Arrowhead Presents Overview of Its Broad RNAi Delivery Platform and Introduces New Subcutaneously Administered Format
October 14 2015 - 9:30AM
Business Wire
Arrowhead Research Corporation (NASDAQ: ARWR),
a biopharmaceutical company developing targeted RNAi therapeutics,
today presented an overview of its broad RNAi delivery platform at
the Annual Meeting of the Oligonucleotide Therapeutics Society in
Leiden, the Netherlands. Included in this presentation was data
from a new program, ARC-LPA, which is designed to reduce production
of apolipoprotein A [apo(a)], a key component of lipoprotein(a)
[Lp(a)]. Lp(a) has been genetically linked with increased risk of
cardiovascular diseases, independent of cholesterol and LDL levels.
Using a new subcutaneous delivery construct that Arrowhead has
developed, an initial lead compound has achieved up to 90%
knockdown of apo(a) in mice.
“Our ability to generate RNAi drugs for a wide
variety of indications has expanded dramatically with our advances
in delivery,” said Chris Anzalone, Ph.D., Arrowhead’s president and
chief executive officer. “We currently have two drugs in the
clinic, ARC-520 and ARC-AAT, that validate our capabilities in IV
liver delivery and provide additional confidence in follow-on
candidates ARC-F12 and ARC-521. We are now able to deliver to
tumors, as exemplified by ARC-HIF2, and report data today on
subcutaneous administration for liver delivery with ARC-LPA. These
capabilities provide broad opportunities to fight diverse diseases
and enable us to drive an aggressive pipeline.”
In a presentation titled, “Development of
RNAi-Based Therapeutics using Dynamic Polyconjugates™ (DPC™)
Technology,” Bruce Given, M.D., Arrowhead’s chief operating
officer, discussed three examples of Arrowhead’s broad RNAi
delivery platform: DPC™ for liver delivery with select data from
the ARC-520 clinical program; DPC™ for extra-hepatic delivery with
select preclinical data from the ARC-HIF2 program; and, a new
proprietary subcutaneous delivery vehicle with initial preclinical
data from the ARC-LPA program.
Arrowhead uses DPC™, or Dynamic
Polyconjugates™, for liver delivery in both of its clinical stage
drugs, ARC-520 for chronic hepatitis B infection and ARC-AAT for
alpha-1 antitrypsin deficiency, as well as additional preclinical
stage programs, including ARC-F12 for hereditary angioedema and
ARC-521 for chronic hepatitis B infection. Arrowhead’s delivery
platform can produce deep and sustained knockdown of mRNA and
proteins, as evidenced by data from the ARC-520 program showing
dramatic reductions in hepatitis B e-antigen, core-related antigen,
and s-antigen in humans. ARC-520 achieved a maximum reduction of
s-antigen of 99% (1.9 log), which is the highest reported
single-dose knockdown in humans with any RNAi therapeutic.
DPC™ for extra-hepatic delivery is being
deployed in Arrowhead’s ARC-HIF2 candidate for the treatment of
clear cell renal cell carcinoma (ccRCC). As Arrowhead previously
reported, in an orthotopic ccRCC tumor model in mice ARC-HIF2
reduced the expression of HIF2α, a well-validated oncogene in this
disease, by more than 80%, leading to statistically significant
reductions in tumor size and weight, extensive tumor cell death,
reduction in the tumor-expressed VEGF-A biomarker, and destruction
of the blood vessels feeding the tumors. DPC™ for extra-hepatic
delivery employs a masked polymer designed to induce endosomal
escape, with RNAi trigger attached directly to the delivery
vehicle. This molecule is actively guided to tumor cells with a
proprietary targeting agent that binds to αVβ3 integrin, which is
highly expressed on the surface of ccRCC cells as well as various
other tumor types.
Dr. Given also introduced Arrowhead’s new
hepatic delivery format being developed for subcutaneous
administration and a new target being explored for cardiovascular
disease. The new delivery construct discussed includes an RNAi
trigger against apo(a). Apo(a) is a critical component of Lp(a) and
effective knockdown results in marked Lp(a) reductions. Single dose
knockdown of around 90% in apo(a) has been achieved in mice.
Initial data through day 15 suggest a good duration of effect
(study ongoing). Similar to the intravenously administered vehicle
used for hepatic delivery, this construct employs N-acetyl
galactosamine for hepatic targeting. However, it does not employ
the same peptide-based active endosomal escape. An active lead
compound has been identified and additional lead optimization is
ongoing. This program has not been officially designated as
entering pre-IND development.
A copy of the presentation can be accessed by
visiting the Events section of the company’s website at
http://ir.arrowheadresearch.com/events.cfm after the presentation
concludes.
About Arrowhead Research Corporation
Arrowhead Research Corporation is a
biopharmaceutical company developing targeted RNAi therapeutics.
The company is leveraging its proprietary Dynamic Polyconjugate™
delivery platform to develop targeted drugs based on the RNA
interference mechanism that efficiently silences disease-causing
genes. Arrowhead’s pipeline includes ARC-520 and ARC-521 for
chronic hepatitis B virus, ARC-AAT for liver disease associated
with Alpha-1 antitrypsin deficiency, ARC-F12 for hereditary
angioedema and thromboembolic diseases, and ARC-HIF2 for renal cell
carcinoma.
For more information please visit
http://www.arrowheadresearch.com, or follow us on Twitter
@ArrowRes. To be added to the Company's email list and receive news
directly, please visit
http://ir.arrowheadresearch.com/alerts.cfm.
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. Arrowhead Research Corporation's
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 Research Corporation.
Source: Arrowhead Research
Corporation
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version on businesswire.com: http://www.businesswire.com/news/home/20151014005513/en/
Arrowhead Research CorporationVince Anzalone,
CFA626-304-3400ir@arrowres.comorInvestor Relations:The Trout
GroupChad Rubin, 646-378-2947ir@arrowres.comorMedia:Russo
PartnersMatt Middleman,
M.D.212-845-4272matt.middleman@russopartnersllc.com
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