Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.US-based Arrowhead Research has started dosing patients in Part B of a Phase I clinical trial of its RNAi-based drug candidate ARC-AAT to treat liver disease associated with the rare genetic disorder alpha-1 antitrypsin deficiency (AATD).
Subscribe to our email newsletter
Recently, the US Food and Drug Administration (FDA) has granted orphan drug designation for ARC-AAT to treat AATD.
Currently, the company is enrolling patients at a single center in Australia and expects to open additional sites for enrollment in Europe, pending regulatory permission to proceed.
The company intends to complete patient enrollment in this Phase I trial by the end of this year.
Arrowhead chief operating officer Bruce Given said: "Dosing the first alpha-1 patient with ARC-AAT is a milestone for Arrowhead and for patients with AATD.
"The goal of treatment with ARC-AAT is to halt progression and possibly reverse the liver injury and fibrosis associated with AATD, which currently has no approved therapy short of liver transplant. This is becoming a larger clinical problem that we believe ARC-AAT holds great potential to address.
"We would also like to thank the Alpha-1 Foundation and The Alpha-1 Project, who have agreed to help support the development of ARC-AAT through funding and assistance with patient recruitment."
The company said that the ongoing Phase I trial is a multi-center, randomized, placebo-controlled, double-blind, single dose-escalation study and is designed to evaluate the safety, tolerability and pharmacokinetics of ARC-AAT and the effect on circulating AAT levels.
The trial has been enrolling 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.
The Phase I trial includes two parts; Part A in healthy volunteers, which has been completed, and Part B will be conducted in patients with PiZZ genotype AATD.