Product Pipeline

TTR Amyloidosis Treatment Product Pipeline

Tafamidis meglumine  for the Treatment of ATTR-PN & ATTR-CM

Background on TTR Amlyoidosis: TTR is an amyloidogenic protein secreted by the liver. Mutations in the TTR gene have been linked to several amyloid conditions. Deposition of TTR amyloid in the peripheral nerve tissue results in ATTR-PN, a sensory neuropathy starting in the lower extremities and progressing to include autonomic and motor dysfunction. The disease usually begins in the third or fourth decade and the outcome is invariably progressive and fatal. It is estimated that ATTR-PN affects about 5,000-10,000 patients worldwide. Liver transplantation is currently the only disease modifying treatment available for these patients. ATTR-CM is caused by either specific point mutations in the TTR gene or age-associated TTR deposition, causing amyloid deposits that infiltrate the heart and result in a potentially fatal restrictive cardiomyopathy. ATTR-CM is a late onset disease generally occurring in individuals over 60 years of age. It is estimated that ATTR-CM affects about 400,000 patients in the U.S alone.

Background on tafamidis meglumine (Fx-1006A): Tafamidis is a new chemical entity, first in class, disease-modifying agent that stabilizes the protein transthyretin (TTR), dissociation of which is implicated in TTR amyloidosis. Tafamidis, a novel and specific TTR stabilizer, prevents dissociation of the native TTR tetramer into monomers, which results in the inhibition of TTR amyloid fibril formation. Tafamidis has orphan drug designation for ATTR-PN in both the U.S. and European Union (EU) and Fast Track designation in the U.S.

Pivotal Phase II/III in ATTR-PN: FoldRx  recently completed a large (128 patients) international, multicenter Phase II/III clinical study of tafamidis for the treatment of TTR-associated polyneuropathy. NDA filing for this orphan indication is expected in Q2 2010.

Phase II in ATTR-CM: FoldRx is currently running an open-label, interventional trial with tafamidis in patients with TTR-associated cardiomyopathy.