Alnylam Pharmaceuticals, Inc.
(Nasdaq: ALNY), a leading RNAi therapeutics company, and Arrowhead
Research Corporation, (Nasdaq: ARWR) a nanomedicine company with
development programs in RNAi and obesity, announced today that they
have entered into a collaboration and joint licensing
agreement.
Alnylam has granted Arrowhead a license under its intellectual
property that enables the discovery, development, and
commercialization of an RNAi therapeutic targeting the hepatitis B
virus (HBV). Alnylam is eligible to receive from Arrowhead
milestone payments and royalties on sales of product resulting from
the license. In addition, Alnylam has received a license from
Arrowhead to utilize their Dynamic Polyconjugate (DPC) delivery
technology for an RNAi therapeutic product. Alnylam expects to
deploy this technology for an undisclosed target in its “Alnylam
5x15” pipeline which is focused on genetically defined targets and
diseases. Arrowhead is eligible to receive from Alnylam milestone
payments and royalties on sales of product resulting from the
license. No additional financial details were disclosed.
“We view Arrowhead’s DPC technology as a promising emerging
delivery approach, with the potential to complement our existing
delivery platform which currently includes lipid nanoparticles and
our siRNA conjugate platform,” said Laurence Reid, Ph.D., Senior
Vice President and Chief Business Officer of Alnylam. “In addition,
by granting Arrowhead a license for their HBV program, we are
enabling their efforts with access to Alnylam intellectual property
which we believe is critical for the development and
commercialization of RNAi therapeutics. We look forward to
continuing to work with Arrowhead, who is already a partner and
licensee of Alnylam.”
“This license from Alnylam is an important step for us as we
expand our pipeline to include our first DPC-enabled candidate
targeting hepatitis B,” said Christopher Anzalone, Ph.D., President
and CEO of Arrowhead. “With over 350 million carriers world-wide,
HBV represents a large underserved medical need, and one that RNAi
and DPCs are well-suited to address. We are also very pleased to
grant Alnylam the first commercial license to our DPC technology
for one of their genetically defined disease targets. We believe
DPCs represent one of the most promising delivery approaches for
the systemic delivery of RNAi therapeutics, and we look forward to
a close collaboration to help Alnylam bring a DPC-enabled candidate
to the clinic.”
About RNA Interference (RNAi)
RNAi (RNA interference) is a revolution in biology, representing
a breakthrough in understanding how genes are turned on and off in
cells, and a completely new approach to drug discovery and
development. Its discovery has been heralded as “a major scientific
breakthrough that happens once every decade or so,” and represents
one of the most promising and rapidly advancing frontiers in
biology and drug discovery today which was awarded the 2006 Nobel
Prize for Physiology or Medicine. RNAi is a natural process of gene
silencing that occurs in organisms ranging from plants to mammals.
By harnessing the natural biological process of RNAi occurring in
our cells, the creation of a major new class of medicines, known as
RNAi therapeutics, is on the horizon. Small interfering RNAs
(siRNAs), the molecules that mediate RNAi and comprise Alnylam’s
RNAi therapeutic platform, target the cause of diseases by potently
silencing specific mRNAs, thereby preventing disease-causing
proteins from being made. RNAi therapeutics have the potential to
treat disease and help patients in a fundamentally new way.
About DPC Technology
Dynamic Polyconjugate (DPC) technology is a systemic siRNA
delivery platform that enables polymer-based formulation chemistry
to efficiently target gene silencing complexes to specific cells.
As the DPCs attach to and enter the target cells, they respond to
the environmental cues provided by the cell to disassemble and
release the active siRNA molecule. This unique chemistry mimics the
natural viral targeting and disassembly process. Pre-clinical
studies show that DPCs are highly efficacious for delivery to the
liver.
