RG-125(AZD4076), a microRNA Therapeutic Targeting microRNA-103/107 Being Developed for the Treatment of NASH in Patients with Type 2 Diabetes/Pre-Diabetes, Enters Phase I Clinical Development

-Regulus Earns $10.0 Million Milestone Payment from AstraZeneca and Achieves Key Goal for 2015-

Dec 18, 2015, 08:00 ET from Regulus Therapeutics Inc.

LA JOLLA, Calif., Dec. 18, 2015 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today that dosing has begun in a first-in-human Phase I clinical study of RG-125(AZD4076), by its collaboration partner AstraZeneca. RG-125(AZD4076) is a GalNAc-conjugated anti-miR-103/107 oligonucleotide that has been shown to improve insulin sensitivity and glucose tolerance in animal models. RG-125(AZD4076) was jointly identified and selected as a clinical candidate in April 2015 by AstraZeneca under the companies' strategic alliance to discover, develop and commercialize microRNA therapeutics. AstraZeneca will pay Regulus $10.0 million and will assume further development of RG-125(AZD4076).  

Previous research has demonstrated a causative role of microRNAs in pathophysiological processes of metabolic diseases, and increased expression of miR-103/107 in the liver has been associated with insulin resistance in people with non-alcoholic steatopheatitis (NASH), or fatty liver disease.  In mechanistic studies, RG-125(AZD4076) showed effects on biological pathways implicated in NASH progression.

"RG-125(AZD4076) acts as a novel insulin sensitizer which represents a potential new mechanism to treat patients with metabolic diseases such as type 2 diabetes and NASH," said Paul Grint, MD, President and Chief Executive Officer of Regulus. "Regulus is pleased to see the advancement of our third microRNA therapeutic program into clinical development and we look forward to working with AstraZeneca to bring this novel treatment one step closer to patients in need."

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 (RG-125(AZD4076)). In addition to the $2.5 million milestone payment for the clinical candidate selection announced in April and the $10.0 million milestone payment for RG-125(AZD4076) entering Phase I announced today, Regulus is eligible to receive future milestone payments under the terms of the strategic alliance agreement, which include significant launch and commercial milestone payments and royalties.

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: www.astrazeneca.com.

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 entered Phase I clinical development through its strategic alliance with AstraZeneca.  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 http://www.regulusrx.com.

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.



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