There is a great deal of anticipation surrounding the latest results from Arrowhead Research Corporation, who are preparing to present the latest results from their ongoing Phase 2a trial of ARC-520, their leading RNAi therapeutic candidate for treating chronic hepatitis B (HBV) infection. The data will be presented at the American Association for the Study of Liver Diseases (AASLD) Liver Meeting, November 7-11, 201, in Boston. Additionally, Arrowhead will present data on efficacy of ARC-AAT, a second RNAi therapeutic drug, for treating liver disease associated with Alpha-1 antitrypsin deficiency.

Christopher Anzalone, Ph.D., Arrowhead’s President and Chief Executive Officer noted, “ARC-520 represents a novel approach for the treatment of HBV with the potential to achieve functional cures. Our ongoing Phase 2a dose finding study is an important step, and in cohort 1 at a dose of 1 mg/mg and cohort 2 at 2 mg/kg we saw a clear reduction in HBsAg, the surface antigen of HBV.”

In spite of conjecture from the financial media, Dr. Anzalone noted, “Data collection for HBsAg reduction in cohort 3 at 3 mg/kg is still ongoing. However, we are pleased to report that all three dose levels have been well tolerated in patients. These results give us great confidence as we move forward with designing and initiating several upcoming Phase 2b studies of ARC-520 and the ARC-AAT Phase 1 study.”

ARC-520 thus is a RNAi-based drug to treat chronic HBV infection by targeting the mRNA expression and reducing the expression of key viral proteins. ARC-AAT is also a RNAi-based drug highly effective at targeting Alpha-1 antitrypsin (AAT) mRNA leading to reduced expression of AAT protein, a key factor responsible for progressive liver damage.