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July 2011

News Brief: Targeting the Basic Defect 

News Brief: Targeting the Basic Defect - July 2011 - Connections - CF Foundation 
For more than a decade, the Cystic Fibrosis Foundation has collaborated with Vertex Pharmaceuticals, Inc., to discover and develop potential therapies aimed at repairing the defective CFTR protein that causes CF.

Here’s an update on the Foundation’s ongoing collaboration with Vertex and the progress of potential new treatments for CF.  

Promising New CF Therapy on Track for FDA Review

In June, Vertex announced the final results of a Phase 3 trial of VX-770 in people age 12 and older who carry at least one copy of the G551D mutation.

Participants who received VX-770 showed rapid and significant improvements in lung function, weight gain and sweat chloride levels, and they maintained these benefits throughout the nearly yearlong trial.

Vertex also announced results from a Phase 3 study of VX-770 in children age 6 to 11 with the G551D mutation. Data from the trial showed that children who took the drug had improvements in lung function and other key symptoms of CF similar to those seen in the adult trial.

The next step for VX-770 is the submission of a new drug application to the U.S. Food and Drug Administration (FDA) later this year, with anticipated approval in mid-2012. Generally, it takes between 6 and 12 months for the FDA to review and rule on a drug application.

Vertex is setting up a program to provide VX-770 to people with the G551D mutation of CF who are in critical medical need and may benefit from the treatment prior to potential FDA approval of the drug.

The expanded access program will take place at clinical sites in the United States and is designed for people with CF age 6 and older who have highly limited lung function and meet other criteria.

To learn more, speak with your CF doctor or call the VX-770 Expanded Access Call Center at 1-800-745-4484.

The Bottom Line: VX-770 represents a major step in the fight against CF.

Read more in these FAQs About VX-770.

First Phase 2 Study of Combination Treatment Shows Positive Results

The CF Foundation and Vertex recently announced promising results from the first part of an ongoing Phase 2 clinical trial of VX-770 in combination with another potential CF therapy, VX-809.

The combination therapy was tested in 62 people with two copies of the most common CF mutation, Delta F508, and lasted three weeks.

Participants who took the drug regimen showed reduced sweat chloride levels. Excessive sweat chloride is a key symptom of CF. The results suggest that VX-770 and VX-809 taken together may help the defective CFTR protein work the way it should.

In people with the Delta F508 mutation, the malformed CFTR protein does not move to its proper place at the cell surface. VX-809, known as a “corrector,” is designed to move CFTR to the cell surface. VX-770, called a “potentiator,” aims to improve the function of the defective protein once it is there.

The study is the first to examine the two drugs taken together to treat the basic defect in CF, and data from these initial results will provide important information for future studies of potential combination treatments.

The Bottom Line: Vertex plans to start the second part of the Phase 2 VX-770 and VX-809 clinical trial later this year.

Learn more about the combined VX-770 and VX-809 clinical trial in these FAQs.

News Brief: Targeting the Basic Defect - July 2011 - Connections - CF Foundation


Collaboration Aims to Speed Up Development of Therapies Targeting Most Common CF Mutation

This spring the CF Foundation announced that it is expanding its collaboration with Vertex to accelerate the development of additional drugs targeting the underlying cause of CF in people with the Delta F508 mutation.

The new program will support development of VX-661, which, like VX-809, is called a corrector and helps move the CFTR protein to the cell surface. Vertex expects to begin a Phase 2 study of VX-661 in people with the Delta F508 mutation by the end of this year.

The Foundation’s investment of up to $75 million over five years will also help support the discovery and early development of other new correctors. By developing multiple potential drugs, the expanded collaboration with Vertex will increase the possibility of bringing new therapies more quickly to those with the most common CF mutation. 

The Bottom Line: Work is under way to discover additional drugs targeting the basic defect — and the Foundation has committed to expanding its support for this vital research.

Read the news story about the Foundation’s new collaboration with Vertex.

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