The Cystic Fibrosis Foundation recently 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 (CF). The new program will support development of a potential new drug called VX-661, designed to treat people with the Delta F508 mutation, the most common genetic defect in CF.
The drug is known as the “corrector” and aims to move the defective CF protein to its proper place at the cell surface. A similar drug, known as VX-809, is already in clinical trials. The Foundation believes that by developing multiple correctors it increases the chances of bringing new therapies to the CF community as quickly as possible.
“The 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, PhD, president and CEO of the 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 as much as $75 million over 5 years, is also expected to expedite the discovery and early development of other new correctors.
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 already 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.
Source: Cystic Fibrosis Foundation