LA JOLLA, Calif., Dec. 19, 2017 /PRNewswire/ -- Regulus
Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company
leading the discovery and development of innovative medicines
targeting microRNAs, today announced that it has initiated the
first-in-human Phase I study of RGLS4326 and completed dosing of
the first cohort of healthy volunteers. RGLS4326 is in
development for the treatment of autosomal dominant polycystic
kidney disease, or ADPKD.
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"The initiation of this first-in-human study for RGLS4326 marks
an important clinical milestone for Regulus," said Dr. Timothy Wright, Regulus' Chief R&D
Officer. "Based on the strong preclinical evidence for the
role of miR-17 in animal models of PKD and in vitro testing of
human ADPKD cyst cell cultures, we believe that RGLS4326 has great
potential as a disease modifying therapy in ADPKD. This
program highlights the speed with which our scientists can progress
from oligonucleotide library synthesis to human testing, which in
the case of RGLS4326, was less than 24 months."
RGLS4326 is being studied in a Phase I randomized, double-blind,
placebo-controlled, single ascending dose study designed to
investigate the safety, tolerability, pharmacokinetics and
pharmacodynamics of RGLS4326 administered subcutaneously in healthy
volunteers.
About RGLS4326
RGLS4326 is a novel oligonucleotide
designed to inhibit miR-17 using a unique chemistry design to
preferentially target the kidney. Preclinical studies with RGLS4326
have demonstrated a reduction in kidney cyst formation, improved
kidney weight/body weight ratio, decreased cyst cell proliferation,
and preserved kidney function in mouse models of ADPKD.
About Autosomal Dominant Polycystic Kidney Disease
(ADPKD)
ADPKD, caused by mutations in the PKD1 or PKD2
genes, is among the most common human monogenetic disorders and a
leading genetic cause of end-stage renal disease. The clinical
hallmark of this disease is the development of multiple fluid
filled cysts primarily in the kidneys and to a lesser extent in the
liver and other organs. Excessive kidney tubule derived cyst
cell proliferation, a central pathological feature, drives the
expansion of cysts, ultimately causing end-stage renal disease in
approximately 50% of ADPKD patients by age 60. It is estimated that
approximately 1 in 1,000 people possess a mutation in the PKD1 or
PKD2 genes worldwide.
About Regulus
Regulus Therapeutics Inc. (Nasdaq: RGLS)
is a clinical stage biopharmaceutical company leading the discovery
and development of innovative medicines targeting microRNAs.
Regulus is leveraging its oligonucleotide drug discovery and
development expertise to develop a 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-012 or
RGLS4326), 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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