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:
- On 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
- On 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
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 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.