Arrowhead Pharmaceuticals to Present Data on ARC-AAT at The Liver Meeting®
October 20 2016 - 10:00AM
Business Wire
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced
that it will make two presentations on ARC-AAT, the company’s
investigational medicine for the treatment of liver disease
associated with alpha-1 antitrypsin deficiency (AATD), at The Liver
Meeting® 2016, the Annual Meeting of the American Association for
the Study of Liver Disease (AASLD) being held on November 11-15,
2016, in Boston.
“At The Liver Meeting® we will present initial results from Part
A of a Phase 1 study indicating that ARC-AAT is well-tolerated and
induces deep, durable, and dose-dependent knockdown of circulating
alpha-1 antitrypsin. In addition, we will present mouse model data
showing that over time ARC-AAT had the desired effect of preventing
liver production of mutant Z-alpha1 antitrypsin polymers and
reversing liver disease,” said Bruce Given, M.D., chief operating
officer and head of R&D at Arrowhead. “The clinical abstract
shows that ARC-AAT achieved reductions in serum AAT of up to 90%.
Our previously reported primate studies indicate that approximately
10% of AAT may be produced outside the liver, so the Phase 1
clinical results suggest that we achieved near complete suppression
of the liver produced AAT. This was an exciting result that gives
us confidence as we conduct our ongoing Phase 2 multiple-dose
study, which includes biopsies that will allow us to assess
ARC-AAT’s histological effects.”
Oral Presentation:
RNA interference therapeutic ARC-AAT prevents production of
Z-alpha1 antitrypsin polymers and reverses liver disease phenotype
in PiZ mouse model
- Publication Number: 124
- Session: Parallel 19: Pediatric and
Metabolic Liver Diseases: Basic and Translational
- Session Date and Time: November 13,
2016 from 4:45 PM to 6:15 PM
- Authors: Christine Wooddell, et
al.
Poster Presentation:
RNA interference (RNAi) with ARC-AAT provides deep and
prolonged knockdown of alpha-1 antitrypsin levels in healthy
volunteers
- Publication Number: LB-24
- Session: Late-Breaking Poster
Session
- Session Date and Time: November 13,
2016 from 8:00 AM to 5:30 PM
- Authors: Alice Turner, et al.
Additional details including presentation abstracts can be found
on the AASLD website at http://www.aasld.org/. A copy of
presentation materials can be accessed by visiting the Events
section of the Arrowhead website after the presentations
conclude.
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 studies. 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. 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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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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