Company aims to submit tafamidis for approval by end of the year.
FoldRx Pharmaceuticals obtained $29 million in a financing round that it says will fund the final progression of its lead candidate, tafamidis, to market. The firm anticipates filing for approval of the drug as a disease-modifying therapy for TTR amyloid polyneuropathy (ATTR-PN, or familial amyloid polyneuropathy) in the second half of this year.
Participants in the financing round included new investors Novo Ventures and Morgenthaler Ventures along with the firm’s existing investors. “With the addition of two top-tier venture firms to our already strong investor base, we believe we now have access to the resources necessary to comemrcialize tafamidis and build FoldRx into an independent, orphan disease-focused company,” states Richard Labaudinière, Ph.D., president and CEO.
Tafamidis is an oral, small molecule, first-in-class pharmacological chaperone. FoldRx Pharmaceuticals is focusing on first-in-class, disease-modifying, small molecule therapeutics to treat diseases of protein misfolding and aggregation, or amyloidosis. Tafamidis is also in clinical development for the treatment of TR amyloid cardiomyopathy. Positive results from a pivotal Phase II/III trial with tafamidis in patients with ATTR-PN were reported in July 2009.