LA JOLLA, Calif., Feb. 10, 2021 /PRNewswire/ -- Regulus
Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company
focused on the discovery and development of innovative medicines
targeting microRNAs (the "Company" or "Regulus"), today announced
it has completed enrollment of the first cohort of patients into
its Phase 1b clinical study of
RGLS4326. The Phase 1b is an
adaptive design, open-label, multiple dose study in up to three
cohorts of patients with ADPKD. The study is designed to
evaluate the safety, pharmacokinetics, and changes in levels of
polycystin 1 (PC1) and polycystin 2 (PC2) in patients with ADPKD
administered RGLS4326 every other week for a total of four doses.
The dose level for the first cohort is 1mg/kg of RGLS4326 and
the dose level for the second cohort is 0.3mg/kg. The third
and final cohort will be dosed at a level to be determined based on
the results of the first two cohorts. Concurrent with
completion of enrollment in the first cohort and based on the
review of the available interim safety data, the first patient of
the second cohort has received the first dose of RGLS4326.
"We are pleased to have reached this important step in our
ongoing RGLS4326 program for the treatment of ADPKD," said Jay
Hagan, CEO of Regulus. "To date, no serious adverse events have
been reported and results from the first cohort are expected to be
available early in the second quarter of this year."
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, reduction of cyst growth in human
in vitro ADPKD models, and attenuation of cyst proliferation
and improvement of kidney function in mouse models of
ADPKD. The RGLS4326 IND is currently on a Partial Clinical
Hold for treatment of extended duration by FDA until the
second set of requirements outlined by the agency have been
satisfactorily addressed. The Company will use information from the
Phase 1 clinical studies, including the first cohort of the Phase
1b together with information from the
recently completed additional nonclinical studies generated in
2020, in its plan to address the second set of requirements
outlined in the Partial Clinical Hold letter to support studies of
extended duration. Regulus plans to discuss its approach to
addressing the remaining Partial Clinical Hold requirements with
FDA in mid-2021. RGLS4326 has received orphan drug
designation from FDA in July
2020.
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 extended duration 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. In addition, while Regulus expects the
COVID-19 pandemic to adversely affect its business operations and
financial results, the extent of the impact on Regulus' ability to
achieve its preclinical and clinical development objectives and the
value of and market for its common stock, will depend on future
developments that are highly uncertain and cannot be predicted with
confidence at this time, such as the ultimate duration of the
pandemic, travel restrictions, quarantines, social distancing and
business closure requirements in the U.S. and in other countries,
and the effectiveness of actions taken globally to contain and
treat the disease. 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.