VX-770 on Track for New Drug Application and Additional Clinical Trials Planned for More Patient Groups 

July 29, 2011

Vertex Pharmaceuticals Inc. announced today it plans to submit a New Drug Application for VX-770, a potential CF medicine, to the U.S. Food and Drug Administration (FDA) in October. The company is seeking approval for the drug in people age 6 and older with at least one copy of the G551D mutation of cystic fibrosis.

About 4 percent of people with CF in the U.S. have the G551D mutation.

Vertex also said it expects to begin testing the drug in additional CF patient groups in the first half of 2012. This includes a study of the drug in children age 2 to 5 with the G551D mutation and studies of VX-770 in people with CF with other “gating” mutations besides G551D.

“We are thrilled that Vertex is preparing to apply for FDA approval this October, keeping VX-770 on track for possible approval next year,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “We’re also extremely encouraged the company plans to broaden its VX-770 program to evaluate whether greater numbers of people with CF can potentially benefit from this treatment.”

Additional VX-770 Studies in People with Gating and Other CF Mutations

In gating mutations such as G551D, the defective protein in CF moves to its proper place on the cell surface but does not function correctly. It acts instead like a locked gate, impeding the proper flow of salt and fluid in and out of the cell. VX-770 aims to unlock the gate, helping to restore the function of the defective protein.

The new studies may also include people with CF mutations other than gating mutations, Vertex said.

Vertex will provide additional information on these studies after it has completed discussions with regulatory agencies.

More Studies of Combination Therapies to Treat Most Common CF Mutation

In September, Vertex plans to begin the second part of a Phase 2 clinical trial evaluating VX-770 in combination with another experimental drug, VX-809.

The study will enroll people with at least one copy of the Delta F508 mutation, the most common CF mutation, and is expected to evaluate higher dosing levels over a longer period of time than in the first part of the combination trial, completed earlier this year.

Vertex also plans to start a clinical trial by the end of 2011 that will study VX-770 in combination with a third drug in development, VX-661, in people with two copies of the Delta F508 mutation.

The CF Foundation worked with Vertex to discover VX-770, VX-809 and VX-661 and has provided substantial scientific, financial and clinical support throughout the development process.

Earlier this year, the Foundation announced an expanded collaboration with Vertex, which includes an investment of up to $75 million over five years to accelerate the development of new drugs to treat the underlying cause of CF, including VX-661.

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