Pfizer adds wrinkle to FoldRx

Pharma acquires pharmaceutical company to boost its specialty care business

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COLLEGEVILLE, Pa.—Pfizer Inc. has agreed to acquire privately held FoldRx Pharmaceuticals, whose leading drug candidate is a therapy for a genetic neurodegenerative disease for which the only current therapy is a liver transplant.

FoldRx's portfolio includes clinical and preclinical programs for investigational compounds to treat diseases caused by protein misfolding, which is increasingly recognized as an underlying cause in many chronic degenerative diseases. The company's lead product candidate, tafamidis meglumine, is in registration as an oral, disease-modifying therapy for TTR amyloid polyneuropathy (ATTR-PN), a progressively fatal genetic neurodegenerative disease, for which liver transplant is the only treatment option.  

Tafamidis meglumine is in registration in the European Union, which has the largest ATTR-PN patient population, as an oral, disease-modifying, therapy for ATTR-PN. FoldRx is currently in communication with the U.S. Food and Drug Administration (FDA) to define its pathway for filing in the United States. Tafamidis has orphan drug designation in both the United States and the European Union, and fast-track designation in the United States for the treatment of ATTR-PN. Treatments designated by the FDA as orphan medicines get an additional seven years of market exclusivity in addition to the standard 20 years of patent protection.

FoldRx is also working on drugs in earlier stages of testing for Parkinson's disease, Huntington's disease, cystic fibrosis and a rare heart condition called transthyretin amyloid cardiomyopathy. All of its research is focused on developing chemical compounds, rather than injectable biologics.

While specific financial terms were not disclosed, Pfizer said it will make an upfront payment and contingent payments if certain milestones are achieved. The closing of the transaction is subject to regulatory approval in the United States and is expected to be finalized later this year.

Dr. Richard Labaudiniere, president and CEO of FoldRx, points out that over the past five years, the FoldRx team has successfully developed tafamidis from the bench stage to MAA submission.

"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," he adds.

According to Joan Campion, senior director of worldwide communications for Pfizer, the acquisition of FoldRx and its lead candidate 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. That research division, Campion says, was set up in June in Collegeville, Pa., to focus on developing medicines for orphan diseases, classified as conditions affecting less than 200,000 people. According to the National Organization for Rare Disorders, more than 30 million Americans have one of 6,000 orphan diseases.

"The research unit builds on Pfizer's internal expertise in rare, orphan and genetic disease drug development and pursues treatments across all therapeutic areas and modalities," Campion says. "The specialty care business unit also has a specific expertise in rare disease areas, which is highly transferable to orphan disease areas."

Geno Germano, head of that unit, says the acquisition will especially complement research in Pfizer's neuroscience disease pipeline.

"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," he notes.

In the end, it was science that attracted Pfizer to the Cambridge, Mass.-based firm, says Campion. FoldRx has employed its proprietary yeast-based drug target discovery platform to build its portfolio of preclinical and clinical candidates. Its screening engine is rapid and efficient in evaluating potential treatment candidates in a wide range of diseases caused by misfolded proteins.

The acquisition wasn't an instance of a larger company swallowing up a smaller business swimming in red ink. Since its founding, FoldRx has pulled in about $88 million in venture capital backed by investors Alta Partners, Novo Ventures, Morgenthaler Ventures, HealthCare Ventures, Fidelity Biosciences, TPG Biotechnology and Novartis Venture Funds.

The measure of success of this acquisition, according to Campion, will be the resulting treatments that arise from the continued development of FoldRx candidates.

"While we don't provide our internal metrics, the ultimate goal would be the achievement of a non-surgical treatment option for underserved patients who are affected by the deadly disease ATTR-PN," she says.

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