Pfizer buys Massachusetts company with key drug candidate

Pfizer Inc. announced today the acquisition, for an undisclosed price, of a Massachusetts company whose research focuses on neurogenerative disorders that the pharmaceutical giant has targeted, initially including Huntington's and Parkinson's diseases but possibly expanding to include Alzheimer's as well.

Pfizer said it is acquiring FoldRx Pharmaceuticals Inc., a privately held drug-discovery firm in Cambridge that has focused its attention on diseases caused by protein misfolding. The company's lead compound is tafimidis, a pill to treat Transthyretin Amyloid Polyneuropathy, known as ATTR-PN, a progressively fatal disease believed to be similar in action to Alzheimer's and Parkinson's.

Protein folding was first suggested as causing a wide range of neurogenerative diseases by scientists working out of the California-based Scripps Research Institute. One of those scientists is Jeffery Kelly, a co-founder of FoldRx.

"Amyloid diseases are caused by the misfolding of proteins into structures that lead them to cluster together, forming microscopic fibril or plaques, which deposit in internal organs and interfere with normal function, sometimes lethally," according to a summary of the research found online. "In the case of Alzheimer's, these fibrils kill nerve cells in areas of the brain that are crucial for memory."

In all, according to researchers, there are more than 80 amyloid diseases caused by protein misfolding. Alzheimer's alone afflicts about 4.5 million Americans.

Kelly, in announcing positive results from studies last year of FoldRx's tafimidis, said the drug's effect was the "first pharmacologic evidence that the biomedical researcher communities' hypothesis about the etiology of human amyloid diseases, including Alzheimer's disease, is likely correct."

Scripps researchers Evan T. Powers and Hossein Razavi discovered tafamidis, and it was developed by FoldRx starting with the company's founding in 2003.

Tafamidis is the first drug targeting the underlying cause of various amyloid diseases and has been granted orphan drug status in both the United States and Europe, clearing the way for quicker regulatory approvals.

One of Pfizer's three research units in Groton keys in on neurological disorders, and its main focus is on Alzheimer's.

"We are taking a significant step toward potentially bringing, for the first time, a non-surgical treatment option for underserved patients affected by the deadly disease ATTR-PN," said Geno Germano, president and general manager of Pfizer's Specialty Care Business Unit, in a statement. "This transaction will ... complement the current and planned future research and clinical development taking place in Pfizer's Specialty Care Neuroscience disease area."

"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," added Richard Labaudiniere, president and chief executive officer of FoldRx.

Pfizer did not disclose financial terms, but said the sale would include an upfront sum as well as milestone payments as key research targets are hit. The deal is expected to close later this year after regulatory approvals are granted.