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PASADENA, Calif.–(BUSINESS WIRE)–Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it initiated a Phase 2 clinical study of ARC-AAT, an investigational RNAi-based medicine for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD). The study is designed to evaluate safety and tolerability and determine the effect of multiple doses of ARC-AAT on levels …

PASADENA, Calif.–(BUSINESS WIRE)–Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it
initiated a Phase 2 clinical study of ARC-AAT, an investigational
RNAi-based medicine for the treatment of liver disease associated with
alpha-1 antitrypsin deficiency (AATD). The study is designed to evaluate
safety and tolerability and determine the effect of multiple doses of
ARC-AAT on levels of circulating and intrahepatic alpha-1 antitrypsin as
evidenced by changes in liver biopsy in patients with AATD.
Bruce Given, M.D., chief operating officer and head of R&D at Arrowhead
said, “There remains no medical treatment for the liver disease
associated with AATD, which is increasingly being recognized by patients
and physicians as a serious problem. Our Phase 2 2001 study should give
us, and the AATD community in general, the first insights into whether
ARC-AAT can stop the progression of liver disease and possibly even
allow the liver to recover and heal existing damage. This would be a
significant breakthrough for patients.”
The ARC-AAT Phase 2 study (ARCAAT2001) is a multi-center, open-label,
multiple dose-escalation study of ARC-AAT in patients with AATD. In
total, the study will consist of at least 8 and a maximum of 12
participants. Two dose levels, 4 mg/kg and 6 mg/kg, will be evaluated in
two separate cohorts. All subjects will receive a pre-dose biopsy, seven
doses of ARC-AAT (once every 28 days), and a post-dose biopsy completed
at Day 183. The study will be conducted at multiple centers in Canada,
Ireland, and Sweden. The Company may add additional centers in other
countries, pending regulatory and ethics review.
About ARC-AAT
Arrowhead’s ARC-AAT is being investigated for the treatment of liver
disease associated with alpha-1 antitrypsin deficiency (AATD), a rare
genetic disease that severely damages the liver and lungs of affected
individuals. The mean estimated prevalence of AATD in the U.S is 1 per
3000-5000, or approximately 100,000 patients. AATD is also an important
cause of pediatric liver disease with an estimated prevalence in
children of approximately 20,000 patients, and 50-80% likely to manifest
liver disease during childhood. It is a rare disease that is frequently
misdiagnosed or undiagnosed. ARC-AAT employs a novel unlocked nucleobase
analog (UNA) containing RNAi trigger molecule designed for systemic
delivery using the Dynamic Polyconjugate delivery system.
ARC-AAT is highly effective at knocking down the Alpha-1 antitrypsin
(AAT) gene transcript and reducing the hepatic production of the mutant
AAT (Z-AAT) protein in animal models. Reduction of liver production of
the inflammatory Z-AAT protein, which is believed to be the cause of
progressive liver disease in AATD patients, is important as it is
expected to halt the progression of liver disease and potentially allow
fibrotic tissue repair. ARC-AAT was granted orphan drug designation in
both the United States and in Europe, the latter being held on
Arrowhead’s behalf by a local EU representative Pharma Gateway AB.
Arrowhead is conducting a Phase 1 clinical study of ARC-AAT, with part A
in healthy volunteers (now complete) and part B in AATD patients, and a
Phase 2 multiple dose study in AATD 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 follow us on Twitter @ArrowheadPharma.
To be added to the Company’s email list and receive news directly,
please visit
http://ir.arrowheadpharma.com/alerts.cfm.
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 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.

DYNAMIC POLYCONJUGATES is a trademark of Arrowhead
Pharmaceuticals, Inc.
Source: Arrowhead Pharmaceuticals, Inc.

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