Arrowhead’s ARC-AAT Granted EMA Orphan Drug Designation

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Arrowhead’s ARC-AAT Granted...

PASADENA, Calif.–(BUSINESS WIRE)–

Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical

company developing targeted RNAi therapeutics, today announced that the

European Medicines Agency (EMA) granted Orphan Drug Designation to

ARC-AAT for treatment of congenital alpha-1 antitrypsin deficiency.

ARC-AAT was also previously granted Orphan Drug Designation by the

United States Food and Drug Administration (FDA) in 2015.

Orphan Drug Designation from the EMA allows Arrowhead to benefit from a

number of incentives, such as reduced regulatory fees, protocol

assistance, and market exclusivity, to develop a medicine for the

treatment of a rare disease affecting not more than five in 10,000

people in the European Union.

“ARC-AAT has the potential to provide patients and physicians with a

much needed treatment option for liver disease associated with alpha-1

antitrypsin deficiency, a rare genetic disorder. We are pleased that the

EMA has granted Orphan Drug Designation and we view this as an important

regulatory milestone for the program,” said Bruce Given, M.D., Arrowhead

chief operating officer.

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 appears to be

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 models. 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 and potentially

allow fibrotic tissue repair. 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 and part B in AATD patients.

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 and ARC-521

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,

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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 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, the timing of any market authorization, the timing of any market

authorization for competitive products, 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
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Investor

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Source: Arrowhead Research Corporation

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