Arrowhead Begins Dosing in Phase 1/2 Study of ARO-HBV for Treatment of Chronic Hepatitis B

Home/Newsroom/

Arrowhead Begins Dosing in Phase...

PASADENA, Calif.–(BUSINESS WIRE)–Mar. 27, 2018–
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it
has dosed the first subjects in a Phase 1/2 clinical study of ARO-HBV,
which is being developed as a potentially curative therapy for patients
with chronic hepatitis B virus (HBV) infection. ARO-HBV is the second
clinical candidate developed using Arrowhead’s proprietary Targeted RNAi
Molecule (TRiMTM) technology.

Bruce Given, M.D., chief operating officer and head of R&D at Arrowhead,
said, “We are proud of how quickly ARO-HBV moved through preclinical
studies and are excited to be starting first-in-human clinical studies.
The AROHBV1001
Phase 1/2 clinical study has an innovative design that is intended
to assess, in parallel, safety and tolerability in healthy volunteers
and multiple-dose anti-viral activity in HBV patients. Our experience
with two prior compounds, ARC-520 and ARC-521, provides us with
confidence that ARO-HBV, which is designed to target all viral
transcripts, including those produced by both cccDNA and HBV DNA
integrated into host DNA, has the potential to be a backbone therapy in
combinations aimed at achieving a functional cure of chronic HBV
infection.”

AROHBV1001 (NCT03365947)
is a Phase 1/2 study evaluating the safety, tolerability, and
pharmacokinetic effects of single-ascending doses (SAD) of ARO-HBV in
healthy adult volunteers, and to evaluate the safety, tolerability, and
pharmacodynamic effects of multiple-ascending doses (MAD) of ARO-HBV in
patients with chronic HBV.

The SAD portion is designed to include up to 5 cohorts of 6 subjects per
cohort. Each SAD subject will receive a single-dose administration of
either placebo or ARO-HBV at up to 5 dose levels (35, 100, 200, 300, 400
mg). The MAD portion is designed to include up to 8 cohorts of 4 HBV
patients per cohort. Each MAD patient will receive 3 doses of ARO-HBV at
up to 4 dose levels (100, 200, 300, 400 mg).

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 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
212-915-2578
britchie@lifesciadvisors.com
www.lifesciadvisors.com