Arrowhead Initiates Dosing of ARC-AAT in Patients with Alpha-1 Antitrypsin Deficiency
July 02 2015 - 7:30AM
Business Wire
Arrowhead Research Corporation (NASDAQ: ARWR), a
biopharmaceutical company developing targeted RNAi therapeutics,
today announced that it dosed the first patient in Part B of a
Phase 1 clinical trial of ARC-AAT. ARC-AAT is Arrowhead’s
RNAi-based drug candidate for the treatment of liver disease
associated with the rare genetic disorder alpha-1 antitrypsin
deficiency (AATD) that was recently granted orphan drug designation
by the United States Food and Drug Administration. The clinical
trial is currently enrolling patients at a single center in
Australia and the company intends to open additional sites for
enrollment in Europe, pending regulatory permission to proceed. The
company expects to complete enrollment of the Phase 1 study by the
end of 2015.
"Dosing the first alpha-1 patient with ARC-AAT is a milestone
for Arrowhead and for patients with AATD. The goal of treatment
with ARC-AAT is to halt progression and possibly reverse the liver
injury and fibrosis associated with AATD, which currently has no
approved therapy short of liver transplant. This is becoming a
larger clinical problem that we believe ARC-AAT holds great
potential to address,” said Bruce D. Given, M.D., Arrowhead’s Chief
Operating Officer. “We would also like to thank the Alpha-1
Foundation and The Alpha-1 Project, who have agreed to help support
the development of ARC-AAT through funding and assistance with
patient recruitment.”
The ongoing Phase 1 trial of ARC-AAT is a multi-center,
randomized, placebo-controlled, double-blind, single
dose-escalation, first-in-human study to evaluate the safety,
tolerability and pharmacokinetics of ARC-AAT and the effect on
circulating AAT levels. The study has been enrolling in dose
cohorts of six participants each, with participants randomized at a
ratio of 2:1 (active:placebo) to receive a single intravenous
injection of either ARC-AAT or placebo (normal saline). The study
consists of two parts; Part A in healthy volunteers, which has been
completed, and Part B to be conducted in patients with PiZZ
genotype AATD. Dosing in patients begins at the highest dose level
used in healthy volunteers and then continued dose escalation may
proceed under the protocol. The study evaluates participants for 28
days following dosing, with additional follow-up if needed every 2
weeks until AAT levels return to baseline.
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 appears to be frequently
misdiagnosed or undiagnosed. ARC-AAT, which was granted orphan drug
designation, 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 animals. 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. Arrowhead is conducting a single dose
Phase 1 clinical study of ARC-AAT, with part A in healthy
volunteers and part B in AATD patients.
About Arrowhead Research Corporation
Arrowhead Research Corporation is a biopharmaceutical company
developing targeted RNAi therapeutics. The company is leveraging
its proprietary Dynamic Polyconjugate™ delivery platform to develop
targeted drugs based on the RNA interference mechanism that
efficiently silences disease-causing genes. Arrowhead’s pipeline
includes ARC-520 for chronic hepatitis B virus and ARC-AAT for
liver disease associated with Alpha-1 antitrypsin deficiency.
For more information please visit
http://www.arrowheadresearch.com, or follow us on Twitter
@ArrowRes. To be added to the Company's email list and receive news
directly, please visit
http://ir.arrowheadresearch.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. Arrowhead Research Corporation's 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
Research Corporation.
Source: Arrowhead Research Corporation
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Arrowhead Research CorporationVince Anzalone,
CFA626-304-3400ir@arrowres.comorInvestor Relations:The Trout
GroupTodd James646-378-2926ir@arrowres.comorMedia:Russo
PartnersMatt Middleman,
M.D.212-845-4272matt.middleman@russopartnersllc.com
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