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July 2012

Medical News Briefs: Targeting the Basic Defect

Connections - July 2012 - Medical News Briefs

A top priority of the CF Foundation is accelerating the discovery and development of potential therapies to treat the underlying cause of CF. Here’s an update on the CF Foundation’s collaborations with leading pharmaceutical companies to advance the progress of drugs already in the pipeline and speed the development of additional new therapies.

Promising Final Results Released from Phase 2 Study of Kalydeco and VX-809 Combined Treatment

In June, Vertex Pharmaceuticals Inc. announced positive results from a Phase 2 study of its CF drug Kalydeco™ in combination with another potential therapy, VX-809.

The results showed a significant improvement in lung function in people with two copies of the most common CF mutation, Delta F508, who received the combination treatment.

Based on the data, Vertex plans to begin a pivotal trial of Kalydeco and VX-809 in people with two copies of the Delta F508 mutation in 2013. Pivotal trials typically gather data that could be used by the U.S. Food and Drug Administration (FDA) to decide whether to approve a potential drug.

Both Kalydeco and VX-809 are designed to treat the underlying cause of CF, a defective gene and its protein product, called CFTR. Earlier this year the FDA approved Kalydeco when taken alone for people with the G551D mutation of CF ages 6 and older.

Vertex plans to conduct additional studies of VX-809 and Kalydeco in patients with one copy of the Delta F508 mutation. The company is also studying Kalydeco in combination with another potential drug, VX-661. A Phase 2 clinical trial of Kalydeco and VX-661 is now enrolling people with two copies of the Delta F508 mutation ages 18 and older.


Kalydeco Follow-Up Study Results Show Sustained Improvements in Key Areas

In June, Vertex Pharmaceuticals announced data from a follow-up study of Kalydeco in people with the G551D mutation of CF who had completed earlier Phase 3 trials of the drug.

The results showed that people who took Kalydeco demonstrated improvements in lung function, respiratory symptoms and weight gain for up to 96 weeks.

The extended access study enrolled people who had received a placebo as well as those who had taken Kalydeco during the earlier Phase 3 trials.

In addition, the European Medicines Agency (EMA) announced in May that it had recommended approval of Kalydeco for people with the G551D mutation of CF ages 6 and older in the European Union.

The EMA sent the recommendation to the European Commission, which has the authority to approve medicines in the European Union. The European Commission generally follows the EMA’s recommendation and typically decides whether to approve a drug within 3 to 4 months.


PTC Therapeutics Announces Results from Phase 3 Study of Ataluren

On June 8, PTC Therapeutics Inc. announced results from a large, international Phase 3 clinical trial of the potential CF drug ataluren in people ages 6 and older who have what are known as “nonsense mutations.” 

The results showed that people who took ataluren had a lower decline in lung function and a lower rate of pulmonary exacerbations, compared with those who received a placebo.

About 10 percent of people with CF have nonsense mutations, which interrupt the production of the CFTR protein, causing it to be too short and not function normally. Ataluren is designed to enable the production of a full-length, fully functional CFTR protein.  

PTC Therapeutics said it will be discussing the results with its clinical researchers and with advocacy groups to determine the next steps.


New Drug Discovery Collaboration Aims to Address Most Common CF Mutation

The CF Foundation has entered into a collaboration with Proteostasis Therapeutics Inc. (PTI) to find new compounds that could potentially be used to treat people with the most common CF mutation, Delta F508.

The one-year pilot program will focus on identifying small molecule compounds that could help the defective CFTR protein fold correctly and move to the surface of the cell.

In people with the Delta F508 mutation of CF, the protein does not fold into the right shape to reach the cell surface, where it is needed to form a channel that allows the proper flow of salt and fluids on to the surface of the lungs.

“PTI brings a unique technology to our ongoing efforts to identify potential new compounds to treat the most common mutation of CF,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “This new collaboration is part of our strategy to pursue multiple approaches to speed the discovery and development of effective therapies for all people living with this disease.”

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