Arrowhead Pharmaceuticals’ Preclinical Candidate ARC-LPA Achieves 98% Knockdown and Long Duration of Effect after Subcutaneous Administration

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PASADENA, Calif.–(BUSINESS WIRE)–

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today presented a poster

on ARC-LPA, its preclinical development program targeting lipoprotein

(a), or Lp(a), for the treatment of cardiovascular disease at the

Arteriosclerosis, Thrombosis and Vascular Biology | Peripheral Vascular

Disease (ATVB|PVD) 2016 Scientific Sessions in Nashville. ARC-LPA is the

first RNAi therapeutic program to use Arrowhead’s new delivery vehicles

designed for subcutaneous (SQ) administration.

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

said: “These data are exciting for several reasons. First, they

represent continued progress in our SQ delivery platform, creating

opportunities to address various diseases that require chronic treatment

and where SQ administration may be preferable for patients and

physicians. We are enabling very deep target gene knockdown with long

duration of effect that may enable monthly, bi-monthly, or even less

frequent administration. Second, reducing Lp(a), as we have seen now in

multiple animal models, could represent an important leap forward in the

treatment of cardiovascular disease. High levels of Lp(a) are associated

with increased risk of cardiovascular disease independent of cholesterol

and LDL, and there is currently no good way to deeply reduce circulating

levels of Lp(a).”

The poster titled, “Lipoprotein(a) targeting with RNAi delivery

platforms in transgenic mice and cynomolgus monkeys” (presentation 428),

describes data from in vitro screening of RNAi triggers and multiple in

vivo models, including transiently transgenic mice, transgenic mice

(Tg), and non-human primates (NHPs). Key findings from these studies

include the following:

  • Screening of RNAi triggers in Tg mice identified those that exhibited

    substantial and sustained knockdown of serum apo(a) and Lp(a) levels

  • RNAi trigger sequences were active in both intravenous and SQ platforms

  • Structure activity relationship (SAR) studies looking at chemical

    modifications to the RNAi trigger identified a lead that demonstrated

    greater than 98% maximum knockdown after a single 3 mg/kg SQ dose in

    transgenic mice

  • Duration of effect gains were also made with greater than 85%

    knockdown still seen at 6 weeks post dose

  • In NHPs, 85-90% reduction of serum Lp(a) levels was observed after

    three weekly 3 mg/kg SQ doses

  • Duration of effect in NHPs was long, with Lp(a) levels still reduced

    by 75% 6 weeks after the final dose

A copy of the poster presentation will be made available on the Events

and Presentations page under the Investors section of the Arrowhead

website.

About ARC-LPA

Arrowhead’s RNAi-based candidate ARC-LPA is in preclinical development

as a potential treatment for cardiovascular diseases. ARC-LPA is

designed to reduce production of apolipoprotein(a), a key component of

lipoprotein(a), or Lp(a). Lp(a) levels in humans are genetically defined

and higher levels correlate with increased risk of cardiovascular

diseases, independent of cholesterol and LDL levels. ARC-LPA is

Arrowhead’s first drug candidate to use a subcutaneously administered

delivery construct.

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

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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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