Arrowhead Research Corp (NASDAQ:ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, announced the United States Food and Drug Administration (FDA) has granted ARC-AAT orphan drug designation. ARC-AAT is Arrowhead’s RNAi-based therapeutic candidate being investigated for the treatment of liver disease associated with Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disease that severely damages the liver and lungs of affected children and adults. Arrowhead is currently conducting part B of a Phase 1 study of ARC-AAT in patients with PiZZ genotype AATD.

“Receiving orphan drug designation is an important milestone in the development of ARC-AAT, which we think is a very promising program aimed at providing a better option for patients with liver disease associated with alpha-1 antitrypsin deficiency,” said Bruce D. Given, M.D., Arrowhead’s Chief Operating Officer. “The Orphan Drug Act provides important incentives for sponsors to develop drugs that treat rare diseases and we look forward to more engagement with the FDA as the development of ARC-AAT progresses.”

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. The study evaluates participants for 28 days following dosing, with additional follow-up if needed every 2 weeks until AAT levels return to baseline.

The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task, OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. Orphan drug designation provides incentives for sponsors to develop products for rare diseases. These incentives include increased engagement with FDA on drug development activities, exemption from all future product-specific regulatory fees, the opportunity to apply for R&D funding, tax credits, an increased chance of priority review, and 7 years of orphan exclusivity at time of New Drug Application (NDA) approval. (Original Source)

Shares of Arrowhead Research opened today at $6.4 and are currently trading up at $6.5801. 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 $6.57 and its 200-day moving average is $6.79.

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 represents a potential upside of 36.6% from where the stock is currently trading.

According to, Wei has a total average return of 14.8%, a 63.3% success rate, and is ranked #231 out of 3619 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.