Vertex Pharmaceuticals Inc. today announced it plans to submit to the U.S. Food and Drug Administration in October a new drug application for VX-770, a promising CF therapy.  Following is information on the announcement from Vertex and the Cystic Fibrosis Foundation.


FROM THE CYSTIC FIBROSIS FOUNDATION WEBSITE:

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.


EXCERPT FROM THE VERTEX PHARMACEUTICALS PRESS RELEASE:

Submission of VX-770 NDA and MAA on Track for October 2011

The Phase 3 program for VX-770, Vertex's cystic fibrosis transmembrane conductance regulator protein (CFTR) potentiator, is now complete. In October, Vertex plans to submit both its VX-770 NDA to the U.S. Food and Drug Administration (FDA) and its VX-770 MAA to the European Medicines Agency (EMA). Additional regulatory submissions are planned for Canada and other countries following the submissions of the NDA and MAA. Vertex is seeking approval of VX-770 in people six years of age and older who have at least one copy of the G551D mutation in the CFTR gene.

Additional Studies of VX-770 Planned for 2012

Pediatric Study: The Phase 3 program for VX-770 was focused on people ages 6 and older with the G551D mutation. In the first half of 2012, Vertex plans to begin the first study of VX-770 in children younger than 6 years of age. The study is expected to enroll children ages 2 through 5 and will evaluate the safety, tolerability, and effect on sweat chloride and other measures of clinical efficacy using a pediatric formulation of VX-770.

Studies of VX-770 in People with other CFTR Mutations: The G551D mutation is the most common gating mutation, present in approximately 4 percent of all people with CF. In the first half of 2012, Vertex plans to begin two clinical studies of VX-770 in people with CF who have other CFTR mutations where, based on in vitro data, VX-770 may help improve the function of CFTR proteins at the cell surface. These additional studies are expected to enroll people with CF who have certain other gating mutations that result in the CFTR protein functioning abnormally at the cell surface, as well as people with other mutations that result in some residual CFTR function. A goal of the trials will be to generate data to support the evaluation of the use of sweat chloride measurements as a marker for clinical benefit in people with CF.

Additional information on these studies will be provided upon completion of discussions with regulatory agencies in the U.S. and E.U.

Phase 2 Trials Combining Two CFTR Modulators for the Treatment of People with the Most Common Mutation of CF

Vertex is conducting an exploratory Phase 2 clinical trial to evaluate combination regimens of VX-770 and VX-809, a CFTR corrector, in people with the most common mutation in CF, known as F508del. Vertex recently completed the first part of the trial and is on track to initiate the second part of the trial in September 2011. Part Two of this trial will include dosing of VX-809 alone for at least four weeks followed by dosing of VX-770 and VX-809 in combination for at least four weeks. Similar to Part One, the primary goals of the second part of the trial will be to evaluate the safety and tolerability and the effect of the combination of VX-770 and VX-809 on CFTR function as measured by sweat chloride. Lung function will be measured as a secondary endpoint. The trial is expected to evaluate higher doses of VX-809 than the 200 mg dose studied in the first part of the trial and to enroll people with CF who have one copy or two copies of the F508del mutation.

Vertex also plans to initiate a Phase 2a clinical trial to evaluate combination regimens of VX-770 and VX-661, another CFTR corrector, by the end of 2011. This trial will evaluate people with two copies of the F508del mutation.

Source: Vertex Pharmaceuticals and Cystic Fibrosis Foundation

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