Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1/2 Study of ARC-521

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PASADENA, Calif.–(BUSINESS WIRE)–

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR), recently filed a

regulatory submission in New Zealand to begin a Phase 1/2 clinical trial

of ARC-521, its RNAi-based therapeutic candidate for the treatment of

chronic hepatitis B virus (HBV) infection. Pending approval, Arrowhead

intends to proceed with ARC521-1001, a first-in-human study to assess

single and multiple-doses of ARC-521 in healthy volunteers and HBV

patients.

Chris Anzalone, Ph.D., president and CEO of Arrowhead Pharmaceuticals,

said: “ARC-521 is our second pipeline product targeting chronic HBV and

was designed to silence gene products from both HBV cccDNA and

integrated HBV DNA. This is important because our clinical work with

ARC-520 in HBV patients and our long-term chimpanzee study suggest that

different patient groups can have different relative levels of cccDNA.

We think having both ARC-520, which has been very active in patients

with higher cccDNA, and ARC-521, which may be optimal for those with

lower cccDNA, should provide us with the potential to treat all patients

with HBV. We have an aggressive plan for the development of ARC-521 that

includes an accelerated first-in-man Phase 1/2 design intended to allow

rapid transition into multi-dose patient cohorts.”

The application for approval of a clinical trial was submitted to the

New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) for

review by the Standing Committee on Therapeutic Trials (SCOTT).

Arrowhead also intends to seek regulatory clearance to conduct

ARC521-1001 in additional countries.

About ARC-521

Arrowhead’s ARC-521 is being investigated for its potential to produce

functional cures in patients with chronic hepatitis B virus (HBV)

infection. ARC-521 intervenes upstream of the reverse transcription

process where current standard-of-care nucleotide and nucleoside analogs

act, and is designed to silence the production of all HBV gene products.

The small interfering RNAs (siRNAs) in ARC-521 engage the body’s normal

cellular RNAi machinery and direct specific cleavage of HBV RNA

transcripts, thereby reducing the levels of HBV proteins and the RNA

template used to produce viral DNA. Designed to complement ARC-520,

ARC-521 targets HBV mRNA transcripts from both cccDNA and integrated DNA

and is expected to be most suitable for those patients who tend to have

lower levels of viral cccDNA. Arrowhead is investigating ARC-521

specifically to determine if significantly reducing circulating and

non-circulating viral proteins and RNA will allow for re-constitution of

an effective host immune response and ultimately HBsAg seroclearance

resulting in functional cure. As many as 350-400 million people

worldwide are chronically infected with the hepatitis B virus, which can

lead to cirrhosis of the liver and is responsible for 80% of primary

liver cancers globally. Arrowhead is planning to conduct a Phase 1/2

single and multiple-dose study in healthy volunteers and HBV 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.

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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, 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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