Pfizer acquires ‘protein misfolding’ specialist
pharmafile | September 2, 2010 | News story | Research and Development | FoldRx, Pfizer, cystic fibrosis
Pfizer has bolstered its presence in rare and orphan diseases with the acquisition of FoldRx, a US drug discovery firm that specialises in treatments for diseases caused by ‘protein misfolding’.
The accumulation of misfolded proteins, which occur when proteins fail to achieve the correct three-dimensional structure, can be an underlying cause in a number of rare and degenerative diseases.
FoldRx’s lead product candidate tafamadis is a first-in-class oral treatment for transthyretin amyloid polyneuropathy (ATTR-PN), and has been filed with European regulators for approval.
Geno Germano, president and general manager of Pfizer specialty care business unit, said: “By combining FoldRx’s proprietary expertise in identifying and developing treatments for protein misfolding diseases with Pfizer’s commercial, medical and regulatory expertise, and global strengths in patient services and reimbursement, 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.
“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, and will complement the current and planned future research and clinical development taking place in Pfizer’s specialty care neuroscience disease area.”
Pfizer will also gain access to FoldRx’s proprietary yeast-based drug target discovery platform to build upon its portfolio of pre-clinical and clinical candidates.
In a statement Pfizer said this screening engine will also aide future drug discovery by using FoldRx’s platform to identify therapeutic agents for cystic fibrosis, Parkinson’s disease and Huntington’s disease.
In addition to tafamadis’ European filing, for which the product has orphan drug status, FoldRx said it is “currently in communication with the FDA to define its pathway for filing in the US”.
Richard Labaudiniere, president and chief executive of FoldRx, said: “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.”
Transthyretin (TTR) is a protein secreted by the liver and deposits of TTR amyloid in the peripheral nerve tissue result in ATTR-PN, a sensory, motor and autonomic polyneuropathy.
Currently liver transplantation is the only accepted treatment, but this has a limited efficacy with a high mortality rate. It affects around 8,000 patients worldwide, the majority of whom live in the EU.
While specific financial terms of its acquisition of FoldRx were not disclosed, Pfizer said it would make an upfront payment and then contingent payments if certain milestones are achieved.
Ben Adams
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