Arrowhead Research Corp (NASDAQ:ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, announced that it received regulatory permission in the United Kingdom and New Zealand to proceed with Part B of its Phase 1 study of ARC-AAT, Arrowhead’s RNAi-based drug candidate for the treatment of liver disease associated with the rare genetic disorder alpha-1 antitrypsin deficiency (AATD). The Phase 1 study is currently enrolling patients at a single center in Australia and, pending approval from ethics committees, the company intends to begin recruiting patients at additional sites in the United Kingdomand New Zealand. Arrowhead expects to complete enrollment by the end of 2015.

The ongoing Phase 1 trial of ARC-AAT is a multi-center, randomized, placebo-controlled, double-blind, single dose-escalation, first-in-human study to evaluate the safety, tolerability and pharmacokinetics of ARC-AAT and the effect on circulating AAT levels. The study has been enrolling in dose cohorts of six participants each, with participants randomized at a ratio of 2:1 (active:placebo) to receive a single intravenous injection of either ARC-AAT or placebo (normal saline). The study consists of two parts; Part A in healthy volunteers, which has been completed, and Part B to be conducted in patients with PiZZ genotype AATD. Dosing in patients begins at the highest dose level used in healthy volunteers and then continued dose escalation may proceed under the protocol. The study evaluates participants for 28 days following dosing, with additional follow-up if needed every 2 weeks until AAT levels return to baseline. (Original Source)

Shares of Arrowhead Research Corp. closed yesterday at $6.77 . ARWR has a 1-year high of $17.42 and a 1-year low of $4.95. The stock’s 50-day moving average is $6.67 and its 200-day moving average is $6.97.

On the ratings front, Jefferies Co. analyst Thomas Wei downgraded ARWR to Hold, with a price target of $9, in a report issued on April 14. The current price target implies an upside of 32.9% from current levels. According to, Wei has a total average return of 15.3%, a 62.6% success rate, and is ranked #211 out of 3692 analysts.

Arrowhead Research Corp is engaged in developing novel drugs to treat intractable diseases by silencing the genes. The Company’s drug candidate ARC-520 is designed to treat chronic hepatitis B infection.