LA JOLLA, Calif., Jan. 27, 2017 /PRNewswire/ -- Regulus
Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company
leading the discovery and development of innovative medicines
targeting microRNAs, today announced that it received written
communication from the U.S. Food and Drug Administration (FDA) that
the clinical development program for RG-101 remains on clinical
hold. In June 2016, RG-101 was
placed on clinical hold following the Company's submission of a
second serious adverse event (SAE) of jaundice.
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Late last year, Regulus submitted a complete response to the
FDA's initial request for information, which included
identification of a potential mechanism of hyperbilirubinemia. The
Company also submitted a proposal to mitigate this risk.
Subsequently, the FDA has requested the final safety
and efficacy data from on-going RG-101 clinical and pre-clinical
studies before reconsidering the clinical hold. These data
will be available once the current study protocols are complete
through 48 weeks of follow up, which is anticipated in the fourth
quarter. The FDA also requested additional expert review of liver
safety data in light of the proposed mechanism of
hyperbilirubinemia.
"While we are disappointed that the clinical hold was not
lifted at this time, we plan to continue to work with the FDA to
address their additional requests as we seek the removal of
the clinical hold," said Dr. Timothy
Wright, Chief R&D Officer of Regulus.
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
rich intellectual property estate to retain its leadership in the
microRNA field. Regulus is advancing several programs in
renal, hepatic and central nervous systems diseases, both
independently and with our strategic alliance partners, Sanofi and
AstraZeneca. 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.
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.