FOLDRX BEGINS PHASE I STUDY WITH FX-1006A
FoldRx Pharmaceuticals has initiated a Phase I clinical study with its lead candidate, Fx-1006A, a small molecule compound with the potential to treat genetic disorders such as familial amyloid cardiomyopathy (FAC) and familial amyloid polyneuropathy (FAP). Fx-1006A is a first-in-class, disease-modifying compound that is designed to inhibit the formation of amyloid deposits by preventing the misfolding and deposition of the transthyretin protein (TTR), which is associated with these diseases.
The trial will be a double-blind, placebo-controlled single- and multiple-dose escalation study, enrolling approximately 55 healthy volunteers. The study will seek to evaluate safety and tolerability of Fx-1006A, and to determine the compound's pharmacologic properties and TTR stabilization activity.
TTR is a hormone-carrying protein that is produced in the liver and circulates in the blood. In patients with certain genetic mutations, TTR is destabilized and misfolds, resulting in amyloid deposits in various tissues. TTR misfolding is associated with a number of amyloid diseases, which typically occur in patients aged 30 and above. It is believed that stabilization of transthryetin will inhibit further amyloid deposition and stop progression of the disease in FAP and FAC patients.