Arrowhead Pharmaceuticals Initiates Phase 1/2 Single and Multiple Dose Study of Hepatitis B Candidate ARC-521
09 June 2016 - 9:30PM
Business Wire
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced
that it initiated a Phase 1/2 study of ARC-521, the company’s
second RNAi-based investigational medicine for the treatment of
chronic hepatitis B virus (HBV) infection. The study is designed to
evaluate the safety, tolerability, and pharmacokinetics of single
doses of ARC-521 in healthy volunteers and the safety,
tolerability, and antiviral activity of single and multiple doses
of ARC-521 in patients with chronic HBV. This study may have
multiple readouts, including single dose safety data, single dose
antiviral activity data, and multiple dose safety and antiviral
activity data.
Bruce Given, M.D., Arrowhead chief operating officer and head of
R&D said, “Arrowhead is a clear leader in the search for
curative therapies for chronic hepatitis B, which affects up to
350-400 million people worldwide. Designed to complement ARC-520,
ARC-521 targets HBV mRNA transcripts from both cccDNA and
integrated DNA and is expected to be most suitable for those
patients who tend to have lower levels of viral cccDNA. We have
moved very fast with this program, and we think ARC-521 increases
our leadership position in the HBV space and the race to a
functional cure. Our experience working with HBV investigators, key
opinion leaders, and patients around the world has helped guide the
design of an innovative first-in-man study for ARC-521 that is
intended to get to multiple dose data in patients rapidly.”
The ARC-521 study (ARC5211001) is a multicenter,
placebo-controlled, double-blind, dose escalation study of ARC-521
in healthy volunteers including a sequential, multiple dose, open
label part in patients with chronic HBV. Normal healthy volunteers
will enroll sequentially into a total of 6 escalating dose levels
(6 subjects per dose level), randomized to receive a single dose of
ARC-521 or placebo. Chronic HBV patients that are negative for
Hepatitis B e-antigen (HBeAg) at screening will enroll sequentially
into 3 dose levels (8 patients per dose level) to receive 3 monthly
doses of open label ARC-521. The study is currently recruiting at a
single center in New Zealand and the company plans to add
additional centers in other countries, pending regulatory and
ethics review.
About ARC-521
Arrowhead’s ARC-521 is being investigated for its potential to
produce functional cures in patients with chronic hepatitis B virus
(HBV) infection. ARC-521 intervenes upstream of the reverse
transcription process where current standard-of-care nucleotide and
nucleoside analogs act, and is designed to silence the production
of all HBV gene products. The small interfering RNAs (siRNAs) in
ARC-521 engage the body’s normal cellular RNAi machinery and direct
specific cleavage of HBV RNA transcripts, thereby reducing the
levels of HBV proteins and the RNA template used to produce viral
DNA. Designed to complement ARC-520, ARC-521 is a second generation
HBV candidate that targets HBV mRNA transcripts from both cccDNA
and integrated DNA and is expected to be most suitable for those
patients who tend to have lower levels of viral cccDNA. Arrowhead
is investigating ARC-521 specifically to determine if significantly
reducing circulating and non-circulating viral proteins and RNA
will allow for re-constitution of an effective host immune response
and ultimately HBsAg seroclearance resulting in functional cure. As
many as 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.
Arrowhead is conducting a Phase 1/2 single and multiple dose study
in healthy volunteers and HBV patients.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals develops medicines that treat
intractable diseases by silencing the genes that cause them. Using
a broad portfolio of RNA chemistries and efficient modes of
delivery, Arrowhead therapies trigger the RNA interference
mechanism to induce rapid, deep, and durable knockdown of target
genes. RNA interference, or RNAi, is a mechanism present in living
cells that inhibits the expression of a specific gene, thereby
affecting the production of a specific protein. Arrowhead’s
RNAi-based therapeutics leverage this natural pathway of gene
silencing. The company’s pipeline includes ARC-520 and ARC-521 for
chronic hepatitis B virus infection, ARC-AAT for liver disease
associated with alpha-1 antitrypsin deficiency, ARC-F12 for
hereditary angioedema and thromboembolic disorders, ARC-LPA for
cardiovascular disease, and ARC-HIF2 for renal cell carcinoma.
For more information please visit www.arrowheadpharma.com, or
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Safe Harbor Statement under the Private Securities Litigation
Reform 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. Our most recent Annual Report on Form 10-K and subsequent
Quarterly Reports on Form 10-Q discuss some of the important risk
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forward-looking statements to reflect new events or
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DYNAMIC POLYCONJUGATES is a trademark of Arrowhead
Pharmaceuticals, Inc.
Source: Arrowhead Pharmaceuticals, Inc.
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Arrowhead Pharmaceuticals, Inc.Vince Anzalone,
CFA626-304-3400ir@arrowheadpharma.comorInvestor
Relations:The Trout GroupChad
Rubin646-378-2947ir@arrowheadpharma.comorMedia:Russo
PartnersMatt Middleman,
M.D.212-845-4272matt.middleman@russopartnersllc.com
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