Arrowhead Presents New ARO-HBV Clinical Data Demonstrating HBsAg Reductions at World Gastroenterologists Summit

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Arrowhead Presents New ARO-HBV...

  • Data presented from the lowest two dose cohorts (100mg and 200mg
    ARO-HBV)
  • Up to 4.0 log10 reduction in HBsAg observed following three doses of
    ARO-HBV
  • ARO-HBV was generally well-tolerated in HBV patients

PASADENA, Calif.–(BUSINESS WIRE)–Sep. 6, 2018–
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) will present initial
clinical data for ARO-HBV, the company’s third generation subcutaneously
administered RNA interference (RNAi) therapeutic being developed as a
potentially curative therapy for patients with chronic hepatitis B virus
(HBV) infection, at the 18th World Gastroenterologists Summit
in Auckland, New Zealand. Data will be presented from the eight patients
in the lowest two dose cohorts: 100mg and 200mg. The data demonstrate
that three monthly doses of ARO-HBV led to a maximum reduction in
circulating HBV surface antigen (HBsAg) of 4.0 log10, with mean
reductions of approximately 2.0 log10 on day 85 in the 100 mg cohort and
1.4 log10 on day 71 in the 200mg cohort (currently the last complete
data point available). All eight patients achieved greater than 1.0
log10 reductions in circulating HBsAg.

Safety data will be presented across all ten patient cohorts (n=40).
ARO-HBV was generally well-tolerated with generally mild and
self-limiting injection site adverse events being the most common
reported event in chronic HBV patients, occurring in around 10% of
injections. The other most commonly reported events included symptoms
consistent with upper respiratory tract infection and headache.

These results represent the first clinical data presented on ARO-HBV,
which leverages Arrowhead’s proprietary Targeted RNAi Molecules (TRiMTM)
platform. The company intends to submit a late-breaking abstract with
additional clinical data to the Liver Meeting®, the Annual
Meeting of the American Association for the Study of Liver Disease
(AASLD), being held in November 2018.

Bruce Given, M.D., Arrowhead’s chief operating officer and head of R&D,
said, “These initial results from the first two multiple-ascending dose
cohorts of the AROHBV1001 clinical study are encouraging and indicate
that ARO-HBV is highly active. In addition, the drug appears to be
generally well-tolerated, which is consistent with our experience to
date with ARO-AAT, our TRiMTM enabled candidate for the
treatment of Alpha-1 liver disease. We intend to submit late-breaking
abstracts to the AASLD Liver Meeting for both ARO-HBV and ARO-AAT, and,
if accepted, we look forward to presenting more complete data-sets,
including additional dose levels and longer follow-up.”

Key new data to be presented at the 18th World
Gastroenterologists Summit from the AROHBV1001 Phase 1/2 clinical study
in patients with chronic HBV who received three monthly doses of ARO-HBV
include the following:

  • Mean reduction of HBsAg was 2.0 log10 (99%) on day 85 in cohort 2b
    (100 mg) and 1.4 log10 (96%) on day 71 in cohort 3b (200 mg)

    • These may not represent nadir
  • Maximum reduction of HBsAg was 4.0 log10 (99.99%)
  • Minimum HBsAg reduction in all patients from cohorts 2b and 3b was 1.2
    log10 (93%)
  • Activity was demonstrated in all patient types (HBeAg pos/neg, NUC
    naïve/treated)
  • ARO-HBV appeared to be generally well-tolerated

The keynote presentation, titled “Hepatitis B in focus: new biology, new
targets and real hope for finite therapy,” will be delivered by Dr.
Given on September 7 at 09:45 a.m. NZST. A copy of the presentation (see
slides 21-23) can be accessed on the Events
and Presentations page under the Investors section of the Arrowhead
website.

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 evaluating the safety, tolerability, and
pharmacodynamic effects of multiple-ascending doses (MAD) of ARO-HBV in
patients with chronic HBV. Dosing in the SAD portion of the study is
complete, and included five cohorts at dose levels of 35, 100, 200, 300,
and 400 mg. Dosing in the MAD portion of the study is ongoing, and
includes ten cohorts receiving three doses of ARO-HBV either weekly,
bi-weekly, or monthly, and includes dose levels of 100, 200, 300, and
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.
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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:
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Brian Ritchie
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