Pfizer buys FoldRx for rare disease division

With a new neurodegenerative disease therapy up for regulatory review and a fresh $29 million financing round sitting in the bank, FoldRx announced this morning that Pfizer has moved in to scoop up the Cambridge, MA-based biotech to add to its orphan drug pipeline. The companies kept the numbers under wraps, but they did reveal that it's a structured buyout, with FoldRx investors getting an upfront payment with milestones based on future performance.

FoldRx's lead drug is tafamadis, which is designed to treat TTR amyloid polyneuropathy. A second tafamidis program has pushed through Phase II on TTR amyloid cardiomyopathy, which leads to heart failure. Last summer FoldRx touted the results of a II/III trial of tafamidis-calling it the "first disease-modifying agent targeting the underlying cause of a human amyloid disease." The research has been funded by the National Institutes of Health, the Skaggs Institute for Chemical Biology, and The Lita Annenberg Hazen Foundation. FoldRx has filed for European approval and is close to filing its app with the FDA.

"This transaction will add another important component to the growing portfolio of innovative in-line and investigational medicines for orphan and rare diseases within Pfizer's Specialty Care Business," said Geno Germano, the president and general manager of Pfizer's Specialty Care Business Unit, "and will complement the current and planned future research and clinical development taking place in Pfizer's Specialty Care Neuroscience disease area." FoldRx's portfolio includes clinical and pre-clinical programs for investigational compounds to treat diseases caused by protein misfolding.

"Over the past five years the FoldRx team has successfully developed tafamidis from the bench stage to MAA submission," said Richard Labaudiniere, president and chief executive officer of FoldRx. "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 tafamidis to all patients affected by this devastating neurodegenerative disease."

- here's the FoldRx release