LA JOLLA, Calif.,
Oct. 16, 2019
/PRNewswire/ -- Regulus Therapeutics Inc.
(Nasdaq: RGLS), a biopharmaceutical
company focused on the discovery and development of innovative
medicines targeting microRNAs, today
announced that it presented preclinical data that identifies
RGLS4326, a first-in-class anti-miR-17 oligonucleotide, as a
potential disease-modifying treatment for autosomal dominant
polycystic kidney disease (ADPKD) at the Oligonucleotide
Therapeutics Society (OTS) Annual Meeting held October 13-16, 2019 in Munich, Germany.
The poster presentation is available for download
at
http://regulusrx.com/publications.
Poster Presentation #073: Discovery of anti-miR-17
oligonucleotide RGLS4326 for the treatment of autosomal dominant
polycystic kidney disease
"We are pleased to have made a presentation of our
RGLS4326 preclinical data during this year's OTS meeting and the
unique mechanism of targeting miR-17 in order to treat patients
with ADPKD," said Jay Hagan,
President and Chief Executive Officer of Regulus.
About ADPKD
ADPKD, caused by the mutations in the PKD1 or PKD2 genes,
is among the most common human monogenic disorders and a leading
cause of end-stage renal disease. The disease is characterized by
the development of multiple fluid filled cysts primarily in the
kidneys, and to a lesser extent in the liver and other organs.
Excessive kidney cyst cell proliferation, a central pathological
feature, ultimately leads to end-stage renal disease in
approximately 50% of ADPKD patients by age 60.
About RGLS4326
RGLS4326 is a novel oligonucleotide designed to inhibit
miR-17 and designed to preferentially target the kidney.
Preclinical studies with RGLS4326 have demonstrated direct
regulation of PKD1 and PKD2 in human ADPKD cyst cells, 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. The RGLS4326 IND is currently on a partial
clinical hold by the U.S. Food and Drug Administration.
About Regulus
Regulus Therapeutics Inc. (Nasdaq: RGLS) is a
biopharmaceutical company focused on the discovery and development
of innovative medicines targeting microRNAs. Regulus maintains its
corporate headquarters in La Jolla,
CA.
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
completion of preclinical and clinical activities concerning the
RGLS4326 program, including statements regarding the sufficiency of
the data resulting from the ongoing or planned preclinical studies
required to recommence the clinical studies and the timing of
preclinical and clinical activities. 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, and
feedback from the FDA. These and other risks 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.