Phase 1b
Multiple Ascending Dose (MAD) Study Initiated
RGLS8429 in healthy volunteers was
well-tolerated with dose-proportional PK
Topline data from first cohort of patients
with ADPKD anticipated in 1H 2023
SAN
DIEGO, Sept. 12, 2022 /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 positive topline safety and PK data from its Phase 1
SAD clinical trial of RGLS8429. Additionally, the Company announced
the initiation of its Phase 1b MAD
clinical trial of RGLS8429.
Summary of Results from Phase 1 SAD Study
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. Among the 32 subjects treated with
RGLS8429 or placebo, there were nine adverse events all of which
were mild, except one (sinus infection) which was graded moderate
in severity. Preliminary results suggest plasma exposure is
dose proportional across the four doses tested and compare
favorably to the PK data from the first-generation compound,
RGLS4326.
Phase 1b MAD Study
Initiated
The phase 1b MAD study is a
double-blind, placebo-controlled study in patients with
ADPKD. The primary objectives of the MAD study are to assess
safety, tolerability, and pharmacokinetics of RGLS8429 and to
evaluate the efficacy of RGLS8429 treatment across three different
dose levels, including changes in polycystins, cystic kidney volume
(htTKV), and overall kidney function. The first planned dose level
to be tested in patients with ADPKD is 1 mg/kg dosed every other
week for three months. Topline data from the first cohort of
patients is expected in 1H 2023.
More information about the MAD clinical trial is available at
clinicaltrials.gov (NCT05521191).
"The safety and PK data generated in our Phase 1 SAD trial are
highly encouraging and allow us the flexibility to use a range of
dose levels as we explore the safety and efficacy of RGLS8429 in
the Phase 1b MAD study," said
Jay Hagan, President and Chief
Executive Officer of Regulus Therapeutics. "We are excited about
the initiation of the MAD study, and we look forward to presenting
top line data from the first cohort of patients in the first half
of 2023."
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.
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 quarterly report on Form 10-Q. 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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