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RG-125 (AZD4076), a microRNA Therapeutic Targeting microRNA-103/107 for the Treatment of NASH in Patients with Type 2 Diabetes/Pre-Diabetes, Selected as Clinical Candidate by AstraZeneca

-Regulus Earns $2.5 Million Milestone Payment from AstraZeneca-
-RG-125 (AZD4076) is Regulus' 3rd Clinical Candidate, Achieving Key 'Clinical Map Initiative' Goal for 2015-

LA JOLLA, Calif., April 7, 2015 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today the selection of RG-125 (AZD4076), a GalNAc-conjugated anti-miR targeting microRNA-103/107 ("miR-103/107") for the treatment of Non Alcoholic Steatohepatitis ("NASH) in patients with type 2 diabetes/pre-diabetes, as a clinical candidate by AstraZeneca under the companies' strategic alliance to discover, develop and commercialize microRNA therapeutics.  RG-125 (AZD4076) is the first compound from the alliance to be selected for clinical development by AstraZeneca. In connection with the candidate selection, AstraZeneca will pay Regulus $2.5 million and will assume development of the program following acceptance of an Investigational New Drug application.  In the near term, Regulus and AstraZeneca plan to submit key preclinical data on the RG-125 (AZD4076) program to be presented at a scientific meeting later this year and expect to initiate a Phase I study of RG-125 (AZD4076) in humans by the end of 2015.

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"Regulus is very pleased that AstraZeneca has chosen to advance a microRNA therapeutic candidate from this exciting program toward the clinic.  RG-125 acts as a novel insulin sensitizer which we believe may inform a differentiated development path to treat patients with complicated metabolic disorders," said Neil W. Gibson, Ph.D., Chief Scientific Officer of Regulus.  "RG-125 represents our third clinical development candidate to arise from our novel technologies in less than two years, which confirms the productivity of our platform, achieves a key goal under our 'Clinical Map Initiative', and underscores our leadership in the microRNA therapeutics field."

Marcus Schindler, Head of Cardiovascular and Metabolic Diseases, Innovative Medicines and Early Development, AstraZeneca, said: "This is a tremendous achievement for our collaboration and an exciting step for AstraZeneca to be progressing AZD4076 (RG-125), an anti-microRNA molecule, into preclinical development. This again demonstrates AstraZeneca's commitment to innovation in science and to progress medicines for diseases with unmet medical need."

The role of miR-103/107 in insulin sensitivity and resistance was first recognized by the laboratory of Dr. Markus Stoffel, Professor of the Institute of Molecular Health Sciences at ETH Zurich and member of Regulus' Scientific Advisory Board. In mouse models of diabetes, Regulus has demonstrated that inhibition of miR-103/107 with its anti-miRs leads to a sustained reduction in fasting glucose and fasting insulin levels.  Additionally, anti-miRs targeting miR-103/107 function as unique insulin sensitizers as determined by hyperinsulinemic-euglycemic clamp, which is a robust method for assessing insulin sensitivity.  Further, inhibition of miR-103/107 with Regulus' anti-miRs decreases liver triglycerides and steatosis, providing an opportunity to investigate RG-125 (AZD4076) to treat NASH in patients with type 2 diabetes/pre-diabetes.

In August 2012, Regulus and AstraZeneca formed a strategic alliance to discover and develop microRNA therapeutics for cardiovascular diseases, metabolic diseases and oncology. Regulus and AstraZeneca aim to collaborate on three exclusive microRNA targets: AstraZeneca has selected miR-103/107, microRNA-19, and has a contractual right to select a third microRNA target. Today, Regulus announced that AstraZeneca selected the first clinical development candidate under the alliance, RG-125 (AZD4076), targeting miR-103/107 for the treatment of NASH in patients with type 2 diabetes/pre-diabetes.

To date, under the terms of the strategic alliance, AstraZeneca has paid $28.0 million to Regulus, which includes a $25.0 million equity investment (made concurrent with Regulus' initial public offering in October 2012) and a $3.0 million upfront payment. Regulus has earned a $2.5 million milestone payment for the clinical candidate selection announced today.  In addition to these payments, Regulus is eligible to receive up to $495.5 million in future milestone payments.  Under the terms of the agreement, Regulus will lead preclinical development of the programs under the strategic alliance, and if successful, could receive preclinical milestone payments. AstraZeneca will lead and fund the clinical development and commercialization of these programs.  In addition, Regulus could also receive clinical milestones based on the successful development of microRNA therapies in each target area. Regulus is eligible to receive significant launch and commercial milestone payments and royalties, contingent on the successful commercialization of microRNA therapeutic products by AstraZeneca.

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 AstraZeneca

AstraZeneca is a global, innovation-driven biopharmaceutical business with a primary focus on the discovery, development and commercialization of prescription medicines for gastrointestinal, cardiovascular, neuroscience, respiratory and inflammation, oncology and infectious disease. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information please visit:

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 domain dominant 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.  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 Idec 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-125), 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 Contact: Amy Conrad, Senior Director, Investor Relations and Corporate Communications, Regulus Therapeutics Inc., 858-202-6321,, or Media Contact: Liz Bryan, Spectrum Science,, 202-955-6222 x2526