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Phase 3 Study of VX-770 Shows Marked Improvement in Lung Function Among People with Cystic Fibrosis with G551D Mutation

Wed, 02/23/2011 - 5:35am
Bio-Medicine.Org

BETHESDA, Md., Feb. 23, 2011 /PRNewswire-USNewswire/ -- The Cystic Fibrosis Foundation and Vertex Pharmaceuticals announced today that VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis, showed promising results in a Phase 3 clinical trial.

The trial was designed to evaluate patients age 12 and up who carry at least one copy of a CF mutation called G551D. The study included 161 patients who received at least one dose of VX-770 or placebo.

Patients who took the drug, compared to those on placebo, showed a marked improvement in lung function at 24 weeks, which was sustained for the duration of the 48-week trial.

Patients also showed improvement across all key secondary endpoints in the study, including reduced likelihood of experiencing a pulmonary exacerbation, decreased respiratory symptoms and improved weight gain. Each of these areas is critically important to the health of people with CF.

In addition, average sweat chloride levels of patients on VX-770 dropped toward normal levels, while those on placebo did not change—indicating the drug is impacting the underlying defect in CF. Excessive sweat chloride (salt) is a key clinical indicator of CF.

VX-770 is being developed by Vertex, and was discovered in collaboration with the CF Foundation, which provided substantial support to Vertex throughout the development process, including an approximately $75 million investment.

About four percent of people with CF carry the G551D mutation. More studies are needed to determine whether other CF mutations may benefit from VX-770.

"These results are highly encouraging. They provide scientific evidence that support our long-standing belief that targeting the underlying defect of CF may have a profound effect on the disease," said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. "We have much more to do to end the suffering caused by thi

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