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Regulus Receives Orphan Drug Designation for RG-012, a microRNA Therapeutic for the Treatment of Alport Syndrome

 Regulus Receives Orphan Drug Designation for RG-012, a microRNA Therapeutic
                     for the Treatment of Alport Syndrome

PR Newswire

LA JOLLA, Calif., July 21, 2014

LA JOLLA, Calif., July 21, 2014 /PRNewswire/ --Regulus Therapeutics Inc.
(NASDAQ:RGLS), a biopharmaceutical company leading the discovery and
development of innovative medicines targeting microRNAs, announced today that
the U.S. Food & Drug Administration (FDA) has granted orphan drug designation
to RG-012, a single stranded, chemically modified oligonucleotide that binds
to and inhibits the function of microRNA-21 ("miR-21"), as a therapeutic for
the treatment of Alport syndrome, a life-threatening genetic kidney disease
with no approved therapy.

"We are pleased to have received orphan drug designation for RG-012 and are
encouraged by the FDA's recognition for the need of innovative new treatments
like microRNA therapeutics for rare and orphan diseases such as Alport
syndrome," said Paul Grint, M.D., Chief Medical Officer of Regulus. "Alport
syndrome is a life threatening disease and patients have very limited
treatment options because there is currently no approved therapy. We believe
that RG-012 represents an opportunity to make a significant impact in the
lives of patients with Alport syndrome and we look forward to advancing this
program into the clinic."

In the near term, Regulus expects to initiate a natural history of disease
study to gather further information about the progression of Alport syndrome
and to inform future clinical development plans of RG-012, a key program under
Regulus' 'Clinical Map Initiative'.

About Orphan Drug Designation

According tothe FDA, the Orphan Drug Designation program provides orphan
status to drugs and biologics which are defined as those intended for the safe
and effective treatment, diagnosis or prevention of rare diseases/disorders
that affect fewer than 200,000 people in the U.S., or that affect more than
200,000 persons but are not expected the costs of developing and marketing a
treatment drug. The granting of an orphan designation request does not alter
the standard regulatory requirements and process for obtaining marketing
approval. Safety and effectiveness of a drug must be established through
adequate and well-controlled studies.

About Alport Syndrome

Alport syndrome is a life-threatening genetic kidney disease that impacts the
body's ability to create a specific type of collagen highly expressed in the
kidney and essential to normal kidney structure.In the absence of this type
of collagen, the kidneys are unable to effectively filter toxins and waste
products, resulting in end-stage renal disease and also hearing loss or
effects on vision. Currently, there is no approved therapy for Alport
syndrome.

About RG-012 for the Treatment of Alport Syndrome

Regulus has discovered that miR-21 is highly overexpressed in mouse models of
Alport syndrome. Regulus is developing RG-012, a single stranded, chemically
modified oligonucleotide that binds to and inhibits the function of miR-21,
which has demonstrated potent inhibition of miR-21 in vitro and in vivo, a
decrease in the rate of progression of renal fibrosis, an increase in the
lifespan of mice by up to fifty percent, and a favorable pharmacokinetic
profile that supports the potential for a once per week dosing regimen. In
the near term, Regulus expects to initiate a natural history of disease study
to gather greater information about the progression of Alport syndrome and
expects to initiate a Phase I clinical study of RG-012 for the treatment of
Alport syndrome in the first half of 2015.

About microRNAs

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 800 microRNAs have
been identified in the human genome, and over one-third of all human genes are
believed to be regulated by microRNAs. A single microRNA can regulate entire
networks of genes. As such, these molecules are considered master regulators
of the human genome. microRNA expression, or function, has been shown to be
significantly altered or dysregulated in many disease states, including
oncology, fibrosis, metabolic diseases, immune-inflammatory diseases and HCV.
Targeting microRNAs with anti-miRs, chemically modified, single-stranded
oligonucleotides, offers a unique approach to treating disease by modulating
entire biological pathways and may become a new and major class of drugs with
broad therapeutic application.

About the 'Clinical Map Initiative'

Launched in February 2014, Regulus' 'Clinical Map Initiative' outlines certain
corporate goals to advance its microRNA therapeutics pipeline over the next
several years. Regulus expects todemonstrate human proof-of-concept results
in the Phase I clinical study of RG-101 for the treatment of HCV by the end of
2014, initiate a Phase I clinical study of RG-012 for the treatment of Alport
syndrome in the first half of 2015,nominate a third microRNA candidate for
clinical development by the end of 2014, andmaintain a strong financial
position and end 2014 with at least $75.0 million in cash, cash equivalents
and short-term investments.

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 therapeutics
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 and a research collaboration with
Biogen Idec focused on microRNA biomarkers. 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 financial estimates (including Regulus' projected cash at the
end of 2014), the projected sufficiency of Regulus' capital position for
future periods, the expected ability of Regulus to undertake certain
activities and accomplish certain goals (including with respect to development
and other activities related to RG-012 and RG-101 and with respect to the
nomination of a third microRNA candidate for clinical development), the
projected timeline of clinical development activities, and expectations
regarding future therapeutic and commercial potential of Regulus' business
plans, technologies and intellectual property related to microRNA therapeutics
and biomarkers 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' financial position and
programs are described in additional detail in Regulus filings with the
Securities and Exchange Commission. 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: Investor Relations Contact: Amy Conrad, Senior Director, Investor
Relations and Corporate Communications, Regulus Therapeutics Inc.,
858-202-6321, aconrad@regulusrx.com; Media Contact: Liz Bryan, Spectrum
Science, lbryan@spectrumscience.com, 202-955-6222 x2526
 
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