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Regulus to Present Updated Data Supporting RG-101 as Novel microRNA Therapeutic for the Treatment of HCV at The Liver Meeting® 2015 (AASLD)

-Phase I Clinical Data will be Featured in Viral Hepatitis Plenary Session-

LA JOLLA, Calif., Oct. 2, 2015 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today that two abstracts related to RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122 ("miR-122") for the treatment of chronic Hepatitis C Virus (HCV) infection, were accepted for presentation at The Liver Meeting®, the 66th Annual Meeting of the American Association for the Study of Liver Disease (AASLD), to be held November 13-17, 2015 in San Francisco, CA.

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Viral Hepatitis Plenary Session, November 17, 2015, 8:45am - 9:00am: A Single Dose of RG-101, a GalNAc-Conjugated Oligonucleotide Targeting miR-122, Results in Undetectable HCV RNA Levels in Chronic Hepatitis C Patients at Week 28 of Follow-up

  • Investigators will present extended follow-up data from the Phase I clinical study of RG-101 demonstrating that a single subcutaneous administration of either 2 mg/kg or 4 mg/kg of RG-101 as monotherapy resulted in HCV RNA levels below the limit of quantification in 6/22 patients with various HCV genotypes at week 28 of follow-up.

Poster Presentation, November 17, 2015, 8:00am - 12:00pm : Treatment with the Anti-miRNA122 Oligonucleotide RG-101 Results in a Decrease in IP-10 but Does Not Affect the Levels of Other Cytokines in Patients with Chronic Hepatitis C

  • Investigators will present data examining immunological changes observed in the plasma of patients who received a subcutaneous administration of RG-101 in the completed Phase I study.
  • Results demonstrated that HCV patients had significantly higher IP-10 levels at baseline compared to healthy controls. In HCV patients who received a subcutaneous administration of RG-101, a significant decline was observed in IP-10 serum concentrations compared to baseline, and RG-101 did not trigger a systemic immune activation.

"Regulus is pleased that our continued research in HCV will be featured at key sessions during the Liver Meeting, further demonstrating RG-101's novel mechanism, long duration of action and therapeutic potential," said Paul Grint, M.D., President and Chief Executive Officer of Regulus. "Our Phase II study evaluating RG-101 in combination with Harvoni®, Olysio®, and Daklinza® over four weeks is underway, patient dosing has begun, and we look forward to reporting interim data by the end of the year."

The abstracts related to the presentations can be accessed through the AASLD website. 

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), 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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For further information: Investor Relations, 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