Arrowhead Research Corporation has initiated dosing in a Phase 1 clinical trial of ARC-AAT, the company’s candidate for the treatment of liver disease associated with Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disorder that severely damages the liver and lungs of affected individuals.
The primary objectives of the study are to determine the safety and tolerability of escalating doses of ARC-AAT, evaluate the pharmacokinetics, and determine the effect on circulating levels of alpha-1 antitrypsin. Initial data from this study is expected in late 2015.
