Arrowhead Pharmaceuticals Presents ARC-AAT Clinical Data at The International Liver Congress™

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Arrowhead Pharmaceuticals Presents...

PASADENA, Calif.–(BUSINESS WIRE)–

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) presented clinical data

from a Phase 1a/1b study of ARC-AAT, the company’s prior generation

investigational medicine that was being studied for the treatment of

liver disease associated with alpha-1 antitrypsin deficiency (AATD), at

The International Liver Congress™ 2017 (ILC), the annual meeting of the

European Association for the Study of the Liver (EASL).

The data demonstrated that an RNA interference (RNAi) therapeutic can

achieve deep durable, and dose dependent reductions of alpha-1

antitrypsin (AAT), with the highest dose studied in healthy volunteers

achieving near full suppression of the liver production of the AAT

protein. In addition, at doses of 2 and 4 mg/kg in AATD patients,

ARC-AAT produced similar levels of knockdown as seen in healthy

volunteers. These data support the continued advancement of ARO-AAT,

Arrowhead’s follow-on product candidate that utilizes the company’s next

generation, proprietary subcutaneously administered delivery vehicle.

Bruce Given, M.D., chief operating officer and head of R&D for Arrowhead

Pharmaceuticals, said: “The data presented showed strong congruence of

activity in primate studies, in normal healthy volunteers, and in

patients with AATD. We believe these results, together with those from

preclinical studies presented at AASLD last fall that showed that

treatment with ARC-AAT over time may improve liver health and prevent

further damage, provide proof-of-concept for the use of an RNAi

therapeutic against alpha-1 liver disease.”

Presentation materials can be accessed by visiting the Events

section of the Arrowhead website. Additional details including abstracts

can be found on the ILC website at https://ilc-congress.eu/

by searching for the following:

Oral Presentation:

Hepatic targeted RNA interference provides deep and prolonged

knockdown of alpha-1 antitrypsin levels in ZZ patients

  • Presentation Reference: LBO-06

  • Session: Late Breaker session

  • Date and Time: April 22, 2017 at 5:15 PM CET

  • Authors: Alice Turner, et al.

Arrowhead announced on November 29, 2016, that it had discontinued

development of ARC-AAT. The company is currently focused on developing

ARO-AAT, Arrowhead’s follow-on investigational RNAi therapeutic against

alpha-1 liver disease.

The International Liver Congress is a trademark of the European

Association for the Study of the Liver.

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

gene silencing.

For more information, please visit www.arrowheadpharma.com,

or follow us on Twitter @ArrowheadPharma.

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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 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.

Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
626-304-3400
ir@arrowheadpharma.com
or
Investors

and Media:
LifeSci Advisors, LLC
Matthew P. Duffy, 212-915-0685
matthew@lifesciadvisors.com

Source: Arrowhead Pharmaceuticals Inc.

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