SAN
DIEGO, Nov. 2, 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
the dosing of the first patient in the Phase 1b MAD study of RGSL8429 for the treatment of
Autosomal Dominant Polycystic Kidney Disease (ADPKD).
"Dosing of the first patient in the MAD clinical trial of
RGLS8429 marks yet another important achievement in our ADPKD
program," said Jay Hagan, President and Chief Executive
Officer of Regulus Therapeutics. "This study is an important step
as we advance RGLS8429 in ADPKD and look forward to its
continued development as a potential targeted treatment option for
patients with ADPKD."
The Phase 1b MAD study is a
double-blind, placebo-controlled trial to assess safety,
tolerability, and pharmacokinetics of RGLS8429 in adult patients
with ADPKD. The study will evaluate the safety and efficacy of
RGLS8429 treatment across three different dose levels including
measuring changes in polycystins, cystic kidney volume (htTKV), and
overall kidney function. The first cohort will be dosed at 1 mg/kg
of RGLS8429 or placebo every other week for three months. Topline
data from the first cohort of patients is expected in mid-2023.
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. Regulus announced completion of the
Phase 1 SAD study in September 2022. 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. Preliminary results suggest plasma exposure
is dose proportional across the four doses evaluated and are
similar to the PK data from the first-generation compound,
RGLS4326.
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 clinical trials involving RGLS8429
for the treatment of autosomal dominant polycystic kidney disease,
including the planned initiation of clinical trials involving ADPKD
patients, timing of data and potential outcome of such clinical
studies. 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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SOURCE Regulus Therapeutics Inc.