PASADENA, Calif.--(BUSINESS WIRE)--
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it
filed a regulatory submission to begin a first-in-human study of
ARO-AAT, a second-generation investigational medicine for the treatment
of alpha-1 liver disease that leverages Arrowhead's subcutaneously
administered Targeted RNAi Molecule (TRiMTM) technology.
Pending approval, Arrowhead intends to proceed with AROAAT1001, a Phase
1 single and multiple ascending dose study to evaluate the safety,
tolerability, pharmacokinetics, and effect of ARO-AAT on serum alpha-1
antitrypsin levels in healthy adult volunteers.
Chris Anzalone, Ph.D., president and CEO of Arrowhead Pharmaceuticals,
said: "ARO-AAT is our second candidate targeting alpha-1 liver disease
and represents the first of five planned regulatory submissions over the
next twelve months for product candidates built on our TRiMTM
platform. We have moved very rapidly with this program and are thrilled
to now submit the regulatory filing ahead of schedule."
The application for approval of the clinical trial was submitted to the
New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) for
review by the Standing Committee on Therapeutic Trials (SCOTT) as well
as the local Ethics Committee.
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
For more information, please visit www.arrowheadpharma.com,
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Safe Harbor Statement under the Private Securities Litigation Reform
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 the safety and efficacy of our product
candidates, the duration and impact of regulatory delays in our clinical
programs, 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.
Source: Arrowhead Pharmaceuticals, Inc.
View source version on businesswire.com: http://www.businesswire.com/news/home/20171220005091/en/
Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
LifeSci Advisors, LLC
Source: Arrowhead Pharmaceuticals Inc.
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