LA JOLLA, Calif., June 12, 2017 /PRNewswire/ -- Regulus
Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical
company leading the discovery and development of innovative
medicines targeting microRNAs, today announced updates to its
pre-clinical and clinical development programs.
"We are squarely focused on taking the steps necessary to
advance our pipeline and continue building shareholder
value. To that end, we recognize that we must be disciplined
in our investment choices and focus our resources and capital on
our most promising discovery and development programs, including
the application of important development, regulatory and commercial
considerations," said Jay Hagan,
President and Chief Executive Officer of Regulus. "MicroRNA
therapeutics have the potential to become an important new class of
drugs with broad therapeutic
application. Regulus' focus will be in diseases
with significant unmet medical need in organs to which we have been
able to preferentially deliver oligonucleotide therapeutics
effectively, such as the liver and kidney."
RG-012 for Alport syndrome: Initiation
of the Phase II clinical programs for RG-012 for the treatment of
Alport syndrome is on track as planned. Important changes to
the Phase II study design have been incorporated with the goal to
accelerate patient enrollment, improve statistical power, and
potentially achieve proof of mechanism data by the end of
2017. HERA, the Phase II randomized (1:1), double-blinded,
placebo-controlled study evaluating the safety and efficacy of
RG-012 in Alport syndrome patients, has been modified to increase
enrollment to 40 patients to improve its statistical power. Dose
frequency has also been adjusted to once every other week. The
separate renal biopsy study will evaluate RG-012 renal tissue
pharmacokinetics, target engagement and downstream effects on
genomic disease biomarkers. Data from the renal biopsy study is
anticipated by year-end and interim data from HERA is anticipated
mid-2018.
RG-101 (anti-miR122) for
HCV: The Company announced today that
it plans to discontinue clinical development of
RG-101 upon completion of the one remaining clinical study,
which is expected to occur in July
2017. Comprehensive pre-clinical investigation and thorough
evaluation of the clinical data from RG-101 has led to the
identification of a bilirubin transport mechanism as the likely
cause for the cases of hyperbilirubinemia in the RG-101
program. We believe that a combination of factors including
inhibition of conjugated bilirubin transport by RG-101, impaired
baseline bilirubin transport in HCV patients and the preferential
uptake of RG-101 by hepatocytes contributed to this mechanism.
Additional patient specific contributing factors cannot be
excluded. Applying the learnings from the RG-101
program, alternative
compounds targeting miR-122 have been
identified that maintain potent
HCV antiviral activity while lacking inhibition
of the bilirubin transporter. These compounds have the
potential for rapid clinical proof-of-concept of a novel,
markedly shortened treatment regimen for HCV and will be
considered for further development pending an updated global
commercial market assessment for HCV.
RGLS4326 (anti-miR-17) for autosomal dominant
polycystic kidney disease (ADPKD): The
IND for RGLS4326 is on track for filing by year end 2017. IND
enabling toxicology, repeat pharmacology and manufacturing work
have been completed as scheduled to support regulatory submissions.
Data from the pre-clinical program have been recently published in
Nature Communications and support the rationale for
targeting miR-17 for the treatment of ADPKD, an orphan indication
with no treatment options affecting approximately 600,000 people in
the United States.
RGLS5040 (anti-miR-27) for cholestatic
disease: RGLS5040, an unconjugated inhibitor
of microRNA27, has been discontinued based on a positioning of the
compound with respect to the competitive landscape coupled with the
results from repeat pharmacology studies as part of IND-enabling
work. The Company continues to work on developing highly
effective therapeutics for genetic forms of cholestatic disease as
part of its overall research activities targeting unmet diseases of
the liver and kidney.
"With the focus of our efforts on the most promising
programs in our portfolio and the opportunity to explore additional
novel clinical applications of miRNA targeted therapeutics, I am
more excited than ever about the opportunity to deliver
game-changing therapeutics for patients with great unmet medical
need," said Dr. Timothy Wright,
Regulus' Chief R&D Officer.
Separately, AstraZeneca
informed the Company that it intends
to terminate the clinical development program for AZD4076(RG-125)
for the treatment of NASH in Type 2
Diabetes/Pre-diabetes. Pursuant to the terms of the licensing
agreement, AstraZeneca's
rights with respect to AZD4076(RG-125) will
revert to Regulus when the termination
becomes effective in twelve months. AZD4076(RG-125) was
jointly identified and selected as a clinical candidate
in April
2015 by AstraZeneca under the companies'
strategic alliance to discover, develop and commercialize microRNA
therapeutics.
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 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.
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 or 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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SOURCE Regulus Therapeutics Inc.