Regulus Demonstrates Initial Progress in microRNA Biomarkers MS Collaboration and Receives Milestone Payment from Biogen Idec

Regulus Demonstrates Initial Progress in microRNA Biomarkers MS Collaboration
               and Receives Milestone Payment from Biogen Idec

PR Newswire

LA JOLLA, Calif., Nov. 20, 2013

LA JOLLA, Calif., Nov. 20, 2013 /PRNewswire/ -- Regulus Therapeutics Inc.
(NASDAQ:RGLS), a biopharmaceutical company leading the discovery and
development of innovative medicines targeting microRNAs, today announced that
it has demonstrated initial progress in its research collaboration with Biogen
Idec, M.A. ("Biogen Idec") to discover microRNAs as biomarkers for multiple
sclerosis ("MS") and has received a milestone payment.

Regulus has developed a highly reproducible, proprietary platform for
extracting, profiling, and analyzing microRNAs from small volumes of serum.
Under its collaboration with Biogen Idec, Regulus applied this platform to
profile over 400 serum samples from MS patients and compared these samples
against those from healthy volunteers in the largest known MS serum profiling
experiment performed to date. The results of this large and successful
experiment have provided the foundation for identifying potential biomarker
signatures for patients with MS. Based on the initial progress, Regulus and
Biogen Idec will now work on the next phase of the research collaboration to
validate further the microRNA biomarker signatures in patients with
relapse-remitting MS, the most common form of the disease.

"We are very excited about our early success in this collaboration and are
hopeful that our efforts advance Biogen Idec's work in treating MS," said Neil
W. Gibson, Ph.D., Chief Scientific Officer of Regulus."Further, we believe
that achievement of this milestone assigns significant value to our
proprietary technology and demonstrates the potential of our emerging
biomarkers platform to support our microRNA therapeutic pipeline." Regulus
believes that microRNA biomarkers may be used to select optimal patient
segments in clinical trials, to develop companion diagnostics, and to monitor
disease progression or relapse. 

In August 2012, Regulus entered into a research collaboration with Biogen Idec
to explore microRNAs as biomarkers for disease monitoring and response to
therapy.In June 2013, Regulus and Biogen Idec amended the collaboration
agreement to update the research plan and criteria for success. The
collaboration is initially focused on the discovery of microRNAs as biomarkers
for MS. Under the transaction, Regulus has received an upfront payment and has
earned an initial milestone payment and is also eligible to receive additional
milestone payments.Additionally, Biogen Idec made an investment in Regulus in
the form of a promissory note that converted into shares of Regulus' common
stock upon the closing of the Company's initial public offering in October
2012.

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 500 microRNAs have
been identified in the human genome, and over one-third of all human genes are
believed to be regulated by microRNAs. microRNA expression, or function, has
been shown to be significantly altered or dysregulated in many disease states,
including oncology, fibrosis and metabolic diseases. Regulus believes that
microRNAs are clinically relevant therapeutic targets and may be ideally
suited as biomarkers for these disease states and others, like MS.

microRNAs have been detected in bodily fluids such as blood, and emerging data
has demonstrated that microRNA signatures in blood can mimic the expression
profile observed in disease tissues.Regulus has a comprehensive intellectual
property estate and oligonucleotide technology know-how and believes that
microRNA biomarkers may be used to select optimal patient segments in clinical
trials, to develop companion diagnostics, and to monitor disease progression
or relapse.

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.Regulus is also developing 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, Regulus has formed a
research collaboration with Biogen Idec around its emerging microRNA
biomarkers platform.

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 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, Regulus Therapeutics Inc., 858-202-6300,
aconrad@regulusrx.com; Media: Liz Bryan, Spectrum Science,
lbryan@spectrumscience.com, 202-955-6222 x 2526
 
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