Arrowhead’s ARC-AAT is being investigated for the treatment of liver disease associated with the rare genetic disorder alpha-1 antitrypsin deficiency (AATD).
The Phase I trial is currently enrolling patients at a single center in Australia and, pending approval from ethics committees, the company will start recruiting patients at additional sites in the UK and New Zealand.
The company expects to complete patient enrollment in this multi-center, randomized, placebo-controlled, double-blind, single dose-escalation, first-in-human trial by the end of this year.
The trial is designed to evaluate the safety, tolerability and pharmacokinetics of ARC-AAT and the effect on circulating AAT levels.
Patient enrolment is being done in dose groups of six participants each, with participants randomized at a ratio of 2:1 to receive a single intravenous injection of either ARC-AAT or placebo (normal saline).
In this two-part trial, Part A in healthy volunteers has been completed and Part B will be conducted in patients with PiZZ genotype AATD.
The trial will evaluate participants for 28 days following dosing, with additional follow-up if needed every two weeks until AAT levels return to baseline.