Arrowhead Receives Orphan Drug Designation for ARC-AAT
June 10 2015 - 12:27PM
Business Wire
Arrowhead Research Corporation (NASDAQ: ARWR), a
biopharmaceutical company developing targeted RNAi therapeutics,
today announced the United States Food and Drug Administration
(FDA) has granted ARC-AAT orphan drug designation. ARC-AAT is
Arrowhead’s RNAi-based therapeutic candidate 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 children and adults. Arrowhead is
currently conducting part B of a Phase 1 study of ARC-AAT in
patients with PiZZ genotype AATD.
"Receiving orphan drug designation is an important milestone in
the development of ARC-AAT, which we think is a very promising
program aimed at providing a better option for patients with liver
disease associated with alpha-1 antitrypsin deficiency,” said Bruce
D. Given, M.D., Arrowhead’s Chief Operating Officer. “The Orphan
Drug Act provides important incentives for sponsors to develop
drugs that treat rare diseases and we look forward to more
engagement with the FDA as the development of ARC-AAT
progresses.”
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. The study evaluates participants for 28 days
following dosing, with additional follow-up if needed every 2 weeks
until AAT levels return to baseline.
The FDA Office of Orphan Products Development (OOPD) mission is
to advance the evaluation and development of products that
demonstrate promise for the diagnosis and/or treatment of rare
diseases or conditions. In fulfilling that task, OOPD evaluates
scientific and clinical data submissions from sponsors to identify
and designate products as promising for rare diseases and to
further advance scientific development of such promising medical
products. Orphan drug designation provides incentives for sponsors
to develop products for rare diseases. These incentives include
increased engagement with FDA on drug development activities,
exemption from all future product-specific regulatory fees, the
opportunity to apply for R&D funding, tax credits, an increased
chance of priority review, and 7 years of orphan exclusivity at
time of New Drug Application (NDA) approval.
About Alpha-1 Antitrypsin Deficiency (AATD)
AATD is an autosomal recessive genetic disorder associated with
liver disease in children and adults and pulmonary disease in
adults. Alpha-1 antitrypsin is a circulating glycoprotein protease
inhibitor of the serpin family encoded by the AAT gene and
primarily synthesized in the liver. The physiologic function is
inhibition of neutrophil proteases to protect healthy tissues
during inflammation and prevent tissue damage. The Z mutant is the
most common disease variant and has a single amino acid
substitution that results in improper protein folding causing
severe impairment of secretion from hepatocytes. This lack of
secretion leads to accumulation of mutant Z-AAT polymers, which
form globules in the hepatocyte endoplasmic reticulum. This
triggers continuous hepatocyte injury, leading to fibrosis,
cirrhosis, and increased risk of hepatocellular carcinoma.
In clinical practice, approximately 96-98% of AATD-related
disease is due to the homozygous PiZZ genotype. PiZZ individuals
have severe deficiency of functional AAT leading to pulmonary
disease and hepatocyte injury and liver disease. Lung disease is
frequently treated with AAT augmentation therapy. However,
augmentation therapy does nothing to treat liver disease, and there
is no specific therapy for hepatic manifestations. There is a
significant unmet need as liver transplant is currently the only
available treatment for severe liver manifestations.
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 an orphan
disease that appears to be frequently misdiagnosed or undiagnosed.
European prevalence is estimated to be 1 per 2500.
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. 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. Reduction of liver
production of the inflammatory Z-AAT protein, which is likely a
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. The Company is conducting
a single dose Phase 1 clinical study, 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.
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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