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May 2011

CF Drug Development Takes a Giant Step Forward 

Dr. Robert J. Beall, President and CEO
Robert J. Beall, Ph.D., President and CEO of the Foundation, discusses VX-770 clinical trial results.


Earlier this year, the Cystic Fibrosis Foundation announced remarkable results of a late-stage clinical trial of VX-770, a potential therapy targeting the basic defect in CF.

The Phase 3 findings demonstrated that a drug can improve the health of people with CF by addressing the underlying cause of CF — a faulty gene and its protein product, called CFTR.

The study examined VX-770 in people age 12 and older who carry the G551D mutation of CF. Those who received VX-770 showed profound improvements — especially in lung function and weight gain.

VX-770 also is being studied in an ongoing trial of children age 6 to 11 with the G551D mutation. Results from the first half of that study showed similar improvements in key symptoms among young children who took the drug.

VX-770 is being developed by Vertex Pharmaceuticals, Inc., and was discovered in collaboration with the CF Foundation. Since 1999, the Foundation has provided significant support to Vertex to develop therapies using small molecules in the hope of repairing the defective CFTR protein.

Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation, spoke with Connections about this exciting news and the Foundation’s expanded collaboration to accelerate the discovery and development of additional drugs that aim to treat the basic defect.

What was your reaction when you heard the results of this VX-770 clinical trial?

You know the song “I Feel Good” by James Brown? That pretty much sums it up. I was thrilled! The results met and even exceeded what we had hoped for when we began working with Vertex on these therapies 12 years ago.

Can you tell us about the findings?

During the trial of patients age 12 and older, those who received VX-770 showed amazing improvements in a number of key areas, compared with those who received the placebo, a pill with no active medication in it.

Let me tell you about three improvements of real significance. 

Lung function: On average, those receiving VX-770 demonstrated the highest increase on a lung function test that we’ve seen in any clinical trial of a CF drug.

Sweat chloride: Among those taking the drug, sweat chloride levels dropped toward normal. This is a sign that the drug was having an effect on the underlying cause of the disease, since excessive sweat chloride is a key indicator of CF.

And last, but in no way less significant, weight gain: Patients on VX-770 gained nearly 7 pounds on average.

Plus, they reported that they felt better!

Why are the findings so important?

These results are even more important than the discovery of the CF gene in 1989. The gene discovery significantly advanced our understanding of the problem in CF. Now we see, for the first time, that a drug can target the basic genetic defect in CF and improve the most harmful aspects of the disease.

We are more optimistic than ever that if we continue to focus our collective efforts on discovering and developing compounds that target the basic defect, we will be better positioned to bring new therapies to all people with CF.

What is the next step for VX-770?

Vertex plans to apply for U.S. Food and Drug Administration (FDA) approval for VX-770 in the second half of this year, with the hope of approval in mid-2012.

VX-770 also is currently being tested in combination with another Vertex drug, VX-809, in people with two copies of the most common mutation in CF, Delta F508. Researchers believe that using these therapies together may be more beneficial than either drug on its own. We expect to see the results later this year.

If VX-770 is approved, what will it mean for patients?

If approved, VX-770 used alone will impact a small group of CF patients. But this is a historic step toward actually curing and controlling this disease.

Should the CF community contact the FDA to push for approval of VX-770?

No. We are grateful for our many volunteers who advocate on behalf of the CF community. However, outreach from patients and families has not been effective in the past in speeding up the FDA review and approval process.

We’re optimistic that the FDA will review the new drug application for VX-770 without delay.

One thing members of our community can do is to contact their members of Congress and ask them to provide the FDA with the resources the agency needs to review potential drugs like VX-770 quickly.

What is being done to create other drugs targeting the basic defect that might help more people?

I’m very excited that we recently expanded our collaboration with Vertex to speed up the discovery and development of drugs aimed at treating the underlying cause of CF. The Foundation’s investment — up to $75 million over five years — will support the development of VX-661, which is aimed at treating people with the most common mutation of CF, Delta F508, along with other therapies that could benefit a wide variety of people who have the disease.

We are incredibly encouraged by the VX-770 findings and also very inspired: these results confirm that our drug development strategy is on the right track. The Foundation will continue to advance combination trials, including the trial of VX-770 and VX-809 that is under way now.

And, we will push ahead aggressively to put in place the “next generation” of therapies targeting the basic defect to ensure that the hope offered by the VX-770 results becomes a possibility for all people with CF.

To do this we will need to raise more money than ever. We will continue to be tireless in our efforts and will not rest until we find a cure.

What made this breakthrough possible?

We would never have reached this pivotal point without the dedication and generosity of our extraordinary community — people with CF, families, scientists, researchers, donors, friends, volunteers and staff — who have selflessly given their time, money and talent to support our mission. With their help, I know we’ll reach our goal.

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