SAN
DIEGO, April 12, 2023 /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 in the first cohort of patients in the
Phase 1b MAD study of RGSL8429 for
the treatment of ADPKD.
"Completion of enrollment in the first cohort of the Phase
1b MAD study is an important
milestone in the development of this novel treatment for ADPKD,"
said Jay Hagan, CEO of Regulus.
"Topline data from the first cohort are expected to be available
around the end of the third quarter of this year."
The Phase 1b MAD study is a
double-blind, placebo-controlled trial evaluating the safety,
tolerability, pharmacokinetics and pharmacodynamics (PK/PD) of
RGLS8429 in adult patients with ADPKD. The study will evaluate the
safety and PK/PD of RGLS8429 treatment across three different dose
levels, including measuring changes in polycystins, height-adjusted
total kidney volume (htTKV), and overall kidney function. The first
cohort is being dosed at 1 mg/kg of RGLS8429 or placebo every other
week for three months. The Company expects to begin dosing the
second cohort following a review of all available cohort 1 safety
data, which is planned for May 2023.
Cohort 2 patients will receive 2 mg/kg of RGLS8429 or placebo every
other week for three months.
More information about the MAD clinical trial is available at
clinicaltrials.gov (NCT05521191).
About ADPKD
Autosomal Dominant Polycystic
Kidney Disease (ADPKD), caused by 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. Approximately 160,000 individuals
are diagnosed with the disease in the
United States alone, with an estimated global prevalence of
4 to 7 million.
About RGLS8429
RGLS8429 is a novel, next
generation oligonucleotide for the treatment of
ADPKD designed to inhibit miR-17 and to preferentially target
the kidney. Administration of RGLS8429 has shown robust data in
preclinical models, where clear improvements in kidney function,
size, and other measures of disease severity have been demonstrated
along with a superior pharmacologic profile in preclinical studies
compared to Regulus' first-generation compound,
RGLS4326. Regulus announced completion of the Phase 1
SAD study in September 2022 and dosed
the first patient in the Phase 1b MAD
study in early November. The Phase 1 SAD study demonstrated that
RGLS8429 has a favorable safety and PK profile.
RGLS8429 was well-tolerated with no serious adverse events reported
and plasma exposure was approximately linear across the four doses
tested and is similar to the PK data from the
first-generation compound.
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 presentation 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 Company's RGLS8429 program,
including the expected timing for initiating clinical studies, the
expected timing for reporting topline data, and the
timing and future occurrence 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 the
risk additional toxicology data may be negative. 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, including under the "Risk
Factors" heading of Regulus' most recently filed
annual report on Form 10-K. 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.