Arrowhead launches mid-stage study of RNAi drug in rare liver disorder

The first patient has been dosed in an open-label Phase 2 clinical trial, AROAAT2002, evaluating Arrowhead Pharmaceuticals’ (NASDAQ:ARWR) RNAi candidate ARO-AAT in patients with alpha-1 liver disease, a rare inherited liver disorder associated with alpha-1 antitrypsin deficiency (AATD), a Fast Track-tagged indication in the U.S.

The primary endpoint of the 12-subject study is the change from baseline to week 24 in Histological Liver Disease Activity Scale (Cohort 1) or week 48 (Cohort 2).

A pivotal trial, Phase 2/3 SEQUOIA, is in process.

https://seekingalpha.com/news/3527703-arrowhead-launches-mid-stage-study-of-rnai-drug-in-rare-liver-disorder