Arrowhead Presents Promising Preclinical Data on Development of ARO-AAT for Treatment of Alpha-1 Liver Disease at Liver Meeting® 2017

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Arrowhead Presents Promising Preclinical...

Data Presented in Late Breaking Poster

Company Expects to File Clinical Trial Application in First Quarter

2018 to Begin First-in-Human Studies of ARO-AAT

PASADENA, Calif.–(BUSINESS WIRE)–

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today presented data from

studies demonstrating promising preclinical safety and efficacy 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.

Based on these positive results, and pending completion of GLP

toxicology studies, Arrowhead expects to file a clinical trial

application in the first quarter of 2018 to begin first-in-human studies

of ARO-AAT. The data were presented in a late breaking poster at the

Liver Meeting® 2017, the Annual Meeting of the American

Association for the Study of Liver Disease (AASLD), being held in

Washington, D.C.

The poster presentation, titled, “Subcutaneous delivery of a RNA

interference (RNAi) therapeutic candidate for alpha-1 antitrypsin

deficiency (AATD)-related liver disease produces deep and prolonged

knockdown of plasma AAT,” highlighted the results of studies in which

ARO-AAT produced deep and prolonged knockdown of alpha-1 antitrypsin

(AAT) to levels that appear to be near full suppression of the liver

production. The efficacy of ARO-AAT was evaluated in the transgenic PiZ

mouse model and in nonhuman primates (NHPs) by measuring the reduction

in plasma or serum levels of AAT. Inhibition of synthesis and subsequent

reduction of the mutant Z-AAT accumulation in the livers of patients

with alpha-1 liver disease may lead to the prevention, and potential

reversal, of liver injury.

Preliminary safety of ARO-AAT was also assessed in rats and NHPs at dose

levels up to and including 300 mg/kg, which is approximately 100 times

the expected human clinical dose. In the rat study, clinical laboratory

values were indistinguishable between groups receiving ARO-AAT and

control groups. In addition, there were no histopathology findings

deemed to be related to ARO-AAT. In the NHP study, there were no

abnormal clinical observations, body weight changes, clinical

chemistries, nor organ weight findings noted.

Poster Details:

Subcutaneous delivery of a RNA interference (RNAi) therapeutic

candidate for alpha-1 antitrypsin deficiency (AATD)-related liver

disease produces deep and prolonged knockdown of plasma AAT

  • Publication Number: LB-21

  • Session: Late-Breaking Poster Session

  • Session Date and Time: October 23, 2017, from 8:00 AM to 5:30 PM

  • Authors: Christine I. Wooddell, et al.

A copy of the poster may be accessed on the Events

and Presentations page under the Investors section of the Arrowhead

website.

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.

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 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
Brian Ritchie
212-915-2578
britchie@lifesciadvisors.com
www.lifesciadvisors.com

Source: Arrowhead Pharmaceuticals Inc.

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