The Cystic Fibrosis Foundation said today it will spend up to $58 million over six years to expand its drug development with Pfizer to hasten development of drugs for the most common mutation associated with the disease, Delta F508.
The collaboration’s goal is to advance one or more drug candidates into the clinic to start clinical trials by the end of the six-year effort. The drug candidate or candidates will be designed to help restore normal function of a defective protein in people with the Delta F508 mutation.
In people with Delta F508, CFTR does not fold correctly and is unable to reach the cell surface, where it is needed to help maintain the proper flow of salt and fluids into the airways. Thick secretions form in the airways, leading to serious lung infections and lung damage. About 90% of people with CF have at least one copy of Delta F508.
Pfizer’s pipeline has no cystic fibrosis drug candidates at present. However, the pharma giant is set to benefit from the collaboration through development and commercialization milestone payments. “Pfizer brings impressive technical and scientific expertise, along with its commitment to improving the lives of people with cystic fibrosis,” Robert J. Beall, Ph.D., president and CEO of the CF Foundation, said in a statement.
Dr. Beall told Xconomy that if Pfizer decides not to pursue development of any molecule it discovers, the foundation can walk away from the collaboration, taking with it the drug candidate.
Pfizer and the foundation announced last December they would continue the CF research collaboration launched in 2007 by the nonprofit group and FoldRx Pharmaceuticals, at a value back then of up to $22 million. Three years later, Pfizer acquired FoldRx, buying with the company its yeast-based, high-throughput screening platform designed to detect new compounds that could improve the function of a misfolded CFTR protein.
The foundation has successfully collaborated with other drug developers. With Vertex Pharmaceuticals, the foundation persuaded FDA in January to approve ivacaftor (Kalydeco), the first drug for the genetic cause of a rare form of CF. The nonprofit also maintains a partnership with Sanofi’s Genzyme subsidiary, and overall is actively supporting nearly 30 potential new treatments.