Arrowhead Pharmaceuticals Presents Preclinical Data on Expanding Cardiometabolic Pipeline

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Arrowhead Pharmaceuticals Presents...

PASADENA, Calif.–(BUSINESS WIRE)–May 11, 2018–
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced the
presentation of new preclinical data on its expanding pipeline of RNA
interference (RNAi) therapeutics for cardiometabolic diseases, including
ARO-ANG3, which targets angiopoietin-like protein 3 (ANGPTL3), and
ARO-APOC3, which targets apolipoprotein C-III (ApoC3). The invited
lecture, titled “The promise of RNA interference as a therapeutic
approach for treatment of cardiovascular diseases,” was presented at the Vascular
Discovery: From Genes to Medicine – Scientific Sessions 2018, a
symposium organized by the American Heart Association. Arrowhead intends
to file clinical trial applications (CTA) for ARO-ANG3 and ARO-APOC3
before the end of 2018.

Bruce Given, M.D., chief operating officer and head of R&D at Arrowhead,
said, “We have a growing body of compelling preclinical data in a
diverse set of models for ARO-APOC3 and ARO-ANG3, the newest candidates
in Arrowhead’s cardiometabolic pipeline that leverage our Targeted RNAi
Molecule (TRiMTM) platform. We intend to present additional
results from ongoing preclinical studies of ARO-APOC3 and ARO-ANG3 at
scientific conferences later this year. We also look forward to filing
CTAs late this year to advance these promising product candidates into
the clinic.”

In the presentation, Dr. Given discussed the recent progress achieved
using RNA interference as a therapeutic modality for the development of
new drugs to treat cardiovascular diseases. The new preclinical data
presented on ARO-APOC3 and ARO-ANG3 included the following::

  • A single 2 mg/kg dose of ARO-APOC3 in ApoC3 transgenic mice led to the
    following observations:

    • Serum ApoC3 levels were reduced by approximately 90%
    • Maximum knockdown was sustained for over three weeks
    • The lipid profiles improved, with large reductions in serum
      triglycerides and cholesterol, and large increases in HDL
  • In dose ranging studies of ARO-ANG3 in wild type mice, reductions in
    serum ANGPTL3 and liver mRNA were similar, indicating that the
    observed knockdown of serum ANGPTL3 is a direct result of the expected
    RNAi effect.
  • The following mouse disease models were also interrogated:

    • LDL receptor knock-out (LDLr-/-) mice
    • Leptin receptor defective db/db mice
    • Diet-induced obese (DIO) mice
  • A single 3 mg/kg dose in LDLr-/- mice and in db/db mice led
    to:

    • A maximum reduction of ANGPTL3 of 98%
    • Substantial decreases in serum triglycerides and LDL
  • A single 3 mg/kg dose in DIO mice led to:

    • A maximum reduction of ANGPTL3 of 99.7%
    • Approximately 50-60% reductions in serum triglycerides and LDL

A copy of presentation materials 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.
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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 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.

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,
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