— Up to 5.0 log10 reduction in HBsAg observed; data presented at HEP DART 2017 —
In an oral presentation titled, "Looking Back to Move Forward -
Designing Next Gen RNAi for HBV,"
In the 4 patients that appeared to have a sustained host response, observed reductions in HBsAg were 5.0, 3.1, 2.4, and 0.6 log10 from baseline. In addition, these patients achieved absolute levels of HBsAg of 58, 2.6, 0.36, and 0.051 IU/ml. The lower limit of quantitation for this measurement of HBsAg is 0.05 IU/ml, below which would be deemed seroclearance. These patients also achieved reductions in HBV DNA to below the level of quantitation, and deep reductions in core-related antigen (HBcrAg), and HBeAg, with many at or below their respective lower limits of quantitation.
In addition, Arrowhead created a mutated pHBV mouse model that eliminates the HBx trigger site to simulate HBV patients with high levels of integrated HBV DNA relative to cccDNA. In this model, a single dose of ARO-HBV led to a reduction in HBsAg of 2.95 log10. The duration of effect was long, with a HBsAg reduction of approximately 2.0 log10 still observed 8 weeks after the dose.
ARO-HBV is designed to silence the production of all HBV gene products, including transcripts derived from integrated DNA, with the goal of getting to a level where patients' immune systems can reconstitute, leading to a sustained host response and ultimately a functional cure. Arrowhead's learnings from multiple clinical studies of prior generation compounds, ARC-520 and ARC-521, and the extensive non-clinical research completed in multiple species, including long-term treatment of chimpanzees, have guided the rapid development of ARO-HBV. The results presented with respect to ARC-520 are not necessarily predictive of ARO-HBV results.
GLP-toxicology studies are being conducted and Arrowhead is manufacturing the drug supply necessary to begin clinical studies of ARO-HBV. The company anticipates filing a Clinical Trial Application by the second quarter of 2018.
Slides from Dr. Given's presentation may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.
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