Arrowhead Files for Regulatory Permission to Begin Phase 1 Trial of RNAi Therapeutic ARC-AAT
PASADENA, Calif.–(BUSINESS WIRE)–
Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical
company developing targeted RNAi therapeutics, today announced that it
recently filed an application for approval to begin a Phase 1 clinical
trial of its RNAi-based therapeutic candidate, ARC-AAT, for the
treatment of liver disease associated with alpha-1 antitrypsin
deficiency.
Pending approval, Arrowhead intends to proceed with a double blind,
placebo-controlled dose escalation, Phase 1 study to determine the
safety, tolerability, pharmacokinetics and effect on circulating alpha-1
antitrypsin levels of ARC-AAT in healthy volunteers and patients with
alpha-1 antitrypsin deficiency. The study is planned to start in
Australia in healthy volunteers and will dose escalate until
predetermined levels of alpha-1 antitrypsin reduction are reached. Once
these levels of protein knockdown are achieved, the study will
transition into a population of patients with ZZ genotype alpha-1
antitrypsin deficiency to further evaluate escalating doses of ARC-AAT.
The study is designed to enroll up to 48 subjects, including healthy
volunteers and alpha-1 antitrypsin deficiency patients.
The application was submitted to an ethics committee in compliance with
the Clinical Trial Notification process of the Australian Department of
Health and Ageing, Therapeutic Goods Administration.
About ARC-AAT
Arrowhead has developed ARC-AAT 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
has been clearly defined as 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.
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, ARC-AAT for liver disease associated with Alpha-1
antitrypsin deficiency, and partner-based programs in obesity and
oncology.
For more information please visit http://www.arrowheadresearch.com,
or follow us on Twitter @ArrowRes.
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Source: Arrowhead Research Corporation
Arrowhead Research Corporation
Vince Anzalone, CFA
626-304-3400
ir@arrowres.com
or
Investor
Relations:
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Lauren Glaser
646-378-2972
ir@arrowres.com
or
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Martina Schwarzkopf, Ph.D.
212-845-4292
martina.schwarzkopf@russopartnersllc.com
Source: Arrowhead Research Corporation
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