Arrowhead Receives Regulatory Clearance to Begin Additional Phase 2b Studies of Hepatitis B Candidate ARC-520
PASADENA, Calif.–(BUSINESS WIRE)–
Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical
company developing targeted RNAi therapeutics, today announced that its
Clinical Trial Application for ARC-520, its clinical candidate for
chronic hepatitis B infection (HBV), has been approved by Germany’s
Federal Institute for Drugs and Medical Devices. The company now has
regulatory clearance in Germany for two additional Phase 2b
multiple-dose studies of ARC-520 to be conducted in parallel. Arrowhead
awaits final IRB approval from the sites, which are expected shortly,
and in addition, the company is engaged with regulatory authorities from
Hong Kong and South Korea to open additional sites for these studies.
Arrowhead will provide guidance in the future on timing for release of
data from these studies.
“ARC-520 is leading the way in studying the use of RNAi drugs as new
treatment options for chronic HBV. We are very pleased to receive
regulatory clearance in Europe to conduct the parallel design Phase 2b
multiple-dose studies that we proposed,” said Bruce D. Given, M.D.,
Arrowhead’s Chief Operating Officer. “We view these studies as the front
end of a set of global multiple-dose studies designed to identify the
right agents, doses, and regimens that can achieve functional cures.
Based on multiple-dose data from our recently disclosed chimpanzee study
and the long duration of activity ARC-520 has demonstrated in the
single-dose Phase 2a study in patients, we are starting with doses of
1mg/kg and 2mg/kg in parallel. We have incorporated an interim analysis
in both studies to get an early read on whether we may want to
interrogate higher doses. ARC-520 continues to be well tolerated and we
have not seen any signal suggesting that we are approaching a maximum
tolerated dose through 4mg/kg, the highest dose studied in humans to
date.”
The studies titled Heparc-2002 and Heparc-2003 will enroll patients with
chronic HBV infection that are e-antigen (HBeAg) negative and HBeAg
positive, respectively. Each study is a multicenter, randomized,
double-blind, placebo-controlled, multi-dose study intended to evaluate
two dose levels in parallel of ARC-520 in combination with entecavir or
tenofovir in patients with immune active chronic HBV infection. The 2002
and 2003 studies are planned to enroll up to 60 and 90 patients,
respectively. In each study, patients will be randomized to receive four
doses, once every 4 weeks, of either 1 mg/kg ARC-520, 2 mg/kg ARC-520,
or placebo at a ratio of 1:1:1.
For each patient, the duration of the study is approximately 33 weeks,
from screening to the Day 169 follow-up visit. For each study, a
planning interim analysis will be performed when 54 patients have
completed their Day 71 visit. This analysis will be used to plan future
studies only and will not impact the enrollment or conduct of
Heparc-2002 and -2003. Based on the interim results, the company will
decide whether higher multiple-doses would be likely to lead to
increased activity, in which case higher doses may be evaluated in one
or more additional studies.
The primary objective of the 2002 and 2003 studies is to evaluate the
depth of hepatitis B surface antigen (HBsAg) decline in response to
multiple doses of ARC-520 compared to placebo as a measure of drug
activity. Secondary objectives include the assessment of safety and
tolerability and multi-dose pharmacokinetics of ARC-520 when
co-administered with entecavir or tenofovir, in addition to other
exploratory objectives.
About ARC-520
Arrowhead’s RNAi-based candidate ARC-520 is being investigated in the
treatment of chronic HBV infection. The small interfering RNAs (siRNAs)
in ARC-520 intervene at the mRNA level, upstream of the reverse
transcription process where current standard of care nucleotide and
nucleoside analogues act. Arrowhead is investigating ARC-520
specifically to determine if it can be used to achieve a functional
cure, which is an immune clearant state characterized by hepatitis B
s-antigen negative serum with or without sero-conversion. Arrowhead has
completed a Phase 1 single ascending dose study in normal volunteers and
the company is conducting single dose Phase 2a studies and multiple dose
Phase 2b studies in chronic HBV patients. Approximately 350-400 million
people worldwide are chronically infected with the hepatitis B virus,
which can lead to cirrhosis of the liver and is responsible for 80% of
primary liver cancers globally.
About Arrowhead Research Corporation
Arrowhead Research Corporation is a biopharmaceutical company developing
targeted RNAi therapeutics. The company is leveraging its proprietary
Dynamic Polyconjugate™ delivery platform to develop targeted drugs based
on the RNA interference mechanism that efficiently silences
disease-causing genes. Arrowhead’s pipeline includes ARC-520 for chronic
hepatitis B virus and ARC-AAT for liver disease associated with Alpha-1
antitrypsin deficiency.
For more information please visit http://www.arrowheadresearch.com,
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Act:
This news release contains forward-looking statements within the
meaning of the “safe harbor” provisions of the Private Securities
Litigation Reform Act of 1995. These statements are based upon our
current expectations and speak only as of the date hereof. Our actual
results may differ materially and adversely from those expressed in any
forward-looking statements as a result of various factors and
uncertainties, including our ability to finance our operations, the
future success of our scientific studies, our ability to successfully
develop drug candidates, the timing for starting and completing clinical
trials, rapid technological change in our markets, and the enforcement
of our intellectual property rights. Arrowhead Research Corporation’s
most recent Annual Report on Form 10-K and subsequent Quarterly Reports
on Form 10-Q discuss some of the important risk factors that may affect
our business, results of operations and financial condition. We assume
no obligation to update or revise forward-looking statements to reflect
new events or circumstances.
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Corporation.
Source: Arrowhead Research Corporation
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626-304-3400
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Source: Arrowhead Research Corporation
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