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Vertex Achieves Breakthrough In Treating Basic CF Defect


In April, the CF Foundation announced an unprecedented milestone in its effort to treat the basic defect in cystic fibrosis.  Historic results from an ongoing Phase 2 clinical trial of VX-770, a potential therapy being developed by Vertex Pharmaceuticals, Inc., suggest that the compound improves the function of the faulty CFTR protein.  VX-770 is the result of a collaboration that began in 1998 with Vertex Pharmaceuticals and is the largest in the Foundation's history.

The interim data from the Phase 2a study performed in a subset of CF patients indicate that VX-770 improves lung function, sweat chloride levels and nasal potential difference. The first compound of its kind, VX-770 belongs to a category of compounds termed potentiators, which aim to improve the opening of the CFTR chloride channel that is defective in CF.  It is being studied in patients who carry the G551D mutation.

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It's unprecedented for an investigational oral compound for the treatment of CF to have such a marked effect on multiple measures of CF disease activity."


Frank Accurso, M.D.
CF Center Director
University of Colorado

"The results from this trial are among the most important announcements in the history of the Foundation," said Robert J. Beall, Ph.D., president and CEO of the Foundation. "Although we're still early in the process, we have increased confidence that we can treat the basic defect of CF, and these data show that we are on the right path in our quest to cure this disease."

The study, the first part of two in the Phase 2a trial, tracked 20 CF patients, 16 of whom took VX-770 over a 14-day period. Part two of the trial, which is expected to enroll 16 patients, will begin in the second quarter of 2008. Additional studies will evaluate the longer-term safety and efficacy of the compound. The trial focused on a subset of patients with the G551D mutation of CF, approximately four percent of the CF patient population in the U.S.  VX-770 may benefit those with other mutations that result in a malfunctioning CFTR protein; however, further study is necessary to determine how many CF patients could benefit from this compound.

did you know?

The CF Foundation's collaboration with Vertex Pharmaceuticals has been chosen as the subject of a Harvard University Business School case study on venture philanthropy to be used by business students at Harvard and across the world. A second Harvard case study of our decision-making process is also underway.

"Even at this early stage of clinical development, it's unprecedented for an investigational oral compound for the treatment of CF to have such a marked effect on multiple measures of CF disease activity," said Frank Accurso, M.D., director of the Cystic Fibrosis Center and professor of pediatrics at the University of Colorado School of Medicine in Denver. In fact, the Phase 2a trial of VX-770 is the first time that any potential therapy has improved the abnormal sweat chloride levels in a person with CF.

All the therapies currently on the market for CF address the symptoms of the disease, but now it appears that we can target the faulty CFTR protein and possibly restore its function. These early findings could have important implications for studies of other drugs in development.

In addition to VX-770, a second Vertex compound known as VX-809 began clinical testing early this year in healthy volunteers. Vertex plans to study the safety of VX-809 in people with CF by mid-2008.  VX-809 belongs to a category of compounds known as correctors, which may allow the defective CF protein to move to its proper place in the cell, where it is then poised to move chloride out of the cell.  

In 1998, the Foundation made an initial investment to use cutting-edge technology — known as high-throughput screening — to find compounds that address the core defect in CF. Since then, the Foundation has invested $76 million in the project, and the two organizations have closely collaborated to advance both VX-770 and VX-809.

If the clinical development of these compounds is successful, VX-770 and VX-809 could potentially add decades of life for those with CF.


 

"The project carried significant risk that would have dissuaded many companies and investors from moving forward," said Joshua Boger, Ph.D., president and CEO of Vertex Pharmaceuticals. "Today, however, with the Foundation's financial, scientific and clinical network support, we have been able to bring two novel CF compounds into development and are encouraged by these early results for VX-770."

If the clinical development of these compounds is successful, VX-770 and VX-809 could potentially add decades of life for those with CF.