Arrowhead Pharmaceuticals Initiates Phase 2 Study of ARC-AAT
September 06 2016 - 7:30AM
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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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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