Regulus Presents Positive Preclinical Data Demonstrating that microRNA-21 Plays an Important Role in Alport Syndrome

  Regulus Presents Positive Preclinical Data Demonstrating that microRNA-21
                  Plays an Important Role in Alport Syndrome

- New Survival Data Presented at Kidney Week 2013 -

- Alport Syndrome is a Life-Threatening, Genetic Kidney Disease with No
Approved Therapy -

PR Newswire

LA JOLLA, Calif., Nov. 8, 2013

LA JOLLA, Calif., Nov. 8, 2013 /PRNewswire/ --Regulus Therapeutics Inc.
(NASDAQ:RGLS), a biopharmaceutical company leading the discovery and
development of innovative medicines targeting microRNAs, today announced that
positive new preclinical data demonstrating that microRNA-21 (miR-21) plays an
important role in Alport Syndrome will be presented at the American Society of
Nephrology's Kidney Week 2013 meeting being held November 5-10 in Atlanta, GA.

In a poster presentation titled "Identification of the Pathologic Role of
miR-21 in Alport's Kidney Disease" on Friday, November 8, 2013 from 10:00 a.m.
EST to 12:00 p.m. EST, Regulus and Genzyme, a Sanofi company, presented new
preclinical data demonstrating that miR-21 plays an important role in the
disease progression of Alport Syndrome in collagen 4A3 deficient mice. The
study assessed urine albumin, kidney function, pathology and gene expression
in the mice at 4, 6, 9, 12 and 15 weeks of age. Efficacy of an anti-miR-21
candidate was assessed in mice starting at 5 weeks of age. Urine albumin,
renal function, kidney pathology and lifespan were used as efficacy endpoints.
Treatment with an anti-miR-21 candidate significantly improved renal
function, significantly reversed regulated genes and pathways associated with
renal pathology, and increased the lifespan of the mice by 20 percent. The
poster can be accessed on Regulus' website, http://www.regulusrx.com.

"We are extremely pleased with the results from this study and believe these
positive data validate that treatment with an anti-miR-21 candidate may be an
important new therapy for patients with Alport Syndrome," said Neil W. Gibson,
Ph.D., Chief Scientific Officer at Regulus. "These data bring us one step
closer toward the selection of an anti-miR-21 clinical development candidate
and we remain excited about the potential to bring this innovative treatment
to patients with this devastating disease."

Additionally, microRNA therapeutics will be discussed in two oral
presentations at the meeting:

  oOn Friday, November 8, 2013 at 2:00 p.m. EST, Jeremy Duffield M.D., Ph.D.,
    Regulus' collaborator at the University of Washington, will give an oral
    presentation titled "MicroRNAs Are Novel Therapeutic Targets to Treat
    Kidney Injury and Fibrosis"; and
  oOn Saturday, November 9, 2013 at 5:42 p.m. EST, Regulus scientists and
    representatives from its collaborator at the University of Washington will
    give an oral presentation titled "Anti-miR21 Protects Collagen 4A3
    Deficient Mice from Progression of Alport Disease by Decreasing Oxidative
    Stress".

About miR-21 and Alport Syndrome

According to the National Institutes of Health, Alport Syndrome occurs in
approximately 1 in 50,000 newborns. Alport syndrome is a genetic condition
characterized by kidney disease, hearing loss, and eye abnormalities. The
kidneys become less able to function as this condition progresses, resulting
in end-stage renal disease (ESRD).

Mutations in the COL4A3, COL4A4, and COL4A5 genes cause Alport Syndrome. These
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.
Mutations in these genes result in abnormalities of the type IV collagen in
glomeruli, which prevents the kidneys from properly filtering the blood and
allows blood and protein to pass into the urine.

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). Previous reports have
demonstrated that treatment with an anti-miR-21 significantly attenuates
chronic kidney disease progression in a collagen 4A3 deficient mouse model.
Regulus' miR-21 therapeutic development program, which is partnered with
Sanofi, is currently in preclinical testing for both kidney fibrosis and
hepatocellular carcinoma.

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 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, 858-202-6321, aconrad@regulusrx.com, or Media, Liz Bryan,
Spectrum Science, 202-955-6222 x2526, lbryan@spectrumscience.com
 
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