Arrowhead's midstage hep B data shows promise for RNA-based 'functional cure'

RNA interference specialist Arrowhead ($ARWR) presented new data from an ongoing Phase IIa study of its hepatitis B treatment ARC-520, demonstrating a reduction of the disease's surface antigens in what the company says is a first for RNAi.

Arrowhead has been chugging along on its hepatitis B candidate, which makes use of the company's delivery system designed to overcome the vast challenges associated with RNAi. Last year, CEO Christopher Anzalone told FierceDrugDelivery that the polymer-based platform stands apart from other RNA platforms due to its ability to release the genetic material, what they term "endosomal escape." The company's Dynamic Polyconjugate approach allows for a "masking" and "unmasking" process that protects the RNA as it enters the cell, but also allows it to perform its interference duty once inside.


The new human data for ARC-520 showed significant reduction of the hepatitis B surface antigen (HbsAg), according to Arrowhead, as compared to placebo for up to 85 days, with the lowest point at about a month after dosing in the 54-patient study. Back in March, the company called its candidate a "functional cure" due to the presence of an "immunological flare" in primate studies.

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