LA JOLLA, Calif., July 27, 2016 /PRNewswire/ -- Regulus
Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company
leading the discovery and development of innovative medicines
targeting microRNAs, today announced that, as anticipated, it
received written communication from the U.S. Food and Drug
Administration (FDA) outlining information required to resolve the
clinical hold for its Investigational New Drug (IND) for RG-101,
which was announced on June 27,
2016.
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In the written communication, the FDA requested the following
from the company: detailed safety data analysis from preclinical
and clinical studies; exploration of potential mechanisms of
hepatoxicity in non-clinical models; review and input from
independent hepatotoxicity experts; additional PK data from the US
Phase 1 study; and a risk/benefit assessment for the proposed
therapeutic regimens containing RG-101. The company
anticipates submitting the necessary information by early Q4
2016. The FDA will notify Regulus of its decision within 30
days of receipt of the complete response to the issues.
"We are working expeditiously to resolve the issues outlined in
the letter and anticipate a decision from the FDA in the fourth
quarter," said Paul Grint, M.D.,
President and Chief Executive Officer of Regulus. "Timelines of
on-going studies have not been impacted by the clinical hold, and
we remain on track to deliver follow-up results from ongoing RG-101
studies at upcoming relevant scientific meetings later this
year."
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.
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 RG-101 for HCV
RG-101 is Regulus' wholly-owned, GalNAc-conjugated anti-miR
targeting miR-122 for the treatment of HCV. In a completed Phase I
human proof-of-concept study, Regulus demonstrated that treatment
with a single subcutaneous dose of RG-101 as monotherapy resulted
in significant and sustained viral load reductions in all treated
HCV patients, including patients with difficult to treat genotypes,
various liver fibrosis status and those who have experienced viral
relapse after a prior IFN-containing regimen.
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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SOURCE Regulus Therapeutics Inc.