Arrowhead Pharmaceuticals to Present at Upcoming Conferences

PASADENA, Calif.–(BUSINESS WIRE)– Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) will make presentations
at the following upcoming events:

The 25^th Annual Credit Suisse Healthcare
Conference – Scottsdale, AZ, November 6-8, 2016

November 7, 11:30 a.m. MST – Christopher Anzalone, Ph.D.,
Arrowhead president and CEO will deliver a corporate presentation

The Liver Meeting^® 2016, the Annual Meeting
of the American Association for the Study of Liver Disease (AASLD) –
Boston, November 11-15, 2016

November 13, 5:30 p.m. EST – Christine Wooddell, Ph.D., Arrowhead
director of liver targeting will deliver an oral presentation titled, “RNA
interference therapeutic ARC-AAT prevents production of Z-alpha1
antitrypsin polymers and reverses liver disease phenotype in PiZ mouse
model”

November 13, 8:00 a.m. EST – Alice Turner, MBChB (Hons), MRCP,
PGCE (MedEd), PhD, University of Birmingham, Edgbaston Birmingham,
United Kingdom, and coauthors, will deliver a late-breaking poster
presentation titled, “RNA interference (RNAi) with ARC-AAT provides
deep and prolonged knockdown of alpha-1 antitrypsin levels in healthy
volunteers”

American Heart Association’s Scientific Sessions 2016 – New
Orleans, November 12-16, 2016

November 14, 10:40 a.m. CST – Stacey Melquist, Ph.D., Arrowhead
senior scientist will deliver an oral presentation titled, “Targeting
factor XII (F12) with a novel RNAi delivery platform as a prophylactic
treatment for thromboembolism”

November 14, 2:00 p.m. CST – Stacey Melquist, Ph.D., Arrowhead
senior scientist will deliver an oral presentation titled, “Targeting
apolipoprotein(a) with a novel RNAi delivery platform as a prophylactic
treatment to reduce risk of cardiovascular events in individuals with
elevated lipoprotein (a)”

The 28^th Annual Piper Jaffray Healthcare
Conference – New York, November 29-30, 2016

November 30, 8:30 a.m. EST – Christopher Anzalone, Ph.D.,
Arrowhead president and CEO will deliver a corporate overview in a
fireside chat with Ted Tenthoff, senior research analyst, Piper Jaffray

Copies of presentation materials and webcasts can be accessed on the Events
and Presentations page under the Investors section of the Arrowhead
website after each presentation is delivered.

About ARC-AAT

Arrowhead’s ARC-AAT is being investigated for the treatment of liver
disease associated with alpha-1 antitrypsin deficiency (AATD), a rare
genetic disease that severely damages the liver and lungs of affected
individuals. The mean estimated prevalence of AATD in the U.S. is 1 per
3000-5000, or approximately 100,000 patients. AATD is also an important
cause of pediatric liver disease with an estimated prevalence in
children of approximately 20,000 patients, and 50-80% likely to manifest
liver disease during childhood. It is a rare disease that is frequently
misdiagnosed or undiagnosed. ARC-AAT employs a novel unlocked nucleobase
analog (UNA) containing RNAi trigger molecule designed for systemic
delivery using the Dynamic Polyconjugate^™ delivery system.
ARC-AAT is highly effective at knocking down the alpha-1 antitrypsin
(AAT) gene transcript and reducing the hepatic production of the mutant
AAT (Z-AAT) protein in animal studies. Reduction of liver production of
the inflammatory Z-AAT protein, which is believed to be the cause of
progressive liver disease in AATD patients, is important as it is
expected to halt the progression of liver disease. ARC-AAT was granted
orphan drug designation in both the United States and in Europe, the
latter being held on Arrowhead’s behalf by a local EU representative
Pharma Gateway AB. Arrowhead is conducting a Phase 1 clinical study of
ARC-AAT, with part A in healthy volunteers (now complete) and part B in
AATD patients, and a Phase 2 multiple dose study in AATD patients.

About ARC-F12

Arrowhead’s RNAi-based candidate ARC-F12 is in preclinical development
as a potential treatment for factor XII (F12) mediated diseases.
Arrowhead sees clear unmet need in hereditary angioedema (HAE) and
thromboembolic diseases. The biology of factor 12 as part of the
coagulation cascade and the kinin-kallikrein system suggest that its
reduction through RNAi may present opportunities in both disease areas.
The company is currently conducting studies in order to advance ARC-F12
into clinical trials.

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
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.

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