Regulus Advances Orphan Disease Portfolio with Nomination of RG-012 as Clinical Development Candidate for the Treatment of

    Regulus Advances Orphan Disease Portfolio with Nomination of RG-012 as
     Clinical Development Candidate for the Treatment of Alport Syndrome

-RG-012 is an anti-miR Targeting microRNA-21 for the Treatment of Alport
Syndrome, a Life-Threatening, Genetic Kidney Disease with No Approved Therapy-

-Nomination Further Advances 'Road to the Clinic' Strategy-

PR Newswire

LA JOLLA, Calif., Feb. 19, 2014

LA JOLLA, Calif., Feb. 19, 2014 /PRNewswire/ --Regulus Therapeutics Inc.
(NASDAQ:RGLS), a biopharmaceutical company leading the discovery and
development of innovative medicines targeting microRNAs, today announced that
it has advanced its orphan disease portfolio with the nomination of its second
microRNA candidate for clinical development, RG-012, an anti-miR targeting
microRNA-21 ("miR-21") for the treatment of Alport Syndrome, a
life-threatening, genetic kidney disease with no approved therapy.

"The nomination of RG-012 as our second microRNA candidate for clinical
development is a significant achievement on our 'Road to the Clinic'
strategy," said Kleanthis G. Xanthopoulos, Ph.D., President and CEO of
Regulus. "This program underscores Regulus' focus on orphan disease
indications and we look forward to rapidly advancing and expanding our
clinical portfolio of meaningful microRNA therapeutics."

RG-012 has a very favorable preclinical profile to date which Regulus believes
supports clinical studies in man. RG-012 is a potent inhibitor of miR-21 in
both in vitro and in vivo preclinical models. Subcutaneous administration of
RG-012 has significantly decreased the rate of renal fibrosis and increased
the lifespan of the mice up to 50% in a mouse model of Alport Syndrome, which
we believe is a good surrogate for the human disease. Regulus believes these
preclinical survival results may translate to a similar increased lifespan in
human patients. Moreover, RG-012 has been well tolerated to date with a
favorable pharmacokinetic profile that supports the potential for a once/week
dosing regimen.

Neil W. Gibson, Ph.D., Regulus' Chief Scientific Officer added, "We believe
that RG-012 may become a transformative treatment for patients with Alport
Syndrome, a life-threatening, genetic disease with significant unmet medical
need. We are currently performing additional preclinical studies and
finalizing development plans for RG-012 and expect to enter clinical
development in the first half of 2015."

Regulus is responsible for advancing RG-012 to proof-of-concept. At that stage
of development, Regulus' strategic alliance partner, Sanofi, has an exclusive
option exercisable after proof-of-concept to assume all costs,
responsibilities and obligations for further development and commercialization
of RG-012. If Sanofi chooses to exercise its option on RG-012, Sanofi will
reimburse Regulus for a significant portion of its preclinical and clinical
development costs and will pay Regulus an option exercise fee. Regulus is
eligible to receive development and commercialization milestone payments and
will have an option to co-promote in the United States or receive royalty
payments in the mid 10% to 20% range.

Targeting miR-21 for the Treatment of Alport Syndrome

Alport Syndrome is a genetic condition caused by mutations in the COL4A3,
COL4A4, and COL4A5 genes that is characterized by kidney disease, hearing
loss, and eye abnormalities. The mutated genes provide instructions for
making one component of a protein called type IV collagen. This protein plays
an important role in the kidneys, specifically in structures called glomeruli.
Glomeruli are clusters of specialized blood vessels that remove water and
waste products from blood and create urine. The kidneys become less able to
function as this condition progresses, resulting in end-stage renal disease.
According to the National Institutes of Health, Alport Syndrome occurs in
approximately 1 in 50,000 newborns and is an orphan disease with no approved
therapy. 

miR-21 is a 22-mer non-coding RNA that negatively regulates gene/networks and
has been reported to be up-regulated in fibrotic kidney diseases in both
animal models and human patients (Chau, B. N. et al. Sci Transl Med. 2012;
Zhong X, et al. (2013) Diabetologia (2013). Preclinical studies have
demonstrated that treatment with an anti-miR-21 significantly attenuates
chronic kidney disease progression. Mechanistic understanding of inhibition of
miR-21 leading to anti-fibrotic efficacy continues to emerge and Regulus
believes that continued development of RG-012 for the treatment of Alport
Syndrome will contribute to the evolving understanding of targeting miR-21 for
fibrotic conditions.

About the 'Road to the Clinic' Strategy

Launched in February 2013, the 'Road to the Clinic' Strategy outlines certain
corporate goals that seek to advance our microRNA therapeutic pipeline toward
the clinic. Specifically, Regulus set the goal of nominating two microRNA
candidates for clinical development. Regulus has nominated RG-101, a
GalNAc-conjugated microRNA antagonist or anti-miR which targets miR-122 for
the treatment of HCV, and RG-012, an anti-miR targeting miR-21 for the
treatment of Alport Syndrome. Regulus believes its strong financial position
supports these stated goals and expects to end 2013 with approximately $110
million in cash, cash equivalents and short-term investments. Regulus plans
to update its 'Road to the Clinic' strategy and cash guidance on its fourth
quarter and year-end 2013 financial results webcast and conference call.

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