SAN
DIEGO, June 13, 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 healthy volunteer in the Phase 1 SAD study
of RGSL8429 for the treatment of Autosomal Dominant Polycystic
Kidney Disease (ADPKD).
"The initiation of the clinical trial of RGLS8429 with the
dosing of the first subject marks another important milestone for
our program targeting the treatment of ADPKD" said Jay Hagan, President and Chief Executive Officer
of Regulus Therapeutics. "There is a significant unmet need for
patients with this disease and we look forward to the continued
advancement of this trial."
The Company is conducting a Phase 1 SAD study in healthy
volunteers to assess safety, tolerability, and pharmacokinetics of
RGLS8429. Following the SAD study, the Company plans to initiate a
Phase 1b multiple ascending dose (MAD) study to assess
safety, tolerability, and pharmacokinetics of RGLS8429 in adult
patients with ADPKD, 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.
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 clinical
trials involving RGLS8429 for the treatment of autosomal dominant
polycystic kidney disease, including the planned initiation of
clinical trials involving ADPKD patients 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.