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Arrowhead Starts Patient Dosing in Phase I Trial of ARC-AAT in Patients with Alpha-1 Antitrypsin Deficiency
2 July 2015 - - US-based biopharmaceutical company Arrowhead Research Corp. (NASDAQ: ARWR) has dosed the first patient in Part B of a Phase 1 clinical trial of ARC-AAT, the company said on Thursday. ARC-AAT is Arrowhead's RNAi-based drug candidate for the treatment of liver disease associated with the rare genetic disorder alpha-1 antitrypsin deficiency. It was recently granted orphan drug designation by the United States Food and Drug Administration The clinical trial is currently enrolling patients at a single center in Australia and the company intends to open additional sites for enrollment in Europe, pending regulatory permission to proceed. Arrowhead said it expects to complete enrollment of the Phase 1 study by the end of 2015. The ongoing Phase 1 trial of ARC-AAT is a multi-centre, randomised, 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. It 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. Arrowhead's ARC-AAT is being investigated for the treatment of liver disease associated with Alpha-1 Antitrypsin Deficiency, a rare genetic disease that severely damages the liver and lungs of affected individuals. AATD is also an important cause of pediatric liver disease with an estimated prevalence in children of approximately 20,000 patients, and 50-80% likely to manifest liver disease during childhood. It is a rare disease that appears to be frequently misdiagnosed or undiagnosed.
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