Regulus Therapeutics, Inc. Receives Exclusive License From University of Wuerzburg for Intellectual Property to microRNA-21 for Diseases of the Heart, Liver, and Kidney

CARLSBAD, Calif.--(BUSINESS WIRE)--Regulus Therapeutics Inc. announced today that it has obtained exclusive rights from the University of Wuerzburg to intellectual property covering methods for modulating microRNA-21. Regulus scientists and collaborators have shown that microRNA-21, (miR-21), is dysregulated in mouse models of cardiac disease and fibrosis, and that antagonizing miR-21 with therapeutic oligonucleotides targeting miR-21 inhibits fibrosis and attenuates cardiac function in such models. Regulus is currently developing a microRNA therapeutic targeting miR-21 as an anti-fibrotic therapy.

“We are very pleased to obtain exclusive worldwide rights to this intellectual property from our collaborative work with the University of Wuerzburg, as it builds on our leading estate of assets needed for the development and commercialization of microRNA therapeutics,” said Garry E. Menzel, Ph.D., Executive Vice President of Corporate Development and Finance of Regulus Therapeutics. “Regulus scientists and collaborators were the first to show a therapeutic benefit from targeting miR-21 in a preclinical model of heart disease, and we are currently translating this academic discovery into a novel microRNA-based therapeutic for fibrotic diseases.”

The licensed technology relates to the discoveries that miR-21 is induced in fibroblasts in the failing heart, and that targeting miR-21 with therapeutic oligonucleotides can inhibit fibrosis and cardiac hypertrophy [Thum et al. (2008) Nature 456: 980-984].

The license has been grant by the Bayerische Patentallianz GmbH in Munich, the patent agency of Bavarian universities and research organization, including the University of Wuerzburg. Peer Biskup, CEO of the Bayerische Patentalianz, stated, “This license proves again the value of University inventions, the collaboration between industry and academia, and the importance of patent agencies in making technology transfer a success.”

In addition to the newly licensed patent rights covering therapeutic uses of miR-21, Regulus controls fundamental patent rights related to miR-21, including compositions of matter for various anti-miR compounds targeting miR-21.

About microRNAs

The discovery of microRNA in humans is one of the most exciting scientific breakthroughs in the last decade. microRNAs are small RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead regulate gene expression. Nearly 700 microRNAs have been identified in the human genome, and more than one-third of all human genes are believed to be regulated by microRNAs. As a single microRNA can regulate entire networks of genes, these new molecules are considered the master regulators of the 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 the possibility of a novel class of therapeutics and 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 based on microRNAs. Regulus is targeting microRNAs as a new class of therapeutics by working with a broad network of academic collaborators and leveraging oligonucleotide drug discovery and development expertise from its founding companies Alnylam Pharmaceuticals (Nasdaq:ALNY) and Isis Pharmaceuticals (Nasdaq:ISIS). Regulus is advancing microRNA therapeutics towards the clinic in several areas including hepatitis C infection, cardiovascular disease, fibrosis, oncology, immuno-inflammatory diseases, and metabolic diseases. Regulus’ intellectual property estate contains both the fundamental and core patents in the field as well as over 600 patents and more than 300 pending patent applications pertaining primarily to chemical modifications of oligonucleotides targeting microRNAs for therapeutic applications. In 2008, Regulus entered into a major alliance with GlaxoSmithKline to discover and develop microRNA therapeutics for immuno-inflammatory diseases. In 2010, Regulus entered into a new collaboration with GlaxoSmithKline to develop and commercialize microRNA therapeutics targeting microRNA-122 for the treatment of Hepatitis C Viral infection. 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 Isis’, Alnylam’s and Regulus’ business plans, technologies and intellectual property related to microRNA therapeutics being discovered and developed by Regulus, including statements regarding the therapeutic potential of targeting miR-21. 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. 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 Regulus’, Alnylam’s, and Isis’ programs are described in additional detail in Alnylam’s and Isis’ annual reports on Form 10-K for the year ended December 31, 2009 and their most recent quarterly reports on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from Alnylam or Isis.

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