Regulus Presents Positive Preclinical Data on miR-21 and miR-221 Oncology Programs In Oral and Poster Presentations at AACR 2014

  Regulus Presents Positive Preclinical Data on miR-21 and miR-221 Oncology
    Programs In Oral and Poster Presentations at AACR 2014 Annual Meeting

- Data Advances microRNA Oncology Portfolio -

-Oncology Portfolio Protected by Regulus' Strong Intellectual Property Estate-

PR Newswire

LA JOLLA, Calif., April 7, 2014

LA JOLLA, Calif., April 7, 2014 /PRNewswire/ -- Regulus Therapeutics Inc.
(NASDAQ:RGLS), a biopharmaceutical company leading the discovery and
development of innovative medicines targeting microRNAs, today announced that
three abstracts will be presented in poster presentation sessions during the
American Association for Cancer Research (AACR) Annual Meeting, which will be
held April 5-9, 2014 in San Diego, CA. An oral presentation will also be
presented by Regulus' strategic alliance partner, Sanofi. Data to be presented
include preclinical studies investigating the therapeutic potential of
targeting microRNA-21 ("miR-21") and microRNA-221 ("miR-221") for the
treatment of hepatocellular carcinoma ("HCC"), the most common form of liver
cancer, and biomarker discovery studies evaluating the pharmacodynamics
signature for assessing miR-221 inhibition and microRNA expression in tumor
tissues in mouse models of HCC. Each poster, after the time of its
presentation, can be accessed on the investor relations page of Regulus'
website, http://ir.regulusrx.com/

  oPoster Session on miR-221 - April 7, 2014 8:00 a.m. - 12:00 p.m. PDT

       omiR-221: a Potential Therapeutic Target for Hepatocellular Carcinoma
       oA miR-221 Multigene Pharmacodynamics Signature for Assessing miR-221
         Inhibition

  oOral Presentation by Sanofi on miR-21 – April 8, 2014 3:35 p.m. PDT

       oTargeting microRNA-21 in Hepatocellular Carcinoma

  oPoster Session on microRNA Expression - April 9, 2014 8:00 a.m. - 12:00
    p.m. PDT

       oSerum microRNAs Reflect microRNA Expression in Tumor Tissues as Well
         as Systemic Response to Disease in HRAS-driven Mouse Models of HCC

"Utilizing both genetic and pharmacological approaches, the data to be
presented at AACR provide further evidence that miR-21 and miR-221 are
validated preclinical targets for the treatment of hepatocellular carcinoma,"
said Neil W. Gibson, Ph.D., Chief Scientific Officer of Regulus. "In addition,
we've made progress to develop gene signatures that enable demonstration of
target inhibition in tumor tissues.We believe these gene signatures and the
overall approach enable Regulus to demonstrate proof of mechanism in
preclinical studies which may be translated into useful biomarker signatures
in potential future clinical trials."

Update on Regulus' Liver Cancer Intellectual Property Estate

As part of Regulus' rich intellectual property estate, Regulus controls patent
rights related to the modulation of multiple different microRNAs for the
treatment of liver cancer. Regulus has already received patents in the United
States for targeting multiple different microRNAs, including miR-21 and
miR-221/222, for the treatment of liver cancer. Also included in the Regulus
portfolio are fundamental patent rights claiming methods of treating liver
cancer with mimics of microRNA-34a, one of the most well-validated tumor
suppressor microRNAs.

Today, Regulus is pleased to announce receipt of a Decision to Grant a Patent
from the Japan Patent Office for the use of anti-miRs targeting multiple
different microRNAs, including miR-21 and miR-222, for the treatment of liver
cancer.

"We are pleased to see our liver cancer patent estate further bolstered with
new claims in a major market such as Japan. We believe our claims broadly
cover the use of pharmaceutically relevant compounds targeting any of these
multiple different microRNAs for the treatment of liver cancer," said David L.
Szekeres, Chief Business Officer and General Counsel of Regulus. "As
evidenced by these new claims, we continue to extract maximum value from our
broad intellectual property estate, solidifying Regulus' position as the
recognized leader in the field of microRNA therapeutics."

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, composed of approximately 200 patents and patent
applications that the company owns or has in-licensed from academic
institutions and third parties including its founding companies, Alnylam
Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc., related to microRNA and
microRNA drug products. Regulus also has access to approximately 850 patents
and patent applications exclusively 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 Regulus

Regulus Therapeutics Inc. (NASDAQ:RGLS) is a biopharmaceutical company leading
the discovery and development of innovative medicines targeting
microRNAs.Regulus is uniquely positioned to leverage a mature therapeutic
platform that harnesses the oligonucleotide drug discovery and development
expertise of Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc.,
which founded the company. Regulus has a well-balanced microRNA therapeutic
pipeline entering clinical development, an emerging microRNA biomarkers
platform to support its therapeutic programs, and a rich intellectual property
estate to retain its leadership in the microRNA field. Regulus intends to
focus its proprietary efforts on developing microRNA therapeutics for oncology
indications and orphan diseases and is currently advancing several programs
toward clinical development in oncology, fibrosis and metabolic diseases.
Specifically, Regulus is developing 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, and RG-101, a GalNAc-conjugated
anti-miR targeting microRNA-122 for the treatment of chronic hepatitis C virus
infection. Regulus' commitment to innovation and its leadership in the
microRNA field have enabled the formation of strategic alliances with
AstraZeneca, GlaxoSmithKline and Sanofi. In addition, the Company has
established Regulus microMarkers™, a research and development division focused
on identifying microRNAs as biomarkers of human disease, which is designed to
support its therapeutic pipeline, collaborators and strategic partners.

For more information, please visithttp://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 Regulus' expectations regarding future therapeutic and
commercial potential of Regulus' business plans, technologies and intellectual
property related to microRNA therapeutics 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'
programs are described in additional detail in Regulus' SEC filings. 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.

SOURCE Regulus Therapeutics Inc.

Website: http://www.regulusrx.com
Contact: Amy Conrad, Director, Investor Relations and Corporate
Communications, aconrad@regulusrx.com, 858-202-6321, Media: Liz Bryan,
Spectrum Science, lbryan@spectrumscience.com, 202-955-6222 x2526
 
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