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Regulus Therapeutics Receives Exclusive License for Intellectual Property to microRNA-33a and microRNA-33b to Treat Diseases that Include Atherosclerosis and Metabolic Syndrome
Regulus scientists and collaborators from New York University were first to demonstrate reduction of atherosclerotic lesions with anti-miR-33 oligonucleotide in several preclinical models of disease
Regulus controls fundamental patent rights related to miR-33a and miR-33b, including composition of matter covered in the Tuschl III patent series and various chemically modified anti-miR compounds

LA JOLLA, Calif., Mar 30, 2011 (BUSINESS WIRE) --

Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative new medicines targeting microRNAs, today announced that it has obtained exclusive rights from New York University (NYU) for intellectual property covering methods of use in modulating microRNA-33a (miR-33a) and microRNA-33b (miR-33b) for metabolic diseases such as atherosclerosis and metabolic syndrome. Regulus scientists and collaborators from NYU were the first to show that antagonizing miR-33a and miR-33b with therapeutic oligonucleotide anti-miRs have several beneficial effects, including reduction in atherosclerotic plaque and increase in levels of high density lipoprotein (HDL) cholesterol, the 'good' cholesterol. Regulus is developing microRNA therapeutics targeting both miR-33a and miR-33b.

"Regulus scientists and our collaborators at NYU have shown that dysregulation of specific microRNAs, including miR-33a and miR-33b, can drive metabolic disease processes linked to dyslipidemia and atherosclerosis, opening up the possibility of developing a new class of drugs to treat these conditions," said Kleanthis G. Xanthopoulos, Ph.D., president and CEO of Regulus. "miR-33a and miR-33b are promising targets for therapeutic intervention to treat metabolic disease. We are combining our broad and proprietary microRNA therapeutic platform with a deep understanding of microRNA biology to advance this and other programs to the clinic."

The licensed technology relates to the discoveries that miR-33a and miR-33b down-regulate target genes involved in cholesterol transport and genes related to metabolic syndrome. Inhibition of miR-33a and miR-33b by anti-miRs increase circulating HDL cholesterol, suggesting that antagonism of miR-33a and miR-33b may be protective against atherosclerosis. Atherosclerosis is a condition in which artery walls thicken as a result of build-up of fatty material such as cholesterol. This build-up causes atherosclerotic lesions or plaques that can rupture, leading to slowing or stoppage of blood flow and ultimately resulting in myocardial infarction or stroke.

Kathryn Moore, Ph.D., and colleagues at NYU, in collaboration with Regulus, have further demonstrated that anti-miR-33 treatment in mice, which have a single copy of miR-33, reduces arterial lesions in models of atherosclerosis with established atherosclerotic plaques [Rayner et al (2010) Abstract 21739, American Heart Association scientific conference]. Non-human primates and humans have two copies of miR-33 (miR-33a and miR-33b), and technologies developed at Regulus have been used to demonstrate ways to inhibit both.

"Despite the currently available treatment options for atherosclerosis and conditions that increase its risk, like metabolic syndrome, coronary artery disease remains a major killer in the developed world pointing to the need for novel therapeutic approaches such as inhibiting miR-33," said Kathryn Moore, Ph.D., associate professor in NYU Langone Medical Center Department of Medicine. "Our initial work with NYU colleague Carlos Fernandez-Hernando, Ph.D. published in Science last year and additional preclinical research performed in collaboration with Regulus have shown that antagonizing miR-33a and miR-33b with anti-miRs can have significant impact on HDL raising and atherosclerotic plaque reduction. The growing preclinical data provides confidence that a microRNA therapeutic targeting miR-33a and miR-33b can have potential impact on this unmet medical need."

In addition to the newly licensed patent rights covering therapeutic uses of targeting miR-33a and miR-33b, Regulus controls fundamental patent rights related to miR-33a and miR-33b, including compositions of matter for the miR-33a and miR-33b sequence and complement covered in the Tuschl III patent series and various chemically modified anti-miR compounds targeting miR-33a and miR-33b discovered by Regulus.

About microRNAs

The discovery of microRNA in humans during the last decade is one of the most exciting scientific breakthroughs in recent history. microRNAs are small RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead regulate gene expression. More than 700 microRNAs have been identified in the human genome, and over one-third of all human genes are believed to be regulated by microRNAs. A single microRNA can regulate entire networks of genes. As such, these molecules are considered master regulators of the human genome. microRNAs have been shown to play an integral role in numerous biological processes, including the immune response, cell-cycle control, metabolism, viral replication, stem cell differentiation and human development. Most microRNAs are conserved across multiple species, indicating the evolutionary importance of these molecules as modulators of critical biological pathways. Indeed, microRNA expression or function has been shown to be significantly altered in many disease states, including cancer, heart failure and viral infections. Targeting microRNAs with anti-miRs, antisense oligonucleotide inhibitors of microRNAs, or miR-mimics, double-stranded oligonucleotides to replace microRNA function opens potential for a novel class of therapeutics and offers a unique approach to treating disease by modulating entire biological pathways. To learn more about microRNAs, please visit http://www.regulusrx.com/microrna/microrna-explained.php.

About Regulus Therapeutics Inc.

Regulus Therapeutics is a biopharmaceutical company leading the discovery and development of innovative new medicines targeting microRNAs. Regulus is using a mature therapeutic platform based on technology that has been developed over 20 years and tested in more than 5,000 humans. In addition, Regulus works with a broad network of academic collaborators and leverages the oligonucleotide drug discovery and development expertise of its founding companies, Alnylam Pharmaceuticals (NASDAQ:ALNY) and Isis Pharmaceuticals (NASDAQ:ISIS). Regulus is advancing microRNA therapeutics towards the clinic in several key areas including fibrosis, HCV, immuno-inflammatory diseases, metabolic diseases, and oncology. Regulus' intellectual property estate contains both the fundamental and core patents in the field and includes over 600 patents and more than 300 pending patent applications pertaining primarily to chemical modifications of oligonucleotides targeting microRNAs for therapeutic applications. In April 2008, Regulus formed a major alliance with GlaxoSmithKline to discover and develop microRNA therapeutics for immuno-inflammatory diseases. In February 2010, Regulus and GlaxoSmithKline entered into a new collaboration to develop and commercialize microRNA therapeutics targeting microRNA-122 for the treatment of hepatitis C infection. In June 2010, Regulus and sanofi-aventis entered into the largest-to-date strategic alliance for the development of microRNA therapeutics. This alliance is focused initially on fibrosis. For more information, visit http://www.regulusrx.com.

Forward-Looking Statements

This press release includes forward-looking statements regarding the future therapeutic and commercial potential of Regulus' business plans, technologies and intellectual property related to microRNA therapeutics being discovered and developed by Regulus. Any statement describing Regulus' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. 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 forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause the 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 Regulus' management, these statements are based only on facts and factors currently known by Regulus. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Regulus' programs are described in additional detail in each of Alnylam's and Isis' annual report on Form 10-K for the year ended December 31, 2010, which are on file with the SEC. Copies of these and other documents are available from either Alnylam or Isis.

SOURCE: Regulus Therapeutics Inc.

Regulus Therapeutics
Zachary Zimmerman, Ph.D., 858-202-6300
Russo Partners
Heidi Chokeir, Ph.D., 619-528-2217
"Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995: Statements in this press release regarding Alnylam Pharmaceuticals's business which are not historical facts are "forward-looking statements" that involve risks and uncertainties. For a discussion of such risks and uncertainties, which could cause actual results to differ from those contained in the forward-looking statements, see "Risk Factors" in the Company's Annual Report or Form 10-K for the most recently ended fiscal year.