Regulus Achieves All Research Milestones in Collaboration with Biogen to Identify microRNA Biomarkers for Multiple Sclerosis

LA JOLLA, Calif., Oct. 28, 2015 /PRNewswire/ -- Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today that it has earned a fourth milestone payment in its collaboration with Biogen (NASDAQ: BIIB) related to progress in the companies' research collaboration to identify microRNAs as biomarkers for multiple sclerosis ("MS").  To earn the milestone, Regulus' microMarkers^SM division used its robust technology platform and bioinformatics expertise to analyze the treatment effects of a Biogen relapsing MS drug on circulating microRNA profiles previously identified by Regulus. 

"Our partnership with Biogen is one example of how Regulus microMarkers^SM is researching the role and potential of microRNAs as biomarkers for numerous diseases.  We're excited to have identified a promising microRNA biomarker for MS, which we believe was a critical step to understand how microRNAs may change response to drug treatment," said Paul Grint, M.D., President and CEO of Regulus. "We're excited to have realized the full potential of the collaboration, and through this work, we're hopeful that Regulus' microMarkers^SM technology and bioinformatics expertise can improve understanding of multiple sclerosis." 

Regulus and Biogen began their collaboration in August 2012 and expanded their research in August 2014 to identify potential microRNA biomarkers in MS.  Under the scope of the agreements, Regulus has realized the full potential of the collaboration and has earned $3.7 million in payments from Biogen. Following achievement of the final milestone, the scope of the research to be performed under the current collaboration agreement has now concluded. 

About microRNAs and microRNAs as Biomarkers

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 one-third 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.  In addition to being clinically relevant therapeutic targets, microRNAs may be ideally suited as biomarkers for multiple diseases.  Regulus believes that microRNA biomarkers are of significant value and may be used to select optimal patient segments in clinical trials and to monitor disease progression or relapse. 

About Regulus microMarkers^SM

Regulus microMarkers^SM utilizes a highly reproducible technology platform to extract, profile, and analyze microRNAs from small volumes of bodily fluids to differentiate disease from healthy patient samples and to identify microRNAs as potential biomarkers for disease.  Regulus microMarkers^SM uses its proven expertise in microRNA profiling and bioinformatics to achieve meaningful interpretation of biomarker results.  To date, Regulus microMarkers^SM has profiled approximately 3,000 clinical samples in a wide variety of disease states including multiple sclerosis, chronic kidney disease, fatty liver, rheumatoid arthritis and others. Regulus has formed a research collaboration with Biogen focused on the discovery of microRNAs as biomarkers for MS and has also entered into an arrangement with another leading, commercial-stage pharmaceutical company to explore microRNAs as biomarkers for specific patient populations. Regulus also maintains several academic research collaborations focused on the identification of microRNAs as biomarkers in multiple disease areas.

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 microMarkers^SM 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.  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 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 regarding the expected ability of Regulus to undertake certain activities and accomplish certain goals (including with respect to its Regulus microMarkers^SM goals), the projected timeline of clinical development activities, and expectations regarding future therapeutic and commercial potential of Regulus' business plans (including Regulus' expected future activities), 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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