LA JOLLA, Calif., April 7, 2015 /PRNewswire/ -- Regulus
Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company
leading the discovery and development of innovative medicines
targeting microRNAs, announced today the selection of RG-125
(AZD4076), a GalNAc-conjugated anti-miR targeting microRNA-103/107
("miR-103/107") for the treatment of Non Alcoholic Steatohepatitis
("NASH) in patients with type 2 diabetes/pre-diabetes, as a
clinical candidate by AstraZeneca under the companies' strategic
alliance to discover, develop and commercialize microRNA
therapeutics. RG-125 (AZD4076) is the first compound from the
alliance to be selected for clinical development by AstraZeneca. In
connection with the candidate selection, AstraZeneca will pay
Regulus $2.5 million and will assume
development of the program following acceptance of an
Investigational New Drug application. In the near term,
Regulus and AstraZeneca plan to submit key preclinical data on the
RG-125 (AZD4076) program to be presented at a scientific meeting
later this year and expect to initiate a Phase I study of RG-125
(AZD4076) in humans by the end of 2015.
"Regulus is very pleased that AstraZeneca has chosen to advance
a microRNA therapeutic candidate from this exciting program toward
the clinic. RG-125 acts as a novel insulin sensitizer which
we believe may inform a differentiated development path to treat
patients with complicated metabolic disorders," said Neil W. Gibson, Ph.D., Chief Scientific Officer
of Regulus. "RG-125 represents our third clinical development
candidate to arise from our novel technologies in less than two
years, which confirms the productivity of our platform, achieves a
key goal under our 'Clinical Map Initiative', and underscores our
leadership in the microRNA therapeutics field."
Marcus Schindler, Head of
Cardiovascular and Metabolic Diseases, Innovative Medicines and
Early Development, AstraZeneca, said: "This is a tremendous
achievement for our collaboration and an exciting step for
AstraZeneca to be progressing AZD4076 (RG-125), an anti-microRNA
molecule, into preclinical development. This again demonstrates
AstraZeneca's commitment to innovation in science and to progress
medicines for diseases with unmet medical need."
The role of miR-103/107 in insulin sensitivity and resistance
was first recognized by the laboratory of Dr. Markus Stoffel, Professor of the Institute of
Molecular Health Sciences at ETH Zurich and member of Regulus'
Scientific Advisory Board. In mouse models of diabetes, Regulus has
demonstrated that inhibition of miR-103/107 with its anti-miRs
leads to a sustained reduction in fasting glucose and fasting
insulin levels. Additionally, anti-miRs targeting miR-103/107
function as unique insulin sensitizers as determined by
hyperinsulinemic-euglycemic clamp, which is a robust method for
assessing insulin sensitivity. Further, inhibition of
miR-103/107 with Regulus' anti-miRs decreases liver triglycerides
and steatosis, providing an opportunity to investigate RG-125
(AZD4076) to treat NASH in patients with type 2
diabetes/pre-diabetes.
In August 2012, Regulus and
AstraZeneca formed a strategic alliance to discover and
develop microRNA therapeutics for cardiovascular diseases,
metabolic diseases and oncology. Regulus and AstraZeneca aim to
collaborate on three exclusive microRNA targets: AstraZeneca has
selected miR-103/107, microRNA-19, and has a contractual right to
select a third microRNA target. Today, Regulus announced that
AstraZeneca selected the first clinical development candidate under
the alliance, RG-125 (AZD4076), targeting miR-103/107 for the
treatment of NASH in patients with type 2
diabetes/pre-diabetes.
To date, under the terms of the strategic alliance, AstraZeneca
has paid $28.0 million to Regulus,
which includes a $25.0 million equity
investment (made concurrent with Regulus' initial public offering
in October 2012) and a $3.0 million upfront payment. Regulus has earned
a $2.5 million milestone payment for
the clinical candidate selection announced today. In addition
to these payments, Regulus is eligible to receive up to
$495.5 million in future milestone
payments. Under the terms of the agreement, Regulus will lead
preclinical development of the programs under the strategic
alliance, and if successful, could receive preclinical milestone
payments. AstraZeneca will lead and fund the clinical development
and commercialization of these programs. In addition, Regulus
could also receive clinical milestones based on the successful
development of microRNA therapies in each target area. Regulus is
eligible to receive significant launch and commercial milestone
payments and royalties, contingent on the successful
commercialization of microRNA therapeutic products by
AstraZeneca.
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 two-thirds 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 AstraZeneca
AstraZeneca is a global, innovation-driven biopharmaceutical
business with a primary focus on the discovery, development and
commercialization of prescription medicines for gastrointestinal,
cardiovascular, neuroscience, respiratory and inflammation,
oncology and infectious disease. AstraZeneca operates in over 100
countries and its innovative medicines are used by millions of
patients worldwide. For more information please visit:
www.astrazeneca.com.
About Regulus
Regulus Therapeutics Inc. (NASDAQ:RGLS) is a
biopharmaceutical company leading the discovery and development of
innovative medicines targeting microRNAs. Regulus has
leveraged its oligonucleotide drug discovery and development
expertise to develop a well-balanced microRNA therapeutics pipeline
complemented by a maturing microMarkersSM biomarkers
platform and a rich intellectual property estate to retain its
domain dominant leadership in the microRNA field. Under its
'Clinical Map Initiative', Regulus is developing RG-101, a
GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment
of chronic hepatitis C virus infection, and 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. Regulus is also advancing several programs
toward clinical development in orphan disease indications, oncology
and fibrosis. Regulus' commitment to innovation has resulted
in multiple peer-reviewed publications in notable scientific
journals and has resulted in the formation of strategic alliances
with AstraZeneca and Sanofi and a research collaboration with
Biogen Idec focused on microRNA biomarkers. Regulus maintains
its corporate headquarters in La
Jolla, CA. 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 the expected ability
of Regulus to undertake certain activities and accomplish certain
goals (including with respect to development and other activities
related to RG-125), 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.
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