LA JOLLA, Calif., May 20, 2016 /PRNewswire/ -- Regulus
Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company
leading the discovery and development of innovative medicines
targeting microRNAs, today announced that new data on RG-012, the
company's anti-miR targeting microRNA-21 for the treatment of
Alport syndrome, a life-threatening kidney disease driven by
genetic mutations with no approved therapy, is being presented in
three posters during the 53rd European Renal
Association-European Dialysis and Transplant Association (ERA-EDTA)
Congress May 21-24 in Vienna, Austria.
Poster Presentations: May 22,
2016, 09:30-10:45AM
- Abstract SP120: Change in Glomerular Filtration Rate
and Renal Biomarkers in Patients with Chronic Kidney Disease Due to
Alport Syndrome: Interim Results from the ATHENA Study, a
Prospectively Designed Natural History Study (M. Rheault,
United States).
- Abstract SP283: Renal Impairment Effects on Plasma
and Tissue Exposure of Unconjugated and Galnac-conjugated Anti-miRs
in a Chronic Kidney Disease (CKD) Mouse Model (J. Grundy,
United States).
- Abstract SP257: Treatment with the MicroRNA-21
Inhibitor RG-012 Given with and without Ramipril Delays Renal
Impairment Progression and Prolongs Survival when Initiated up to
Chronic Kidney Disease (CKD) Stage 3 in a Mouse Model of Alport
Syndrome (J. Grundy, United
States).
The abstracts can be accessed through the ERA-EDTA website at
www.era-edta2016.org.
About RG-012 for Alport Syndrome
Alport syndrome is an inherited form of kidney disease caused by
mutations in the type IV collagen genes (Col4A3, Col4A4 and
Col4A5). Type IV collagen is important for maintaining the
integrity of the glomerular basement membrane (GBM), a vital
component in the kidney structure and filtration process. The
genetic mutation in the collagen gene results in thickening in the
GBM and impairment of glomerular filtration. Alport syndrome
patients experience a progressive loss of kidney function, which
ultimately leads to end stage renal disease requiring dialysis or
kidney transplantation, or may even lead to death. Alport syndrome
can also cause hearing loss and eye abnormalities during late
childhood or early adolescence. ACE (angiotensin-converting
enzyme) inhibitors are emerging as standard of care in patients
with Alport syndrome to treat proteinuria, or abnormal amounts of
protein in the urine, an indicator of chronic kidney disease.
Alport syndrome represents a high unmet medical need with no
approved therapy.
Currently, there is little known information on exactly how
Alport syndrome progresses, although miR-21 is believed to play a
role in the disease progression. miR-21 is up-regulated in Col4A3
deficient mouse models of Alport syndrome, other renal fibrosis
models and human CKD patients. The role of miR-21 has been
validated through genetic knock-out models and anti-miRs targeting
miR-21 have reduced the severity of fibrosis in two distinct
preclinical rodent models. Regulus is developing RG-012, a
single stranded, chemically modified oligonucleotide that binds to
and inhibits the function of miR-21 for the treatment of Alport
syndrome. In preclinical studies, RG-012 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
the Col4A3 deficient mice by up to fifty percent, and an additive
benefit in combination with an emerging standard of care
therapy. Further, RG-012 has received orphan drug status from
the U.S. Food and Drug Administration and European
Commission as a therapeutic in development for the treatment
of Alport syndrome.
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 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 leadership in the
microRNA field. 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. In addition, RG-125, a GalNAc-conjugated
anti-miR targeting microRNA-103/107 for the treatment of NASH in
patients with type 2 diabetes/pre-diabetes, has entered Phase I
clinical development through its strategic alliance with
AstraZeneca. Regulus is also advancing several programs
toward clinical development in renal, hepatic and central nervous
systems diseases, both independently and with our strategic
alliance partners, Sanofi and AstraZeneca. 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. 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-101), 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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