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Sept/Oct 2009

New Drugs Aren't Made Overnight

On average, it takes ten years and costs between $800 million and $1.2 billion to develop a drug.

The CF Foundation helps reduce the time and cost involved in drug development by supporting biotech collaborators — and testing potential therapies in its network of CF care centers.

Drug development begins in the laboratory. New approaches to treatment are tested for several years—first in the test tube, and ultimately in people with the disease.

Once a drug enters clinical studies in people, it must pass through several phases of testing. Each phase of a clinical trial is designed to help researchers learn about the safety and effectiveness of a drug before it is made available to patients.

Typically, the U.S. Food and Drug Administration (FDA) will consider a drug for approval after three phases of study are complete. Once a drug has reached Phase 3 trials — the final stage of testing — the chances of it successfully becoming a drug available to patients are about 80 percent.

The Foundation is currently supporting the development of more than 30 potential therapies for CF. Several of these are potential drugs aimed at treating the underlying cause of the disease.

Here are three of the potential drugs aimed at treating the basic defect of CF that are in Phase 3 trials and closest to the finish line in the Foundation’s drug development pipeline.

The Potential Drug — VX-770

What does it do?

Cystic fibrosis occurs when the CF gene and its protein product, known as CFTR, do not work properly. Problems with the CFTR protein cause a salt imbalance, which leads to thick, sticky mucus buildup in the lungs and other organs in the body.

Vertex Pharmaceuticals, Inc. is developing two oral drugs designed to treat the underlying genetic cause of cystic fibrosis.

VX-770, the more advanced of the two compounds, is designed to restore function to the CFTR protein. In 2008, the CF Foundation and Vertex announced positive Phase 2 clinical trial results for patients who have the G551D gene mutation of CF.

What are the results so far?

Patient volunteers who participated in the trial showed remarkable improvements, including an unprecedented decrease in sweat chloride levels. Most important, scientists showed that it is possible to treat the root cause of CF.

In May, Phase 3 studies of VX-770 began, and trials are currently underway. The first Phase 3 trial is examining VX-770 in people with CF age 12 and older who have the G551D mutation of CF.

The second clinical trial began in August and is testing the potential drug in patients aged six to 11.

What’s next?

The third and final trial will, for the first time, evaluate VX-770 in patients who have two copies of the Delta F508 mutation of CF, the most common mutation in CF patients. This trial is expected to begin later this year.

The Potential Drug — Ataluren

What does it do?

Developed by PTC Therapeutics, Inc., ataluren (formally known as PTC124) is an oral therapy that targets a particular type of CF mutation known as a nonsense mutation.

In CF, a nonsense mutation interrupts the production of the CFTR protein. It causes CFTR to be too short and non-functioning. Overall, about 10 percent of people with CF have nonsense mutations.

What are the results so far?

Data from Phase 2 clinical trials of ataluren showed that CF patients who took the drug had improved function of the CFTR protein and a decrease in the frequency of cough. Ataluren was also associated with improved lung function.

What’s next?

PTC Therapeutics recently launched a Phase 3 trial of ataluren. The 48-week trial will study ataluren in people with CF age six and older. The main goals of the trial are to understand whether ataluren can improve lung function in people with CF and determine if the drug can be given safely for a long period.

Sixteen care centers in the Foundation’s Care Center Network are participating in the study. As research centers open, details will be listed on

The Potential Drug — Denufosol

What does it do?

Some therapies aim to treat the underlying cause of CF by repairing the faulty CFTR protein. Inspire Pharmaceuticals, Inc., on the other hand, has been working toward correcting the salt imbalance in the body not by addressing CFTR but by bypassing the protein and activating a different pathway to move salt in and out of cells. This method will help hydrate the mucus in the airways so that it can be flushed from the lungs much more efficiently.

What are the results so far?

A Phase 2 study found that people with CF who received the aerosolized drug three times a day for 28 days had significantly better lung function at the end of the trial than those who did not receive the drug.

In June 2008, Inspire announced top-line results from TIGER-1, its first Phase 3 trial with denufosol for CF. Later in the year Inspire launched TIGER-2, the second Phase 3 trial of denufosol.

What’s next?

TIGER-2 is currently enrolling CF patients age five and older in a 48-week study of the therapy.

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