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Regulus Announces Grant of Patents Covering Lead microRNA Therapeutics

LA JOLLA, Calif., Nov. 12, 2015 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs (miR), today announced that the U.S. Patent and Trademark Office ("USPTO") has granted patents related to Regulus' most advanced microRNA therapeutics, RG-101, the company's wholly-owned, GalNAc-conjugated anti-miR-122 being developed to treat HCV, and RG-012, an anti-miR-21 being developed to treat Alport syndrome. 

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  • RG-101 - claims of the patent, owned solely by Regulus, cover the unconjugated anti-miR-122 component of RG-101, the GalNAc-conjugated anti-miR-122 of RG-101, and pharmaceutical compositions comprising either the unconjugated or conjugated compounds. Regulus' patent portfolio related to RG-101 covers compositions and methods of use for the treatment of HCV in the United States and numerous foreign jurisdictions.
  • RG-012 – claims of the patents, owned solely by Regulus, cover the anti-miR-21 component of RG-012, as well as pharmaceutical compositions and methods of treatment of Alport syndrome using any one of a broad class of modified oligonucleotides complementary to miR-21.  Regulus' patent portfolio related to RG-012 covers compositions and methods of use for the treatment of Alport syndrome in the United States and numerous foreign jurisdictions.

"We are pleased with the USPTO's decision to issue these patents related to our lead therapeutic candidates, RG-101 and RG-012, further demonstrating our leadership in the microRNA field," said Paul Grint, MD., President and CEO of Regulus.  "We believe that Regulus' broad intellectual property estate, including the granted patents announced today, will protect the innovative medicines we hope to bring to patients in need."

About Regulus' Intellectual Property Estate

Regulus believes that it has a leading intellectual property position and substantial know-how relating to the development and commercialization of microRNA therapeutics in a wide range of therapeutic areas.  Regulus has built a portfolio of over 250 patents and patent applications world-wide, protecting the company's clinical candidates, therapeutic modulation of microRNAs with anti-miRs or mimics, and platform technologies. Regulus also has access to approximately 850 patents and patent applications related to RNA technologies, including patents and patent applications relating to chemical modification of oligonucleotides that are useful for the development of microRNA therapeutics.

About microRNAs

The discovery of microRNAs 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 800 microRNAs have been identified in the human genome, and over two-thirds 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.  microRNA expression, or function, has been shown to be significantly altered or dysregulated in many disease states, including oncology, fibrosis, metabolic diseases, immune-inflammatory diseases and HCV. Targeting microRNAs with anti-miRs, chemically modified, single-stranded oligonucleotides, offers a unique approach to treating disease by modulating entire biological pathways and may become a new and major class of drugs with broad therapeutic application.

About Regulus

Regulus Therapeutics Inc. (NASDAQ:RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs.  Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a well-balanced microRNA therapeutics pipeline complemented by a maturing microMarkersSM biomarkers platform and a rich intellectual property estate to retain its leadership in the microRNA field.  Under its 'Clinical Map Initiative', Regulus is developing RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment of chronic hepatitis C virus infection, and RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport syndrome, a life-threatening kidney disease driven by genetic mutations with no approved therapy.  In addition, RG-125, a GalNAc-conjugated anti-miR targeting microRNA-103/107 for the treatment of NASH in patients with type 2 diabetes/pre-diabetes, has been selected for clinical development.  Regulus is also advancing several programs toward clinical development in orphan disease indications, oncology and fibrosis.  Regulus' commitment to innovation has resulted in multiple peer-reviewed publications in notable scientific journals and has resulted in the formation of strategic alliances with AstraZeneca and Sanofi and a research collaboration with Biogen focused on microRNA biomarkers.  Regulus maintains its corporate headquarters in La Jolla, CA.  For more information, please visit

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Regulus to undertake certain activities and accomplish certain goals (including with respect to development and other activities related to RG-101 and RG-012), the projected timeline of clinical development activities, and expectations regarding future therapeutic and commercial potential of Regulus' business plans, technologies and intellectual property related to microRNA therapeutics and biomarkers being discovered and developed by Regulus.  Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect.  Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with 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 drugs.  These and other risks concerning Regulus' financial position and programs are described in additional detail in Regulus filings with the Securities and Exchange Commission.  All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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SOURCE Regulus Therapeutics Inc.

For further information: Investor Relations, Amy Conrad, Regulus Therapeutics Inc., 858-202-6321,; Media, Liz Bryan, Spectrum Science,, 202-955-6222 x2526