Pfizer has decided to acquire FoldRx Pharmaceuticals, which is developing drugs to treat diseases caused by protein misfolding. With this purchase Pfizer furthers its interest in rare and orphan diseases as well as CNS diseases.
FoldRx' lead candidate, tafamidis meglumine, stabilizes the protein transthyretin (TTR) and prevents dissociation of the tetramer, the rate-limiting step in TTR amyloid polyneuropathy (ATTR-PN). FoldRx has completed a 128-patient, international, multicenter Phase II/III study. Currently, liver transplant is the only treatment option available for this progressively fatal genetic neurodegenerative disease. In Europe the firm has filed an MAA. Tafamidis has orphan drug designation in the U.S. and the EU and Fast Track designation in the U.S. for the treatment of 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," remarks Geno Germano, president and GM, Pfizer specialty care business unit.
"By combining FoldRx' 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 nonsurgical treatment option for underserved patients affected by the deadly disease ATTR-PN," Germano adds.
FoldRx uses a yeast-based drug target discovery platform. Besides its focus on diseases caused by misfolded proteins, the company is also trying to identify therapeutic agents for cystic fibrosis, Parkinson disease, and Huntington disease. Tafamidis is its only clinical candidate.
While specific financial terms were not disclosed, Pfizer will make an up-front payment and contingent payments if certain milestones are achieved. Pfizer moved into the orphan drug space in December 2009 when it paid Protalix $60 million up front for its Gaucher disease enzyme replacement therapy (ERT), taliglucerase alfa. The company also agreed to pony up another $55 million in milestones related to regulatory approvals.
Competition for Gaucher disease will come from Shire, which has both FDA and EC approval for its ERT, VPRIV™ (velaglucerase alfa for injection). Genzyme already has a drug on the market, Cerezyme, but there have been significant shortfalls over the past year due to manufacturing issues. On August 30, sanofi-aventis made its $18.5 million bid for Genzyme public, and the latter responded with a resounding no to offer.
Orphan drug development, which has been the mainstay of biotech firms for about two decades, has been attracting big pharmaceutical companies in recent past. In 2009, big pharma accounted for 43% of the total orphan drug approvals by the FDA and claimed over 70% of the market share—up from an estimated 53% in 2006, according to BCC Research.