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Vertex Announces Phase 2 Results of Combination Treatment in People with One Copy of the F508del CF Mutation

July 31, 2014

Vertex Pharmaceuticals Inc. announced results from a Phase 2 clinical trial of the cystic fibrosis drug ivacaftor (Kalydeco™) in combination with another potential therapy, lumacaftor, in people with one copy of the F508del mutation, ages 18 and older.

In the study, those who received the two drugs in combination did not demonstrate a significant improvement in lung function, a primary endpoint of the 8-week trial.

Both ivacaftor and lumacaftor are aimed at treating the underlying cause of CF — a defective protein, called CFTR, caused by mutations in the CF gene.

“While these results are disappointing, we knew from earlier trials that correcting the faulty protein in people with one copy of the F508del mutation would be especially challenging,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “For this reason, the Foundation expanded our research funding to speed development of an additional compound, VX-661, and to support other second-generation programs to find more compounds that could be coupled with ivacaftor to treat those with one copy of this mutation.”

Vertex said it may evaluate the potential ivacaftor and VX-661 combination treatment in those with only one copy of the F508del mutation.

“We are actively pursuing other promising opportunities to find new therapies to address the basic defect in all people with CF,” added Beall. “It is likely that a three-drug combination will be needed to bring the greatest benefit to people with one copy of F508del.”

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