About “Alnylam 5x15™”
The “Alnylam 5x15” strategy, launched in January 2011,
establishes a path for development and commercialization of novel
RNAi therapeutics to address genetically defined diseases with high
unmet medical need. Products arising from this initiative share
several key characteristics including: a genetically defined target
and disease; the potential to have a major impact in a high unmet
need population; the ability to leverage the existing Alnylam RNAi
delivery platform; the opportunity to monitor an early biomarker in
Phase I clinical trials for human proof of concept; and the
existence of clinically relevant endpoints for the filing of a new
drug application (NDA) with a focused patient database and possible
accelerated paths for commercialization. This strategy leverages
Alnylam’s clinical progress on siRNA delivery, including definitive
human proof-of-concept data for systemic delivery. By the end of
2015, the company expects to have five such RNAi therapeutic
programs in advanced clinical development. These include ALN-TTR
for the treatment of transthyretin-mediated amyloidosis (ATTR),
ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN
for the treatment of refractory anemia, ALN-APC for the treatment
of hemophilia, and one additional program from the company’s
ongoing discovery efforts that will be designated at or around the
end of 2011. Alnylam intends to focus on developing and
commercializing certain products arising under the “Alnylam 5x15”
strategy itself in the United States and potentially certain other
countries; the company will seek development and commercial
partners for other core products both in the United States and in
other global territories.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel
therapeutics based on RNA interference, or RNAi. The company is
leading the translation of RNAi as a new class of innovative
medicines with a core focus on RNAi therapeutics for the treatment
of genetically defined diseases, including ALN-TTR for the
treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for
the treatment of severe hypercholesterolemia, ALN-HPN for the
treatment of refractory anemia, and ALN-APC for the treatment of
hemophilia. As part of its “Alnylam 5x15TM” strategy, the company
expects to have five RNAi therapeutic products for genetically
defined diseases in advanced stages of clinical development by the
end of 2015. Alnylam has additional partner-based programs in
clinical or development stages, including ALN-RSV01 for the
treatment of respiratory syncytial virus (RSV) infection, ALN-VSP
for the treatment of liver cancers, and ALN-HTT for the treatment
of Huntington’s disease. The company’s leadership position on RNAi
therapeutics and intellectual property have enabled it to form
major alliances with leading companies including Merck, Medtronic,
Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and
Cubist. In addition, Alnylam and Isis co-founded Regulus
Therapeutics Inc., a company focused on discovery, development, and
commercialization of microRNA therapeutics; Regulus has formed
partnerships with GlaxoSmithKline and Sanofi. Alnylam has also
formed Alnylam Biotherapeutics, a division of the company focused
on the development of RNAi technologies for application in
biologics manufacturing, including recombinant proteins and
monoclonal antibodies. Alnylam’s VaxiRNA™ platform applies RNAi
technology to improve the manufacturing processes for vaccines;
GlaxoSmithKline is a collaborator in this effort. Alnylam
scientists and collaborators have published their research on RNAi
therapeutics in over 100 peer-reviewed papers, including many in
the world’s top scientific journals such as Nature, Nature
Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam
maintains headquarters in Cambridge, Massachusetts. For more
information, please visit www.alnylam.com.
About Arrowhead Research Corporation
Arrowhead Research Corporation is a clinical stage nanomedicine
company developing innovative therapies at the interface of biology
and nanoengineering. Arrowhead's world-class capabilities and
intellectual property covering nucleic acid delivery, siRNA
chemistry, and tissue targeting allow it to design and develop
therapeutic agents for a wide range of diseases. The company's lead
products include CALAA-01, an oncology drug candidate based on the
gene silencing RNA interference (RNAi) mechanism, and Adipotide™,
an anti-obesity peptide that targets and kills the blood vessels
that feed white adipose tissue. Arrowhead is leveraging its
proprietary Dynamic Polyconjugate™ (DPC), Liposomal Nanoparticle
(LNP), and RONDEL™ delivery platforms to support its own pipeline
of preclinical and clinical candidates and to secure external
partnerships and collaborations with biotech and pharmaceutical
companies. For more information, please visit
www.arrowheadresearch.com.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future
expectations, plans and prospects, including without limitation,
statements regarding Alnylam’s views with respect to the potential
for RNAi therapeutics and DPC technology, and Alnylam’s
expectations regarding its “Alnylam 5x15” product strategy,
constitute forward-looking statements for the purposes of the safe
harbor provisions under The Private Securities Litigation Reform
Act of 1995. Actual results may differ materially from those
indicated by these forward-looking statements as a result of
various important factors, including, without limitation, Alnylam’s
ability to discover and develop novel drug candidates, successfully
demonstrate the efficacy and safety of its drug candidates,
including those utilizing DPC technology, the pre-clinical and
clinical results for its product candidates, which may not support
further development of product candidates, actions of regulatory
agencies, which may affect the initiation, timing and progress of
clinical trials, obtaining, maintaining and protecting intellectual
property, obtaining regulatory approval for products, competition
from others using technology similar to Alnylam’s and others
developing products for similar uses, as well as those risks more
fully discussed in the “Risk Factors” section of its most recent
quarterly report on Form 10-Q on file with the Securities and
Exchange Commission. In addition, any forward-looking statements
represent Alnylam’s views only as of today and should not be relied
upon as representing its views as of any subsequent date. Alnylam
does not assume any obligation to update any forward-looking
statements.
Arrowhead Forward-Looking Statements
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 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.
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