Regulus Therapeutics LLC, a joint venture between Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY) and Isis Pharmaceuticals, Inc.
(Nasdaq: ISIS) formed to discover, develop, and commercialize microRNA
(miRNA) therapeutics, announced today that it has obtained exclusive
rights from Stanford University to worldwide patent applications
covering methods and compositions for antagonizing miR-181a to
regulate immune responses. Changes in miR-181a levels have been shown
to modify the response of immune cells such as T lymphocytes to
specific stimuli and its antagonism could lead to a new way to treat
inflammatory diseases.
"We are excited about obtaining exclusive rights to this
intellectual property, as it is another step in Regulus' overall
strategy to build a broad and leading platform of technology and
intellectual property for the development of miRNA therapeutics, and
adds to our already dominant patent position," said Kleanthis G.
Xanthopoulos, Ph.D., President and Chief Executive Officer of Regulus.
"At Regulus, we are aggressively exploring a variety of therapeutic
areas including viral, metabolic, and inflammatory diseases. Access to
this intellectual property allows us to apply our expertise in the
biology and potential therapeutic uses of miRNAs to develop novel
treatments for inflammatory diseases."
Data published in 2007 by scientists at Stanford University and
Alnylam in the journal Cell (Li et al. (2007) Cell 129, 147-161)
demonstrated that modulation of miR-181a levels in an immune cell
modified the sensitivity of the cell to specific stimuli. Researchers
found that by increasing expression of miR-181a, an increase in the
immune cell's response to an inflammatory stimulus occurred.
Conversely, decreasing levels of miR-181a in the immune cell led to a
diminution in the cell's response to an inflammatory stimulus, thereby
de-sensitizing the cell to the stimulus. Using a selective miRNA
antagonist to inhibit miR-181a function resulted in efficient
reduction in the immune cell's response to a stimulus. These data
suggest that controlling miR-181a levels with selective antagonists
may lead to a novel approach to treating inflammatory diseases.
About microRNAs
microRNAs (miRNAs) are a recently discovered class of genetically
encoded small RNAs, approximately 20 nucleotides in length, and are
believed to regulate the expression of a large number of human genes.
miRNA therapeutics represent a new approach for the treatment of a
wide range of human diseases. The inappropriate absence or presence of
specific miRNAs in various cells has been shown to be associated with
specific human diseases including cancer, viral infection, and
metabolic disorders. Targeting miRNAs with novel therapeutic agents
could result in novel and broadly acting treatments for human
diseases.
About Regulus
Regulus Therapeutics LLC is a biopharmaceutical company formed to
discover, develop and commercialize miRNA therapeutics. Regulus
aspires to successfully translate one of the most important new
properties in biology into a novel new approach for innovative
medicine and to build the leading microRNA company. Regulus was
created as a joint venture between Alnylam Pharmaceuticals, a leader
in RNAi therapeutics, and Isis Pharmaceuticals, a leader in antisense
technologies and therapeutics. Isis and Alnylam scientists and
collaborators were the first to discover miRNA antagonist strategies
that work in vivo in animal studies (Krutzfeldt et al. Nature 438,
685-689 (2005); Esau et al. Cell Metab., 3, 87-98 (2006)). Isis and
Alnylam have also created and consolidated key intellectual property
(IP) for the development and commercialization of miRNA therapeutics.
This IP estate includes over 900 patents and patent applications,
including 600 issued patents, owned by Isis and Alnylam and pertaining
to chemical modification of oligonucleotides for therapeutic
applications. In addition, Regulus has collaborations with more than
60 academic researchers to build on the company's understanding of
microRNAs and recently formed a major alliance with GlaxoSmithKline to
explore new therapeutic areas for microRNA therapeutics. Regulus,
founded in September 2007, maintains facilities in Carlsbad,
California. For more information, visit www.regulusrx.com.
About Isis Pharmaceuticals, Inc.
