LA JOLLA, Calif., July 23, 2019 /PRNewswire/ -- Regulus
Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company
focused on the discovery and development of innovative medicines
targeting microRNAs, today announced a program update for RGLS4326
for the treatment of Autosomal Dominant Polycystic Kidney Disease
(ADPKD). The Company has been working with the United States Food
and Drug Administration (FDA) since July
2018, when Regulus voluntarily paused the Phase 1 clinical
program due to unexpected findings in the mouse chronic toxicity
study being run in parallel with the Phase 1 program. In
consultation with FDA, Regulus provided the interim analyses from a
new mouse chronic toxicity study and the non-human primate (NHP)
chronic toxicity study and supporting information to enable
re-initiation of the Multiple Ascending Dose (MAD) study. After
review of the requested submission, FDA notified the Company of
additional nonclinical data requirements and placed the IND on a
partial clinical hold, formalizing the specific requirements to
reinitiate the MAD study and further proceed into chronic dosing in
ADPKD patients.
The additional data requirements have been outlined in two
parts. In order to resume the MAD study, FDA has requested the
final reports from the chronic toxicity studies in both mice and
NHP and satisfactory related analyses to ensure subjects can be
safely dosed. Additional data and analyses from new
nonclinical studies, planned to be generated over the next several
quarters, will be required for chronic dosing, and may also be used
to support the resumption of the MAD study.
Regulus is allowed to proceed with additional Single Ascending
Dose (SAD) clinical studies as part of the process to gather
additional supporting information to guide the future development
of the program. The Company previously announced that it had
completed both the SAD 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.
"We appreciate the productive dialog with FDA throughout
this process," stated Jay Hagan, CEO
of Regulus. "They have provided clear requirements that we believe
we can address with data anticipated in the next several months
from ongoing studies, augmented by data to be generated from
additional studies that we expect to be completed early next
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 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.
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.