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Arrowhead Research Corporation (NASDAQ: ARWR), a targeted therapeutics company, today announced that its hepatitis B virus (HBV) program has completed all internal preclinical requirements and has named a clinical candidate. As such, the Company has initiated the final IND-enabling steps, including GMP manufacturing, GLP toxicology, and preparation of a pre-IND data package for the US FDA and foreign counterparts. The candidate, named ARC-520, is an RNAi therapeutic actively targeted to the liver using the company’s Dynamic Polyconjugate (DPC) delivery system and includes two siRNA sequences targeting two different regions of the HBV genome.
“Chronic HBV infection is a serious global health problem with no cure, and we believe ARC-520 can potentially have a substantial impact on patient care worldwide,” said Dr. Bruce Given, Arrowhead’s Chief Operating Officer and head of R&D. “We will continue to take an aggressive stance on development timelines for ARC-520. We anticipate filing an IND in Q2 of 2013 and our plan is to conduct a phase 1 clinical trial in chronic HBV carriers to provide early proof of concept.”
The RNA sequences used in the clinical candidate were part of a large-scale screening program initiated by Roche prior to the acquisition by Arrowhead. The RNAs target regions highly conserved across the major HBV genotypes and ARC-520 contains two distinct RNAs as a strategy to minimize the potential development of resistance in individual patients. Consistent with the company’s overarching strategy, the DPCs used in ARC-520 employ active ligand-mediated targeting specific to a receptor on hepatocytes. This approach results in high-potency knockdown of a target gene, validated in mice, rats, and non-human primates and it has demonstrated a low toxicity profile in primates enabling long term dosing.
“The promotion of ARC-520 as a clinical candidate, so soon after acquiring the Roche RNAi assets, represents an important milestone for Arrowhead,” said Dr. Chris Anzalone, President and Chief Executive Officer of Arrowhead. “This program follows in the footsteps of our clinical stage targeted delivery programs in obesity with Adipotide® and in cancer with RONDEL
TM and further validates our commitment to ligand-mediated delivery in fields where most delivery is untargeted. We believe that targeting holds great promise in allowing delivery of currently undeliverable agents, such as many RNAs, and may also dramatically improve the balance of safety and effectiveness for many types of small molecule drugs.”
Highlights from the preclinical development program for ARC-520 have been presented at recent scientific conferences and publication of additional data is anticipated over the coming months in peer-reviewed scientific journals.