Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1 Study of ARO-ANG3

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Arrowhead Pharmaceuticals Files...

— Arrowhead to host R&D Day October 16, 2018 to discuss ARO-ANG3 and its
emerging pipeline of RNAi therapeutics

PASADENA, Calif.–(BUSINESS WIRE)–Oct. 15, 2018–
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it
has filed an application for approval to begin a Phase 1 clinical trial
of ARO-ANG3, an RNAi-based investigational medicine targeting
angiopoietin like protein 3 (ANGPTL3) being developed for the treatment
of dyslipidemias and metabolic diseases. ARO-ANG3 will be the fourth
therapeutic candidate to enter clinical studies that leverages
Arrowhead’s Targeted RNAi Molecule (TRiM™) platform.

Chris Anzalone, Ph.D., president and CEO of Arrowhead Pharmaceuticals,
said: “The ARO-ANG3 program has moved rapidly through preclinical
development and we are thrilled to now submit the regulatory filing
ahead of schedule. After positive initial signs of tolerability and
activity with ARO-AAT and ARO-HBV, our first two clinical programs
leveraging the TRiM™ platform, we are moving into the ARO-ANG3 clinical
program with confidence.”

Pending approval, Arrowhead intends to proceed with AROANG1001, a Phase
1 single and multiple dose study to evaluate the safety, tolerability,
pharmacokinetics, and pharmacodynamic effect of ARO-ANG3 in adult
healthy volunteers and dyslipidemic patients. The study is designed to
enroll up to 70 subjects.

An application was submitted to an ethics committee in compliance with
the Clinical Trial Notification process of the Australian Department of
Health and Ageing, Therapeutic Goods Administration.

As a reminder, Arrowhead will host a Research & Development (R&D) Day to
discuss ARO-ANG3 and its emerging pipeline of RNAi therapeutics that
leverage the Company’s proprietary Targeted RNAi Molecule (TRIM™)
platform on October 16, 2018 in New York City.

The R&D Day will feature presentations by Ira Goldberg, M.D. (NYU
Langone Medical Center), who will discuss the current treatment
landscape and unmet medical need for patients with dyslipidemia and
hypertriglyceridemia.

Arrowhead’s management team will provide an overview of the company’s
pipeline of RNAi-based therapeutics. Discussion topics will include:

  • ARO-APOC3 for patients with hypertriglyceridemia. A CTA filing is
    planned by the end of 2018
  • ARO-ANG3 for patients with dyslipidemia
  • ARO-ENaC for patients with cystic fibrosis. A CTA filing is planned in
    2019
  • ARO-HIF2 for patients with renal cell carcinoma. A CTA filing is
    planned in 2019

This event is intended for institutional investors, sell-side analysts,
investment bankers, and business development professionals only. Please
RSVP in advance if you plan to attend, as space is limited. To reserve a
seat, please click
here to register.

A live and archived webcast of the event, with slides, 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.
To be added to the Company’s email list and receive news directly,
please visit http://ir.arrowheadpharma.com/email-alerts.

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 likelihood and
timing of the receipt of future milestone and licensing fees, the future
success of our scientific studies, our ability to successfully develop
and commercialize 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
212-915-2578
britchie@lifesciadvisors.com
www.lifesciadvisors.com