Regulus Therapeutics Inc. today announced that it achieved the initial
milestone with GlaxoSmithKline (GSK) as part of their ongoing worldwide
strategic alliance established to discover, develop and market novel
microRNA-based therapeutics to treat inflammatory diseases. The two
companies are working to identify drugs directed at four different
microRNA targets related to inflammatory disease. Regulus has now
reached an important discovery milestone, which triggered a payment,
concurrent with the first demonstration of a pharmacological effect in
immune cells by specific microRNA inhibition. Scientists successfully
delivered specific microRNA inhibitors, known as anti-miRs, to mice and
clearly identified changes in expression of the genes regulated by the
microRNA in immune cells.
"This milestone is further evidence that microRNAs represent disease
targets whose therapeutic modulation could revolutionize the way we
treat immune diseases,” said Peter S. Linsley, Ph.D., Chief Scientific
Officer at Regulus. "We believe that we are the leaders in microRNA and
are delighted to have such a good partner as GSK supporting the
translation of our groundbreaking research into therapeutic
opportunities. We are particularly pleased that this has been achieved
in the first year of the collaboration.”
The Regulus-GSK alliance, which has a potential value of nearly $600
million, was established in April 2008 to focus on inflammatory
diseases. Regulus is responsible for the development of antagonists to
four microRNA targets from discovery through completion of clinical
proof of concept. GSK has an exclusive license for worldwide development
and commercialization of drugs developed by Regulus under each program
for the relevant microRNA. The alliance combines Regulus’ unique
expertise and comprehensive intellectual property estate in the microRNA
therapeutics field with GSK’s extensive inflammatory disease expertise.
About microRNAs
microRNAs are a recently discovered class of genetically encoded
endogenous RNAs, approximately 20 nucleotides in length, that are
believed to regulate the expression of a large number of human genes.
microRNAs have been described as being involved in the regulation of
gene expression as part of physiology and development. microRNA-based
therapeutics represent a new approach for the treatment of a wide range
of human diseases. The inappropriate absence or presence of specific
microRNAs in various cells has been shown to be associated with specific
human diseases including cancer, viral infection, cardiovascular,
metabolic disorders and immuno-inflammatory disease. Targeting microRNAs
with novel oligonucleotide therapeutic agents could result in novel and
broadly acting treatments for a number of serious human diseases.
About Regulus Therapeutics Inc.
Regulus Therapeutics Inc. is a biopharmaceutical company aiming to
discover, develop, and commercialize microRNA-based therapeutics.
Regulus aspires to translate one of the most important new discoveries
in biology into a novel approach for innovative medicines and to build
the leading microRNA company. Regulus was established by Alnylam
Pharmaceuticals (Nasdaq: ALNY) and Isis Pharmaceuticals (Nasdaq: ISIS)
in September of 2007 and benefits substantially from their RNA
therapeutic drug discovery expertise, unique and established
oligonucleotide-based technologies, and a broad and dominant
intellectual property estate specific to microRNA-based therapeutics.
Regulus’ patent estate includes over 600 patents and more than 300
pending patent applications pertaining primarily to chemical
modifications of oligonucleotides targeting microRNA for therapeutic
applications. In addition, through its numerous collaborations with
academic researchers, Regulus continues to expand its knowledge on the
biological role of therapeutically relevant microRNAs. In April 2008,
Regulus formed a major alliance with GlaxoSmithKline to explore microRNA
therapeutics for immuno-inflammatory diseases. Regulus is headquartered
in Carlsbad, California. For more information, visit www.regulusrx.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 the relationship between Regulus and GSK. 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, 2008, and its most recent quarterly report on Form 10-Q, and in
Alnylam’s annual report on Form 10-K for the year ended December 31,
2008, and its most recent quarterly report on Form 10-Q, which are on
file with the SEC. Copies of these and other documents are available
from Isis or Alnylam.
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