Arrowhead’s ARC-AAT Granted EMA Orphan Drug Designation
January 19 2016 - 7:30AM
Business Wire
Arrowhead Research Corporation (NASDAQ: ARWR), a
biopharmaceutical company developing targeted RNAi therapeutics,
today announced that the European Medicines Agency (EMA) granted
Orphan Drug Designation to ARC-AAT for treatment of congenital
alpha-1 antitrypsin deficiency. ARC-AAT was also previously granted
Orphan Drug Designation by the United States Food and Drug
Administration (FDA) in 2015.
Orphan Drug Designation from the EMA allows Arrowhead to benefit
from a number of incentives, such as reduced regulatory fees,
protocol assistance, and market exclusivity, to develop a medicine
for the treatment of a rare disease affecting not more than five in
10,000 people in the European Union.
“ARC-AAT has the potential to provide patients and physicians
with a much needed treatment option for liver disease associated
with alpha-1 antitrypsin deficiency, a rare genetic disorder. We
are pleased that the EMA has granted Orphan Drug Designation and we
view this as an important regulatory milestone for the program,”
said Bruce Given, M.D., Arrowhead chief operating officer.
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 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 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 and ARC-521 for chronic hepatitis B virus, ARC-AAT
for liver disease associated with alpha-1 antitrypsin deficiency,
ARC-F12 for hereditary angioedema and thromboembolic diseases, and
ARC-HIF2 for renal cell carcinoma.
For more information please visit
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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, the timing of any market
authorization, the timing of any market authorization for
competitive products, 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
GroupChad Rubin646-378-2947ir@arrowres.comorMedia:Russo
PartnersMatt Middleman,
M.D.212-845-4272matt.middleman@russopartnersllc.com
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