Arrowhead Nominates ARC-HIF2 As First RNAi Therapeutic Candidate Using Extrahepatic DPC™ Delivery System

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Arrowhead Nominates ARC-HIF2 As...

Data to be presented September 27 at the European Cancer Conference

2015 in Vienna

PASADENA, Calif.–(BUSINESS WIRE)–

Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical

company developing targeted RNAi therapeutics, today announced that it

has nominated ARC-HIF2 as its first therapeutic candidate delivered

using a new Dynamic Polyconjugate™ (DPC™) designed to target tissues

outside of the liver. Arrowhead believes that ARC-HIF2, which uses RNA

interference to silence transcription factor hypoxia-inducible factor 2α

(HIF-2α), is a promising new candidate for the treatment of clear cell

renal cell carcinoma (ccRCC). The company will present preclinical data

at the European Cancer Congress 2015 (ECC2015) in Vienna on September 27

in a session starting at 16:45 CEST. In a poster titled “HIF-2α

targeting with a novel RNAi delivery platform as therapy for renal cell

carcinoma,” (abstract #353), Arrowhead scientists will show data

suggesting that HIF-2α inhibition through RNA interference may

significantly impact late stage ccRCC progression. The company is in the

process of manufacturing scale up to allow for initiation of

IND-enabling studies. Timing for anticipated regulatory submission will

be announced in the future.

“Preclinical data using our new extrahepatic DPC™ delivery system has

been very promising. We think the ability to target tissues outside of

the liver, including tumors, opens additional opportunities for

Arrowhead to develop differentiated RNAi-therapeutics that address

numerous diseases without adequate treatment options,” said Christopher

Anzalone, Ph.D., Arrowhead’s president and chief executive officer.

“This is an important milestone for Arrowhead and we look forward to

continued development of the DPC™ delivery platform and product

candidates based on it.”

ARC-HIF2 is designed to inhibit the production of HIF-2α, which has been

linked to tumor progression and metastasis in ccRCC. ARC-HIF2 employs a

novel extrahepatic-targeted DPC™ that comprises a membrane active

polymer to promote RNAi trigger endosomal release, an active ligand that

targets the DPC™ to tumor cells, reversible masking to prevent polymer

activity prior to cellular uptake, and an RNAi trigger to HIF-2α

conjugated directly to the DPC™.

Using ARC-HIF2 in a preclinical ccRCC tumor model, mice treated with

weekly injections led to greater than 80% knockdown of HIF-2α mRNA in

tumors. Furthermore, tumors from treated mice exhibited statistically

significant reductions in size and weight, extensive tumor cell death,

reduction in the tumor-expressed VEGF-A biomarker, and destruction of

the blood vessels feeding the tumors.

Therapies for metastatic ccRCC including agents that target the

VEGF/VEGFR or mTor signaling pathways, which are validated cancer

targets, have become the standard-of-care and have improved patient

outcomes. However, since emergence of resistance to these agents is

common, novel therapies targeting alternative pathways are needed for

patients with resistant tumors. Arrowhead believes that HIF2α is an

attractive target for intervention because over 90% of ccRCC tumors

express a mutant form of the Von Hippel-Landau protein that is unable to

degrade HIF-2α, leading to its accumulation during tumor hypoxia and

promoting tumor growth.

An abstract of the data to be presented at ECC2015 is available on the

conference website at www.europeancancercongress.org/.

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, ARC-F12 for hereditary angioedema and

thromboembolic diseases, and ARC-HIF2 for renal cell carcinoma.

For more information please visit http://www.arrowheadresearch.com,

or follow us on Twitter @ArrowRes.

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please visit http://ir.arrowheadresearch.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. Arrowhead Research Corporation’s

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 Research

Corporation.

Source: Arrowhead Research Corporation

Arrowhead Research Corporation
Vince Anzalone, CFA
626-304-3400
ir@arrowres.com
or
Investor

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

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

Source: Arrowhead Research Corporation

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