurses, scientists and those with CF as a driving force in the search for new CF medicines, and we are pleased to further expand our strong collaboration with them," said Matthew Emmens, Chairman, President and Chief Executive Officer of Vertex. "The collaboration announced today underscores our commitment to CF and accelerates our efforts to develop new medicines as quickly as possible for people with the most common type of this disease. By advancing VX-809 and VX-661 in parallel, we hope to generate data to inform future studies of corrector regimens while continuing to invest in additional research for CF."

"With the recently announced positive Phase 3 results for VX-770, we believe that - together with Vertex - we are on the right path to fundamentally change the treatment of CF by targeting the cause of the disease," said Robert J. Beall, Ph.D., President and CEO of the CF Foundation and CFFT. "This new collaboration is a milestone in our long-standing relationship with Vertex and provides for additional opportunities to accelerate our discovery efforts and to potentially improve treatment for people with the most common type of CF."

CF is caused by defective or missing CF transmembrane conductance regulator (CFTR) proteins, which result in poor ion flow across cell membranes, including in the lungs, causing the accumulation of abnormally thick, sticky mucus that leads to chronic lung infections and progressive lung damage. In people with the F508del mutation, which is the most common CFTR mutation, CFTR proteins do not reach the cell surface in normal amounts. VX-809 and VX-661, known as CFTR correctors, aim to increase CFTR function by increasing the movement of CFTR to the cell surface. In people with the G551D mutation in the CFTR gene, CFTR proteins are present at the cell surface but do not function properly. VX-770, known as a CFTR potentiator, aims to increase the function of defective CFTR proteins by increasing their ability to transport ions across the cell membrane of CFTR at the cell surface.