The Cystic Fibrosis Foundation says it has agreed to invest up to $58 million over six years to expand its drug development partnership with New York-based Pfizer (NYSE: PFE), in an attempt to speed up the development of drugs that will treat people with the most common mutation associated with the deadly lung disease.
Pfizer currently has no cystic fibrosis drug candidates; the goal of the research program is to discover one or more potential drugs and advance them to the point of starting clinical trials, with Pfizer getting payments as it reaches certain milestones along the way. “We will support the program through proof of concept and then Pfizer will take the molecule into the clinic,” Cystic Fibrosis Foundation President Robert Beall told me. He says that if Pfizer decides not to pursue development of any molecule it discovers the foundation can walk away, taking the drug candidate with it.
Pfizer and the non-profit, based in Bethesda, MD, ended up as partners when Pfizer acquired FoldRx Pharmaceuticals in 2010. FoldRx had a five-year development agreement in place with the foundation, going back to 2007, that was worth up to $22 million.
Although Pfizer is the world’s largest pharmaceutical company and seemingly not lacking for resources to pursue drug development, Beall says the company, facing the loss of patent protection for some of its best-selling drugs, “is being very careful about which development areas they are going into, and cutting a lot of programs.” Pfizer launched an orphan and genetic diseases unit in Cambridge, MA, in 2010 “and they convinced us that our continued commitment [to the cystic fibrosis drug development program] would assure that CF has a major role in their new rare diseases program.”
The foundation funds more cystic fibrosis research than any other organization in the world, and has invested about $375 million to date in drug development for the disease. It has become a model for how to apply philanthropy to commercial drug development since its partnership with Cambridge, MA-based Vertex (NASDAQ: VRTX) led to FDA approval last January of ivacaftor (Kalydeco), the first drug to treat the genetic cause of a rare form of cystic fibrosis.
Ivacaftor targets a gene mutation called G551D that is the cause of about 4 percent of all cystic fibrosis cases. The Pfizer partnership is searching for drug candidates that target the Delta F508 mutation, found in almost 90 percent of people with cystic fibrosis. Beall says that Pfizer has one of the largest chemical libraries of any drug company, enabling it to screen for thousands of molecules that might work against cystic fibrosis.
Cystic fibrosis is an inherited disease that causes the lungs to clog up with thick, sticky mucus that attracts bacteria and makes it increasingly difficult for patients to breathe; few people with the condition live beyond their 30s. About 30,000 people in the U.S. have the disease and 70,000 worldwide, according to the foundation.
The foundation is not counting on Pfizer alone to come up with new cystic fibrosis drugs. It continues to support Vertex’s work on a second molecule, VX-809, that targets a genetic defect found in some 70 percent of patients, and has a development project with Genzyme, a subsidiary of France’s Sanofi-Aventis (NYSE: SNY). In total the foundation has or is supporting some 30 existing or experimental drugs.
“When I first started talking about venture philanthropy in 1999-2000 there wasn’t a lot of interest in the approach,” says Beall. “Now over $500 million a year is being invested by health-related organizations in drug discovery.” His foundation is specifically interested in finding drugs that target the root causes of disease, and its success with ivacaftor has proven that this strategy is working, he says.
Beall says the foundation had no plans to end its venture philanthropy efforts if some of the current candidates from Vertex, Genzyme, or other companies prove successful, as a second generation treatment might be even better.“ We are obviously very excited and we expect to continue to invest in and jump start companies to look at small molecules,” he says.
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