LA JOLLA, Calif., March 25, 2015 /PRNewswire/ -- Regulus
Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company
leading the discovery and development of innovative medicines
targeting microRNAs, announced today that the European Commission
has granted orphan medicinal product designation for RG-012, a
single stranded, chemically modified oligonucleotide that binds to
and inhibits the function of microRNA-21 ("miR-21") for the
treatment of Alport syndrome, a life-threatening genetic kidney
disease with no approved therapy. In July
2014, the U.S. Food & Drug Administration granted orphan
drug designation to RG-012 for the treatment of Alport
syndrome.
"We are pleased to have received orphan medicinal product
designation in the European Union for RG-012, a key microRNA
therapeutic program under our 'Clinical Map Initiative'," 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."
Regulus is currently enrolling patients in a natural history of
disease study called ATHENA to gather information about the changes
in renal function over time in patients with Alport syndrome.
Data from the ATHENA study will provide the clinical basis for the
design of a Phase II study to monitor the therapeutic effect of
RG-012 on the decline in renal function and time to end-stage renal
disease in Alport syndrome patients. Under Regulus' 'Clinical
Map Initiative', the company expects to initiate a Phase I study
evaluating RG-012 in healthy volunteers in the first half of 2015
and a Phase II proof-of-concept study thereafter.
Orphan drug designation by the European Commission provides
regulatory and financial incentives for companies to develop and
market therapies that treat a life-threatening or chronically
debilitating condition affecting no more than five in 10,000
persons in the European Union (EU), and where no satisfactory
treatment is available. In addition to a 10-year period of
marketing exclusivity in the EU after product approval, orphan drug
designation provides incentives for companies seeking protocol
assistance from the European Medicines Agency during the product
development phase, and direct access to the centralized
authorization procedure.
About 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 glomerulus 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 used 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.
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 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-012), 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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