SAN DIEGO, June 21, 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
that it will present new preclinical data and additional clinical
data from the first cohort of patients in the Company's Phase
1b clinical trial of RGLS4326 for the
treatment of ADPKD at the PKD Connect Conference 2021, being held
virtually Friday, June 25.
Presentation Details:
- Title: Preclinical Evaluation and Results from the First
Cohort of Phase 1b Clinical Trial of
RGLS4326 for the Treatment of Patients with Autosomal Dominant
Polycystic Kidney Disease
- Date: June 25, 2021, 2:00–
3:00 p.m. CDT
- Free registration for the conference is available through
June 23, 2021 at
pkdcure.org/conference/
"We are excited to present the latest data from our RGLS4326
program for the treatment of ADPKD," commented Jay Hagan, President and Chief Executive Officer
of Regulus. "This year's conference is an opportune occasion to
share with the PKD community our additional data that further
validate miR-17 as a target for ADPKD. We look forward to an
engaging and productive event."
About RGLS4326 Phase 1b
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 PC1 and PC2 in patients with ADPKD
administered RGLS4326 every other week for a total of four
doses. To characterize the effect of RGLS4326 within each
cohort, biomarker values at the end of study are compared to
baseline values using a two-sided paired t-test. P-values less than
0.05 are considered statistically significant with no adjustment
for multiplicity. 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.
For more information about the clinical trial design, please
visit www.clinicaltrials.gov (NCT04536688).
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 study 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 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 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 San Diego,
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 clinical
activities concerning the RGLS4326 program, including the
preliminary biomarker, pharmacokinetic and safety data resulting
from the first cohort of patients from the ongoing clinical study,
the sufficiency of the data required to recommence clinical studies
for extended duration dosing, the timing of the Company's
interactions with FDA regarding the clinical hold and the timing
and of other 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.