Arrowhead Research (ARWR) Initiated Phase 2 Clinical Study of ARC-AAT in AATD

September 6, 2016 7:31 AM EDT

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Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it initiated a Phase 2 clinical study of ARC-AAT, an investigational RNAi-based medicine for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD). The study is designed to evaluate safety and tolerability and determine the effect of multiple doses of ARC-AAT on levels of circulating and intrahepatic alpha-1 antitrypsin as evidenced by changes in liver biopsy in patients with AATD.

Bruce Given, M.D., chief operating officer and head of R&D at Arrowhead said, “There remains no medical treatment for the liver disease associated with AATD, which is increasingly being recognized by patients and physicians as a serious problem. Our Phase 2 2001 study should give us, and the AATD community in general, the first insights into whether ARC-AAT can stop the progression of liver disease and possibly even allow the liver to recover and heal existing damage. This would be a significant breakthrough for patients.”

The ARC-AAT Phase 2 study (ARCAAT2001) is a multi-center, open-label, multiple dose-escalation study of ARC-AAT in patients with AATD. In total, the study will consist of at least 8 and a maximum of 12 participants. Two dose levels, 4 mg/kg and 6 mg/kg, will be evaluated in two separate cohorts. All subjects will receive a pre-dose biopsy, seven doses of ARC-AAT (once every 28 days), and a post-dose biopsy completed at Day 183. The study will be conducted at multiple centers in Canada, Ireland, and Sweden. The Company may add additional centers in other countries, pending regulatory and ethics review.

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