Pasadena, CA-based Arrowhead's hepatitis B candidate ARC-520 showed in a primate study to substantially reduce the viral DNA, as well as antigens associated with the disease, for over two months. And importantly, an "immunological flare" results from an increase in serum alanine transaminase after four weeks, which the company said could lead to antigen conversion and a "functional cure," as presented this week at the American Association for the Study of Liver Diseases' annual meeting.
This is a major step for hepatitis B in particular and for RNAi
capability in general. The delivery of genetic drugs has been a major
hurdle in the past, but recent clinical and financing successes for
Arrowhead and other companies such as Alnylam ($ALNY)
and Arcturus have demonstrated a turning point. Arrowhead's Dynamic
Polyconjugate delivery vehicle uses a "masking" and "unmasking" process
that allows RNA to enter the cell unharmed and then "escape" once it
reaches the target.