EXCLUSIVE: Pfizer plans to move fast on rare disease pacts

Pfizer went public with its new rare disease unit earlier this week, but one of its top execs has been working away for the past few months setting up a division that is preparing to team up with a range of biotechs on new therapeutics.

"This morning at 7 a.m. I was at breakfast, talking to a company," Jose Carlos Gutierrez-Ramos, Pfizer's Cambridge-based senior vice president for biotherapeutics research and development, tells FierceBiotech. "We are actively talking to people and have been for the last two months."

Pfizer isn't just interested in partnering with biotechs working on new drugs for rare diseases. The company also wants to forge close ties with leading academics as well as key patient associations, several of which have taken an ambitious lead in funding studies of experimental therapies. In some cases, three-way pacts partnering Pfizer with a patient association and a drug developer could make sense.

"We'll do internally what we do well," he adds, "and what we don't do well we'll do externally with others." Dr. Edward Masciolo, who had founded Dapis Capital, will head the unit and report to Gutierrez-Ramos.

The goal is to create a small, "highly virtualized, highly focused" group that will start inking pacts in the next two to three months as it builds a significant pipeline of new drugs over the next two to three years, says the Pfizer exec.

"We are very interested in certain areas," he adds, highlighting hemophilia, muscular dystrophy and protein misfolding. "There are certain areas where we have biases," with the seriousness of the disease and the impact Pfizer can make on it the two leading indicators of where it will focus its resources.

But Pfizer isn't starting completely from scratch, either. The pharma giant has spent years developing new hemophilia therapies and recently licensed Protalix's Gaucher's drug, which will put it in direct competition with Genzyme, a large cap company that has made a big mark selling some very expensive medications for rare diseases. Genzyme's successes with drugs like Cerezyme helped inspired GlaxoSmithKline to start their own rare disease unit back in February.

"We have significant expertise in rare diseases," Gutierrez-Ramos says. "We've worked in hemophilia for 10 years and as a consequence of that we have developed a protein therapeutic and protein replacement capability and know-how that is unlike few other companies." - John Carroll