Pfizer, CFFT expand cystic fibrosis collaboration

BETHESDA, Md.—Cystic Fibrosis Foundation Therapeutics Inc.,the nonprofit drug discovery and development affiliate of the Cystic FibrosisFoundation, has announced a major expansion of its research collaboration withPfizer Inc. The collaboration is focused on discover new drugs to treat peoplewith Delta F508, the most common mutation of cystic fibrosis.

The new expansion agreement is based on a collaborationestablished between the two organizations in 2010 when Pfizer acquired biotechcompany FoldRx Pharmaceuticals Inc. The initiative was part of the company'seffort to discover and develop medicines for rare diseases, and the transactionincluded FoldRx's cystic fibrosis research program in collaboration with CysticFibrosis Foundation Therapeutics, which started in 2007.

"Innovative collaborations between industry and patientorganizations are increasingly critical in expediting the translation ofscience into new treatments," Jose-Carlos Gutierrez-Ramos, senior vicepresident of Pfizer BioTherapeutics R&D, said in a press release regardingthe expansion. "We look forward to continued collaboration with the CFFoundation and to applying our leading science with the goal of identifyingnovel therapies for the treatment of this devastating disease."

Per the terms of the new six-year preclinical researchprogram, Cystic Fibrosis Foundation Therapeutics will invest up to $58 millionto support and accelerate the discovery and development of therapies that havethe potential to target the intrinsic cause of the condition. The ultimate aimof the partnership is to see one or more drug candidates advance to the clinicby the end of the collaboration.

The agreement will seek to identify therapies that can helppatients with the Delta F508 mutation. In patients that have this particularmutation, a defective protein called CFTR does not fold correctly, which keepsit from reaching the surface of the cell and carrying out its function inmaintaining the proper flow of salt and fluids into the airways. The result isthat thick secretions form in the airways, which in turn leads to serious lunginfections as well as lung damage. Close to 90 percent of cystic fibrosissufferers carry at least one copy of the Delta F508 mutation. Pfizer and CysticFibrosis Foundation Therapeutics will work to discover therapies capable ofrestoring the normal function of the protein.

"We are excited to expand our efforts with Pfizer toaccelerate the development of more therapies that treat the root cause of CFand benefit the greatest number of people with the disease," Robert J.Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation, said in astatement. "Pfizer brings impressive technical and scientific expertise,along with its commitment to improving the lives of people with cysticfibrosis."

SOURCE: Cystic Fibrosis Foundation