Regulus Renews Strategic Alliance with Sanofi to Focus on Orphan Disease and Oncology Targets and Receives Additional $10

 Regulus Renews Strategic Alliance with Sanofi to Focus on Orphan Disease and
    Oncology Targets and Receives Additional $10 Million Equity Investment

-Regulus to Develop microRNA-21 for Alport Syndrome, a Life-Threatening Orphan
Kidney Disease, and microRNA-21 in Oncology, to Human Proof-of-Concept-

-Sanofi Obtains Exclusive Option to Regulus' Program Targeting
microRNA-221/microRNA-222-

-Regulus' Milestones Remain with Upside in Potential Royalties-

PR Newswire

LA JOLLA, Calif., Feb. 5, 2014

LA JOLLA, Calif., Feb. 5, 2014 /PRNewswire/ -- Regulus Therapeutics Inc.
(NASDAQ:RGLS) announced today that it has renewed its strategic alliance with
Sanofi to discover, develop, and commercialize microRNA therapeutics to focus
on specific orphan disease and oncology targets. Regulus will lead
development of its fibrosis program targeting microRNA-21 ("miR-21") for the
treatment of Alport Syndrome, an orphan, life-threatening genetic kidney
disease with no approved therapy, and for its microRNA-21 ("miR-21") program
in oncology. Sanofi has retained its interest in these microRNA-21 programs
and has gained rights to Regulus' preclinical program targeting
microRNA-221/microRNA-222 ("miR-221/222"). Regulus is responsible for
advancing the clinical candidates in these programs to proof-of-concept.
Sanofi shall have the exclusive option, exercisable after proof-of-concept, to
take over further development and commercialization of each microRNA
therapeutic program. At this stage, Regulus will have the option to
co-promote any microRNA therapeutic product in the United States.

"The renewal of our strategic relationship with Sanofi further underscores the
commitment of both companies to realize the tremendous promise of RNA
therapeutics and the possibility to transform the field of drug discovery by
targeting microRNAs," said Kleanthis G. Xanthopoulos, Ph.D., President and CEO
of Regulus. "We believe that Regulus' microRNA therapeutic platform, coupled
with our focus on orphan diseases and oncology indications, combine perfectly
with Sanofi's resources and their proven capabilities as a global-healthcare
leader to bring innovative medicines to patients in need. We look forward to
advancing our programs together and building a meaningful clinical
portfolio."

The refocused relationship allows Sanofi and Regulus to continue to
collaborate on several meaningful microRNA therapeutic programs, with a
greater focus on orphan diseases and oncology. Under the original agreement
from 2010, Sanofi had rights on up to four microRNA targets, which included
Regulus' lead fibrosis program targeting miR-21. In 2012, the companies
expanded the alliance to collaborate on an oncology program targeting miR-21.
In 2013, the companies entered into an option letter agreement to allow for
negotiation of the extended strategic alliance announced today. Sanofi
retained its interest in developing microRNA-21 therapeutics for fibrosis and
oncology indications and now has opt-in rights to Regulus' miR-21 and
miR-221/222 program. If Sanofi chooses to exercise its option on any of these
programs, Sanofi will reimburse Regulus for a significant portion of its
preclinical and clinical development costs. Regulus continues to be eligible
to receive royalties on microRNA therapeutic products commercialized by
Sanofi.

Additionally, Sanofi has increased its ownership stake in Regulus through an
additional $10 million common stock investment at $7.67 per share, which
represents the volume-weighted average share price over the last 30 trading
days.

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, 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, or Media: Liz Bryan,
Spectrum Science, lbryan@spectrumscience.com, 202-955-6222 x2526
 
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