Pfizer Gobbles FoldRx in Big Pharma’s Latest Rare Disease Play in Boston Area

Big drug companies can’t seem to resist buying Boston-area biotech firms in the rare disease drug business. Pfizer, the world’s largest drugmaker, said this morning that it has agreed to purchase Cambridge, MA-based FoldRx Pharmaceuticals for an undisclosed sum.

Venture-backed FoldRx will give New York-based Pfizer (NYSE:PFE) its lead molecule for a neurodegenerative disorder called TTR amyloid polyneuropathy, which affects a mere 8,000 patients worldwide. The buyout follows Pfizer’s announcement in June that it would set up a center of rare disease research in Cambridge. In December, the drug behemoth struck a deal to license a drug for the rare genetic disorder Gaucher’s disease from Israel-based Protalix BioTherapeutics.

Of course, Cambridge-based Genzyme (NASDAQ:GENZ), the granddaddy of the rare disease drug business, recently has become the acquisition target of French drug giant Sanofi-Aventis. While companies like Pfizer and Sanofi have historically focused on developing drugs for diseases that affect perhaps millions of patients, the rare disease drug business offers relatively low competition and high profit margins. Genzyme gets hundreds of thousands of dollars per patient for some its treatments for rare diseases.

While Pfizer isn’t yet saying how much it’s paying for FoldRx, it’s highly likely that FoldRx’s venture backers are in high spirits today. FoldRx, which was founded in 2004 to focus on treating diseases caused by protein misfolding, has disclosed financing rounds totaling $88 million. Its venture backers include Alta Partners, Fidelity Biosciences, Healthcare Ventures, Morgenthaler Ventures, Novartis Venture Funds, Novo Ventures, and TPG Biotechnology. These firms helped raise $29 million in a bridge round of financing for FoldRx in June.

Richard Labaudiniere, FoldRx’s president and chief executive, said in a statement that “Pfizer’s strong clinical and regulatory resources, global marketing reach, and commitment to the treatment of rare diseases will significantly enhance the ability to pursue the goal of efficiently bringing [our lead drug] to all patients affected by this devastating neurodegenerative disease.”

FoldRx has filed an application with the European Medicines Agency for approval of its lead drug, tafamidis, for patients with TTR amyloid polyneuropathy. In fact, most of the estimated 8,000 patients with the disease reside within the European Union. The company said it has been in talks with the FDA about submitting a similar marketing application in the U.S. The company’s drug targets a mutated protein from the liver that is believed to be the root cause of the disease. A liver transplant is the only existing treatment for the disease, which robs people of their ability to control how their arms and legs move and other motor functions.

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