Isis is exploiting its expertise in RNA to discover and develop
novel drugs for its product pipeline and for its partners. The Company
has successfully commercialized the world's first antisense drug and
has 19 drugs in development. Isis' drug development programs are
focused on treating cardiovascular and metabolic diseases. Isis'
partners are developing antisense drugs invented by Isis to treat a
wide variety of diseases. Ibis Biosciences, Inc., Isis' majority-owned
subsidiary, is developing and commercializing the Ibis T5000(TM)
Biosensor System, a revolutionary system to identify infectious
organisms. Isis is a joint owner of Regulus Therapeutics LLC, a joint
venture focused on the discovery, development and commercialization of
miRNA therapeutics. As an innovator in RNA-based drug discovery and
development, Isis is the owner or exclusive licensee of over 1,500
issued patents worldwide. Additional information about Isis is
available at www.isispharm.com.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel
therapeutics based on RNA interference, or RNAi. The company is
applying its therapeutic expertise in RNAi to address significant
medical needs, many of which cannot effectively be addressed with
small molecules or antibodies, the current major classes of drugs.
Alnylam is leading the translation of RNAi as a new class of
innovative medicines with peer-reviewed research efforts published in
the world's top scientific journals including Nature, Nature Medicine,
and Cell. The company is leveraging these capabilities to build a
broad pipeline of RNAi therapeutics; its most advanced program is in
Phase II human clinical trials for the treatment of respiratory
syncytial virus (RSV) infection. In addition, the company is
developing RNAi therapeutics for the treatment of a wide range of
disease areas, including hypercholesterolemia, liver cancers, and
Huntington's disease. The company's leadership position in fundamental
patents, technology, and know-how relating to RNAi has enabled it to
form major alliances with leading companies including Medtronic,
Novartis, Biogen Idec, and Roche. To reflect its outlook for key
scientific, clinical, and business initiatives, Alnylam has
established "RNAi 2010" which includes the company's plan to
significantly expand the scope of delivery solutions for RNAi
therapeutics, have four or more programs in clinical development, and
to form four or more new major business collaborations, all by the end
of 2010. Alnylam is a joint owner of Regulus Therapeutics LLC, a joint
venture focused on the discovery, development, and commercialization
of miRNA therapeutics. Founded in 2002, Alnylam maintains headquarters
in Cambridge, Massachusetts. For more information, visit
www.alnylam.com.
Forward-Looking Statement
This press release includes forward-looking statements regarding
the future therapeutic and commercial potential of Isis', Alnylam's
and Regulus' business plans, technologies and intellectual property
related to microRNA therapeutics being discovered and developed by
Regulus, including statements regarding Regulus' patent licensing
agreement with Stanford University and the potential to develop
microRNA antagonists to miR-181a. Any statement describing Isis',
Alnylam's or Regulus' goals, expectations, financial or other
projections, intentions or beliefs is a forward-looking statement and
should be considered an at-risk statement, including those statements
that are described as such parties' goals. Such statements are subject
to certain risks and uncertainties, particularly those inherent in the
process of discovering, developing and commercializing drugs that are
safe and effective for use as human therapeutics, and in the endeavor
of building a business around such products. Such parties'
forward-looking statements also involve assumptions that, if they
never materialize or prove correct, could cause their results to
differ materially from those expressed or implied by such
forward-looking statements. Although these forward-looking statements
reflect the good faith judgment of the management of each such party,
these statements are based only on facts and factors currently known
by Isis, Alnylam or Regulus, as the case may be. As a result, you are
cautioned not to rely on these forward-looking statements. These and
other risks concerning Isis', Alnylam's and Regulus' programs are
described in additional detail in Isis' annual report on Form 10-K for
the year ended December 31, 2007 and in Alnylam's annual report on
Form 10-K for the year ended December 31, 2007, which are on file with
the SEC. Copies of these and other documents are available from Isis
or Alnylam.
