LA JOLLA, Calif., Feb. 13, 2020 /PRNewswire/ -- Regulus
Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company
focused on the discovery and development of innovative medicines
targeting microRNAs, today announced that it initiated dosing of
the second cohort of its Phase 1 multiple ascending dose clinical
trial (the "MAD study") of RGLS4326 for the treatment of autosomal
dominant polycystic kidney disease (ADPKD). The Company
expects to complete this study in mid-2020 with topline results
available thereafter. The Company is also planning to initiate a
Phase 1b short-term dosing study in
patients with ADPKD in the second half of 2020 to evaluate RGLS4326
for safety, pharmacokinetics, and biomarkers of pharmacodynamic
activity. RGLS4326 is a novel oligonucleotide designed to
inhibit miR-17 and to preferentially target the kidney.
"We are pleased to advance the RGLS4326 program with dosing of
the second cohort in the MAD study, which will allow us to further
characterize the safety and pharmacokinetic profile of RGLS4326
and establish the dose range that we plan to evaluate in the
Phase 1b study in ADPKD patients,"
said Jay Hagan, CEO of Regulus. "RGLS4326 represents a novel
approach to treating ADPKD, a genetic disease leading to
progressive loss of kidney function and kidney failure in the
majority of these patients."
The Company previously announced that it had completed both the
single ascending dose study in healthy volunteers up to the planned
highest dose, as well as the first dose level of the MAD study in
healthy volunteers. Clinical data generated to date showed that
RGLS4326 administration was generally well-tolerated with no
serious adverse events for all doses tested. Information from the
clinical studies together with information from additional
nonclinical studies will be used to address the requirements
outlined by FDA to support studies of extended duration.
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 for long-term chronic dosing 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 has leveraged its
oligonucleotide drug discovery and development expertise to develop
a pipeline complemented by a rich intellectual property estate in
the microRNA field. 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, the sufficiency of the data resulting from the ongoing or
planned preclinical studies required to recommence clinical studies
for long-term chronic dosing 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.