Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1/2 Study of ARC-521
April 28 2016 - 6:30AM
Business Wire
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR), recently filed a
regulatory submission in New Zealand to begin a Phase 1/2 clinical
trial of ARC-521, its RNAi-based therapeutic candidate for the
treatment of chronic hepatitis B virus (HBV) infection. Pending
approval, Arrowhead intends to proceed with ARC521-1001, a
first-in-human study to assess single and multiple-doses of ARC-521
in healthy volunteers and HBV patients.
Chris Anzalone, Ph.D., president and CEO of Arrowhead
Pharmaceuticals, said: “ARC-521 is our second pipeline product
targeting chronic HBV and was designed to silence gene products
from both HBV cccDNA and integrated HBV DNA. This is important
because our clinical work with ARC-520 in HBV patients and our
long-term chimpanzee study suggest that different patient groups
can have different relative levels of cccDNA. We think having both
ARC-520, which has been very active in patients with higher cccDNA,
and ARC-521, which may be optimal for those with lower cccDNA,
should provide us with the potential to treat all patients with
HBV. We have an aggressive plan for the development of ARC-521 that
includes an accelerated first-in-man Phase 1/2 design intended to
allow rapid transition into multi-dose patient cohorts.”
The application for approval of a clinical trial was submitted
to the New Zealand Medicines and Medical Devices Safety Authority
(MEDSAFE) for review by the Standing Committee on Therapeutic
Trials (SCOTT). Arrowhead also intends to seek regulatory clearance
to conduct ARC521-1001 in additional countries.
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 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 planning to conduct
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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DYNAMIC POLYCONJUGATES is a trademark of Arrowhead
Pharmaceuticals, Inc.
Source: Arrowhead Pharmaceuticals, Inc.
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version on businesswire.com: http://www.businesswire.com/news/home/20160428005640/en/
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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