Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1 Study of ARO-APOC3 for Treatment of Hypertriglyceridemia

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Arrowhead Pharmaceuticals Files...

PASADENA, Calif.–(BUSINESS WIRE)–Jan. 7, 2019–
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it
has filed an application for approval to begin a Phase 1 clinical trial
of ARO-APOC3, an RNAi-based investigational medicine targeting
Apolipoprotein C-III (apoC-III) being developed for the treatment of
hypertriglyceridemia.

Chris Anzalone, Ph.D., president and CEO of Arrowhead Pharmaceuticals,
said: “ARO-APOC3 will be our fifth product candidate leveraging the
Targeted RNAi Molecule, or TRiMTM, platform to enter clinical
studies. Addressing hypertriglyceridemia through reduction of
liver-produced apoC-III offers multiple potential development
opportunities, including both orphan indications, such as FCS, and large
market indications. In AROAPOC31001, our first-in-human study, we intend
to evaluate ARO-APOC3 in healthy volunteers and various patient
populations with elevated triglycerides, which will help inform our
strategy around the ideal development and regulatory paths to pursue.”

Pending approval, Arrowhead intends to proceed with AROAPOC31001 (NCT03783377),
a Phase 1 single and multiple dose-escalating study to evaluate the
safety, tolerability, pharmacokinetics, and pharmacodynamic effects of
ARO-APOC3 in adult healthy volunteers, hypertriglyceridemic patients,
and patients with familial chylomicronemia syndrome (FCS). The study is
designed to enroll up to 63 subjects.

The application for approval of the clinical trial is being submitted to
a local Ethics Committee and to the New Zealand Medicines and Medical
Devices Safety Authority for review by the Standing Committee on
Therapeutic Trials.

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 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

Investors and Media:
LifeSci Advisors, LLC
Brian Ritchie
212-915-2578
britchie@lifesciadvisors.com
www.lifesciadvisors.com