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Trial Shows Cystic Fibrosis Drug Helped Ease Breathing

What follows is an excerpt from a recent article in the New York Times, dated February 23, 2011:

The Business of Health Care

By Andrew Pollack

The gene responsible for cystic fibrosis was discovered in 1989. Now, 22 years later, a drug that tries to compensate for the genetic defect might be nearing the market.

Vertex Pharmaceuticals announced Wednesday morning that the drug, VX-770, improved lung function in people with cystic fibrosis in a late-stage clinical trial. The drug also reduced the frequency of disease exacerbations that required treatment with antibiotics.

The caveat is that VX-770 is designed to counter one specific genetic mutation that accounts for about 4 percent of cases of cystic fibrosis. Vertex is working on another drug for the most common mutation, but that one is further behind in development.

Still, the news is expected to be greeted favorably by doctors and patients and by Wall Street.

“I’ve been doing clinical trials for 30 years in C.F. and these are amazing results,’’ Dr. Bonnie W. Ramsey, a lead investigator in the trial, said in an interview.

Dr. Ramsey, a professor of pediatrics at the University of Washington, was briefed on the results by Vertex.

The results were announced by a press release and have not been peer reviewed by experts.

About 30,000 Americans and 70,000 people worldwide have cystic fibrosis, a disease caused by defects in a gene responsible for the transport of chloride ions across cell membranes. People with the disease tend to have very thick mucus in their lungs, which leads to infections and lung damage. Many do not live past age 40.

Two inhaled antibiotics and one drug that loosens mucus are approved to treat cystic fibrosis, but nothing that directly improves chloride ion transport.

In the trial, those who received VX-770 gained 10.6 percentage points more on a lung function test after 24 weeks than those getting a placebo, a difference that statistically was highly significant. Patients continued to take either drug or placebo for another 24 weeks and the improvement was sustained. Lung function, the primary endpoint of the trial, was measured by how much a person could exhale in one second, a standard test.

Investors had been expecting around a 5 percentage point improvement. In a note to clients Tuesday evening, before the results were known, an analyst at ISI Group, Mark Schoenebaum, said that an improvement of 10 percent would be a “home run’’ that could lead to $600 million in annual sales for the drug.

Dr. Ramsey, who has received research grants from Vertex, said that some patients could perceive a 10 percentage point change in lung function. She said other results of the trial were encouraging.

These included fewer exacerbations of the disease in those who got the drug, fewer self-reported respiratory symptoms, and a gain in weight, which is good for people with cystic fibrosis, who often have digestive problems. The saltiness of their sweat — a measure used to diagnose the disease — was markedly reduced, suggesting that the drug was having an effect on chloride ion transport.

The trial involved 161 people age 12 and older, all with at least one copy of the particular mutation, known as G551D. The main side effects, Vertex said, were headache, upper respiratory tract infections, nasal congestion, rash and dizziness.

Vertex said it hoped to apply in the second half of the year for approval of VX-770 in the United States and Europe. The company is awaiting results of a second trial of the drug, this one in younger children.

Vertex has not said how much it will charge for VX-770. But since there are only about 1,200 Americans who are candidates for the drug, the price is likely to be tens of thousands of dollars a year.

The long time needed to develop a drug for cystic fibrosis is a lesson for those expecting a quick payout from the sequencing of the human genome, which was completed a decade ago. It is not enough to know the gene behind a disease. It can take years of research to determine how a mutation actually causes a disease and then to design a drug that corrects the problem.

Vertex received $75 million in financial support from the Cystic Fibrosis Foundation, one of the first nonprofit disease groups to give money to companies.

“These results are highly encouraging,’’ Robert J. Beall, president of the foundation, said in a statement Wednesday. “They provide scientific evidence that support our long-standing belief that targeting the underlying defect of C.F. may have a profound effect on the disease.’’

CF Foundation Announces Investment to Speed Development of Additional Drugs to Treat the Most Common CF Mutation

What follows is an excerpt from a recent article on the Cystic Fibrosis Foundation website, dated April 7, 2011:

Today the Cystic Fibrosis Foundation announced the expansion of its collaboration with Vertex Pharmaceuticals for the discovery and development of additional drugs aimed at treating the underlying cause of cystic fibrosis.

The new program will support development of a potential new drug called VX-661, designed to treat people with the most common genetic defect in CF, the Delta F508 mutation. Nearly 90 percent of people with CF in the United States have at least one copy of this mutation. 

VX-661 is known as a “corrector” and aims to move the defective CF protein to its proper place at the cell surface. Another corrector, known as VX-809, is already in clinical trials. By developing multiple correctors, the Foundation increases the chances of bringing new therapies to the CF community as quickly as possible.

“This new agreement will further leverage the successful collaboration with Vertex to accelerate the discovery and development of new drugs to treat a wide variety of CF patients,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “Given the recent announcement of promising data of other compounds in the CF pipeline, we’re optimistic that the CF Foundation is on the right path to fundamentally change the treatment of CF by targeting the cause of the disease.”

The Foundation’s investment, which will be up to $75 million over five years, will also expedite the discovery and early development of other new correctors.

“The CF Foundation is widely recognized by doctors, nurses, scientists and those with CF as a driving force in the search for new CF medicines, and we are pleased to further expand our strong collaboration with them,” said Matthew Emmens, chairman, president and chief executive officer of Vertex. “The collaboration announced today underscores our commitment to CF and accelerates our efforts to develop new medicines as quickly as possible for people with the most common type of this disease.”

With the Foundation’s expanded support, Vertex plans to begin a Phase 2 study of VX-661 by the end of 2011 and expects to enroll people with CF who have the Delta F508 mutation.

A Phase 2 clinical trial is underway to test combinations of VX-770 and VX-809 in individuals with two copies of Delta F508 mutation. Data from the first part of this trial is expected in the middle of 2011.

Cystic Fibrosis Foundation Now on YouTube!

The Cystic Fibrosis Foundation has established its own YouTube channel, CysticFibrosisUSA! YouTube, the leading video-sharing network, has changed the way people communicate. More companies and nonprofits than ever before are harnessing the power of this new media tool to spread awareness of their missions.

The CFF channel features videos created by the Foundation, as well as links to videos from the CF community. The Foundation's videos provide the latest CF news and share stories about people with CF and their families. The Cystic Fibrosis Foundation invites everyone to visit the new channel and view the videos, including the latest posting "One Goal." This video documents the CF Foundation's work as the leading organization committed to curing cystic fibrosis and the outstanding progress they are making.

To help CFF become the #1 nonprofit on YouTube:

  • View and comment on the channel's latest videos.
  • Bookmark and subscribe to the channel.
  • Share it with everyone you know.
  • Donate to support the efforts of the Cystic Fibrosis Foundation.