Arrowhead completes dosing of ARO-AAT Phase 1 study to treat rare genetic liver disease

Reviewed by Alina Shrourou, B.Sc. (Editor)Aug 31 2018

Arrowhead Pharmaceuticals Inc. today announced that it has completed dosing of a Phase 1 clinical study of ARO-AAT, the company's second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency. Arrowhead intends to submit a late-breaking abstract with initial clinical data on ARO-AAT to the Liver Meeting^®, the Annual Meeting of the American Association for the Study of Liver Disease (AASLD), being held in November 2018.

AROAAT1001 (NCT03362242) is a Phase 1 single- and multiple-ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and effect of ARO-AAT on serum alpha-1 antitrypsin levels in healthy adult volunteers. The study includes 7 cohorts in which subjects receive placebo, a single dose of ARO-AAT, or three monthly doses of ARO-AAT at doses of 35 (single dose only), 100, 200, or 300 mg. Additional cohorts were planned at a dose of 400 mg, but were deemed unnecessary based on observed activity at lower doses.