Vertex Pharmaceuticals Incorporated and Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) 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 non-profit 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 CEO 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 III 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, PhD, 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.

The collaboration is focused on development activities for VX-661 and on the accelerated discovery and development of additional correctors for the treatment of people with the F508del mutation. As part of the collaboration, CFFT will provide Vertex with up to $75 million to support research and development activities. Vertex expects to receive payments from CFFT as reimbursement for research and development activities over a period of five years beginning in 2011.

The collaboration will help support the development of VX-661. Vertex intends to begin a phase II study of VX-661 by the end of 2011 and expects the study to enroll people with CF who have the F508del mutation. It will also help support the accelerated discovery and early development of next-generation correctors that aim to treat the underlying cause of CF in people with the F508del mutation.

Under the terms of the collaboration, CFFT is entitled to receive a royalty on future net sales of correctors developed as part of the research collaboration. As part of previous collaborations, CFFT is entitled to receive a royalty on future net sales of VX-770, VX-809 and VX-661. Vertex retains worldwide rights to VX-770, VX-809 and VX-661.

Vertex recently reported positive results from two phase III studies of VX-770 — the STRIVE trial in adolescents and adults with CF and the ENVISION trial in children ages 6 to 11. The phase III programme for VX-770 is focused on people with CF who have at least one copy of the G551D mutation. The results from the phase III programme will form the basis for Vertex's planned submission of applications for approval in the United States and Europe in the second half of 2011.

Vertex is also conducting a phase II clinical trial to evaluate different dose combinations of VX-809 and VX-770 in people with two copies of the F508del mutation. The first part of the study is designed to evaluate VX-809, or placebo, dosed alone for 14 days and in combination with VX-770, or placebo, for seven days. Vertex expects to obtain data from Part One of the trial in the first half of 2011.

VX-661 is the third potential new medicine for CF to emerge from Vertex's CF research efforts. As a corrector, VX-661 aims to increase CFTR function by increasing the movement of CFTR to the cell surface. In in vitro studies, a combination of VX-661 and VX-770 resulted in greater CFTR activity, as compared to treatment with VX-661 alone. Vertex plans to initiate a phase II study of VX-661 by the end of 2011.

CF is a life-threatening genetic disease affecting approximately 30,000 people in the United States and 70,000 people worldwide. Today, the median predicted age of survival for a person with CF is approximately 36 years. According to the 2008 Cystic Fibrosis Foundation Patient Registry Annual Data Report, approximately 4 percent of the total CF patient population in the United States have at least one copy of the G551D mutation, 48 percent of the total CF patient population in the United States have two copies of the F508del mutation and an additional 39 percent of the total CF patient population have one copy of the F508del mutation.

Vertex initiated its CF research programme in 1998 as a part of a collaboration with CFFT, the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation. Vertex and CFFT expanded the agreement in 2000 and again in 2004, and in March 2006 entered into a collaboration for the accelerated development of VX-770. In addition to the development collaboration for VX-770, in January 2006 Vertex and CFFT entered into an expanded research collaboration to develop novel corrector compounds. As part of these collaborations, Vertex has received approximately $75 million from CFFT to date to support CF research and development efforts led by Vertex.

The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization and nearly every CF drug available today was made possible because of Foundation support.

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