Investors clambered aboard as Arrowhead unveiled the top-line findings from the Heparc-2001 Phase 2a clinical study of ARC-520, which is designed to treat chronic hepatitis B infection by inhibiting the production of all HBV gene products.
The study indicated the drug can effectively and consistently knock down target genes in humans that are HBV E-antigen positive.
To date, 84 humans have received ARC-520 and no adverse events have been rated as serious or severe, no discontinuations have occurred due to an adverse event, and no laboratory results have indicated any end organ toxicity, Arrowhead revealed.
Additionally, nine chimps received six-11 monthly doses of ARC-520 and no safety signals were detected in any chimp.
“These are exciting data that represent a significant leap forward for our DPC platform, ARC-520, and the HBV field,” said Christopher Anzalone, president and chief executive officer of Arrowhead.
“We have achieved the highest knock-down ever reported in humans with RNAi and a safety profile that continues to be excellent. We are optimistic that this will ultimately translate into powerful clinical outcomes for ARC-520 and follow-on candidates against multiple indications,” he added.
Shares rose US$1.23 to US$7.97 in the morning session.