Arrowhead Files to Begin Phase 1 Trial of RNAi Therapeutic ARC-520 Against Chronic Hepatitis B Infection

  Arrowhead Files to Begin Phase 1 Trial of RNAi Therapeutic ARC-520 Against
  Chronic Hepatitis B Infection

Business Wire

PASADENA, Calif. -- May 28, 2013

Arrowhead Research Corporation (NASDAQ: ARWR), a targeted therapeutics
company, today announced that it recently filed an application for approval to
begin a phase 1 clinical trial of its RNAi-based therapeutic candidate,
ARC-520, against chronic hepatitis B virus infection. Pending approval,
Arrowhead intends to proceed with a phase 1, randomized, double-blind,
placebo-controlled, dose-escalating study to evaluate the safety, tolerability
and pharmacokinetics of ARC-520 in normal adult volunteers.

The application was submitted to an ethics committee in compliance with the
Clinical Trial Notification process of the Australian Department of Health and
Ageing, Therapeutic Goods Administration.

“We are pleased to meet our stated goal of a regulatory submission during the
second quarter. This represents an important milestone for Arrowhead as we
move our HBV candidate, ARC-520, and the DPC siRNA delivery system towards
first-in-man studies,” said Dr. Christopher Anzalone, President and Chief
Executive Officer.

About ARC-520

Hepatitis B virus is the world’s most common serious liver infection, with an
estimated 350 million patients worldwide that are chronically infected. There
are thought to be approximately 2 million patients in the U.S., 14 million in
Western Europe, over 100 million in the Asia Pacific Region, and another 220+
million throughout the rest of the world. HBV can lead to cirrhosis of the
liver and is responsible for 80% of primary liver cancers globally. The annual
death toll for HBV is estimated as high as one million. Arrowhead’s RNAi-based
candidate ARC-520 has the potential to treat chronic hepatitis B virus
infection in a fundamentally different manner than current therapies. 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, and
can deeply knockdown all HBV gene products, including proteins and the viral
intermediates necessary to produce viral DNA. In transient and transgenic
mouse models of HBV infection, a single co-injection of the 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. Arrowhead is on track to conduct a phase 1 single ascending dose study
in normal volunteers in mid-2013, which the company expects will be followed
by a phase 2a study in chronic HBV patients in Hong Kong.

About Arrowhead Research Corporation

Arrowhead Research Corporation is a clinical stage targeted therapeutics
company with development programs in oncology, obesity, and chronic hepatitis
B virus. The company is leveraging its platform technologies to design and
develop peptide-drug conjugates (PDCs) that specifically home to cell types of
interest while sparing off-target tissues, create targeted drugs based on the
gene silencing RNA interference (RNAi) mechanism, and work with partners to
create improved versions of traditional small molecule drugs.

For more information please visit, or follow
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Arrowhead Research Corporation
Vince Anzalone, CFA
The Trout Group
Lauren Glaser
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