Timmerman wrote an aricle in Xconomy today that highlights Arrowhead Research (a company emerging from the ashes of Mirus Bio and Roche) and its progress in the field of RNAi therapeutics. As described in the piece, Arrowhead (like most of the field of RNAi) has been able to rebound from a near death experience to make progress toward being able to deliver RNAi products to the right spot in the cell to be effective -- one of the toughest problems in the field of RNAi.
They have done this by developing what is increasingly looking to be a viable polymer-based delivery system. It has a couple pre-clinical trials going now to battle hepatitis B. Their next step is to pass a safety study in people and show signs tha the drug is active. Arrowhead's best hope to exploit the utility of its "dynamic polyconjugates" is with its durg candidate, ARC-520. The drug is designed to reduce the production of hepatitis B antigen in infected patients. The antigen is thought to suppress immune function and thus hamper the body's fight against the infectious virus. In animal studies, not only was the antigen knocked down but liver functions appear to return closer to normal. Arrowhead is now poised to engage in a phase 2a clinical study through mid-2014, and if that goes well roll into phase 2b later in the year.
If Arrowhead is successful in its upcoming clinical work, not only might it have a therapy that could work for hepatitis, it may have a delivery system that could be more universally applied to the delivery of RNAi drugs. Other developers would be expected to more aggressively pursue it for licenses to its delivery technology.
Posted by Bruce Lehr Dec 20th 2013.