Arrowhead Pharmaceuticals to Present Data on ARC-AAT at The Liver Meeting®

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Arrowhead Pharmaceuticals to Present...

PASADENA, Calif.–(BUSINESS WIRE)–

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it

will make two presentations on ARC-AAT, the company’s investigational

medicine for the treatment of liver disease associated with alpha-1

antitrypsin deficiency (AATD), at The Liver Meeting® 2016,

the Annual Meeting of the American Association for the Study of Liver

Disease (AASLD) being held on November 11-15, 2016, in Boston.

“At The Liver Meeting® we will present initial results from

Part A of a Phase 1 study indicating that ARC-AAT is well-tolerated and

induces deep, durable, and dose-dependent knockdown of circulating

alpha-1 antitrypsin. In addition, we will present mouse model data

showing that over time ARC-AAT had the desired effect of preventing

liver production of mutant Z-alpha1 antitrypsin polymers and reversing

liver disease,” said Bruce Given, M.D., chief operating officer and head

of R&D at Arrowhead. “The clinical abstract shows that ARC-AAT achieved

reductions in serum AAT of up to 90%. Our previously reported primate

studies indicate that approximately 10% of AAT may be produced outside

the liver, so the Phase 1 clinical results suggest that we achieved near

complete suppression of the liver produced AAT. This was an exciting

result that gives us confidence as we conduct our ongoing Phase 2

multiple-dose study, which includes biopsies that will allow us to

assess ARC-AAT’s histological effects.”

Oral Presentation:

RNA interference therapeutic ARC-AAT prevents production of Z-alpha1

antitrypsin polymers and reverses liver disease phenotype in PiZ mouse

model

  • Publication Number: 124

  • Session: Parallel 19: Pediatric and Metabolic Liver Diseases: Basic

    and Translational

  • Session Date and Time: November 13, 2016 from 4:45 PM to 6:15 PM

  • Authors: Christine Wooddell, et al.

Poster Presentation:

RNA interference (RNAi) with ARC-AAT provides deep and prolonged

knockdown of alpha-1 antitrypsin levels in healthy volunteers

  • Publication Number: LB-24

  • Session: Late-Breaking Poster Session

  • Session Date and Time: November 13, 2016 from 8:00 AM to 5:30 PM

  • Authors: Alice Turner, et al.

Additional details including presentation abstracts can be found on the

AASLD website at http://www.aasld.org/.

A copy of presentation materials can be accessed by visiting the Events

section of the Arrowhead website after the presentations conclude.

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 is 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 studies. 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. 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 (now complete) and part B in

AATD patients, and a Phase 2 multiple dose study in AATD patients.

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. The company’s pipeline includes ARC-520 and ARC-521 for

chronic hepatitis B virus infection, ARC-AAT for liver disease

associated with alpha-1 antitrypsin deficiency, ARC-F12 for hereditary

angioedema and thromboembolic disorders, ARC-LPA for cardiovascular

disease, and ARC-HIF2 for renal cell carcinoma.

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

or follow us on Twitter @ArrowheadPharma.

To be added to the Company’s email list and receive news directly,

please visit http://ir.arrowheadpharma.com/alerts.cfm.

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, 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.

DYNAMIC POLYCONJUGATES is a trademark of Arrowhead

Pharmaceuticals, Inc.

Source: Arrowhead Pharmaceuticals, Inc.

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

Relations:
The Trout Group
Chad Rubin
646-378-2947
ir@arrowheadpharma.com
or
Media:
Russo

Partners
Matt Middleman, M.D.
212-845-4272
matt.middleman@russopartnersllc.com

Source: Arrowhead Pharmaceuticals, Inc.

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