PASADENA, Calif. — July 23, 2013 — Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced that it has initiated dosing in a Phase 1 clinical trial of ARC-520, the Company’s candidate for the treatment of chronic hepatitis B virus infection. Trial initiation followed successful completion of the Clinical Trial Notification (CTN) regulatory process in Australia. The objectives of the study are to characterize the safety profile of ARC-520, determine maximum tolerated dose, and evaluate pharmacokinetics in healthy volunteers. ARC-520 is the first candidate to use Arrowhead’s proprietary Dynamic Polyconjugate (DPC) delivery platform and includes two distinct siRNA sequences that have pan-genotypic coverage for 99.6% HBV GenBank sequences.
Phase 1 trial is a single-center, randomized, double-blind,
placebo-controlled, single dose-escalation, first-in-human study of
ARC-520 administered intravenously to healthy adult volunteers and is
being conducted in Melbourne, Australia. Each dose cohort includes 6
subjects randomized at ratio of 1:2 (placebo: active) to receive a
single intravenous injection of either placebo or ARC-520. Arrowhead
expects to complete this Phase 1 trial in the fourth quarter of 2013 and
begin a Phase 2a trial in chronic HBV patients in 2014.
Phase 1 study will establish a safety profile for ARC-520 as well as
provide the first human data for our DPC delivery platform. This is an
important step forward as we seek to advance ARC-520 into HBV patients
and build additional RNAi therapeutics based on what we believe is the
most potent delivery system in the industry,” said Dr. Christopher Anzalone, President and Chief Executive Officer.
B is the world’s most common serious liver infection. It is estimated
that 350 million people worldwide are chronically infected with HBV,
representing approximately 1 in 20 people on the planet. No currently
available treatment methods can reliably achieve meaningful cure rates.
ARC-520 is designed to reduce the production of new viral particles and
viral proteins. Many experts believe that reducing key viral proteins
can revive patients’ adaptive immune response and potentially lead to a
functional cure of chronic HBV infection with a finite treatment
regimen. Arrowhead previously presented data generated in rodent models
and in a chimpanzee chronically infected with HBV, showing that ARC-520
induces rapid, deep, and durable knockdown of both circulating HBV DNA
and key viral proteins, including hepatitis B s-antigen, e-antigen, and
the core protein that forms the capsid.
350 million people worldwide are chronically infected with the
hepatitis B virus. Chronic HBV infection can lead to cirrhosis of the
liver and is responsible for 80% of primary liver cancers globally.
Arrowhead’s RNAi-based candidate ARC-520 is designed to treat chronic
HBV infection by reducing the expression and release of new viral
particles and key viral proteins. The goal is to achieve a functional
cure, which is an immune clearant state characterized by hepatitis B
s-antigen negative serum with or without sero-conversion. The siRNAs in
ARC-520 intervene at the point of DNA transcription, upstream of where
nucleotide and nucleoside analogues act. In transient and transgenic
mouse models of HBV infection, a single co-injection of Arrowhead’s DPC
delivery vehicle with cholesterol-conjugated siRNA targeting HBV
sequences resulted in multi-log knockdown of HBV RNA, proteins and viral
DNA with long duration of effect. In a chimpanzee chronically infected
with HBV and high viremia and antigenemia, ARC-520 induced rapid
reductions of 90-95% in HBV DNA, e-antigen, and s-antigen. Arrowhead is
conducting a phase 1 single ascending dose study in normal volunteers,
which the company expects to follow with a phase 2a study in chronic HBV
About Arrowhead Research Corporation
Research Corporation is a biopharmaceutical company developing targeted
RNAi therapeutics. The company is leveraging its proprietary drug
delivery technologies to develop targeted drugs based on the RNA
interference (RNAi) mechanism that efficiently and specifically silence
target genes. Arrowhead technologies also enable partners to create
peptide-drug conjugates (PDCs) that specifically home to cell types of
interest while sparing off-target tissues. Arrowhead’s pipeline includes
programs in chronic hepatitis B virus, obesity, and cancer.
For more information please visit http://www.arrowheadresearch.com, or follow us on Twitter @ArrowRes. To be added to the Company's email list to receive news directly, please send an email to firstname.lastname@example.org