Company remains on track to initiate Phase 1 clinical study
in second quarter 2022
DIEGO, May 11, 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
that the U.S. Food and Drug Administration (FDA) has accepted its
Investigational New Drug (IND) Application for RGLS8429 for the
treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD),
enabling the Company to initiate its planned Phase 1 clinical study
of RGSL8429 in healthy volunteers.
"With FDA's acceptance of our IND and the Phase 1 trial
preparations well underway, we look forward to advancing this
program which ultimately may provide a transformative treatment
option for patients with ADPKD," said Jay Hagan, President and
Chief Executive Officer of Regulus Therapeutics. "We are on track
to initiate the study in the second quarter and expect data from
the healthy volunteer study and initiation of dosing in patients
with ADPKD in the second half of this year."
The Company will conduct a Phase 1 single-ascending dose (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 in adult patients with ADPKD 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.
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
140,000 individuals are diagnosed with the disease in the United States alone, with an estimated
global prevalence of 4 to 7 million.
RGLS8429 is a novel, next generation oligonucleotide 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 and demonstrated a superior
pharmacologic profile compared to Regulus' first-generation
compound in preclinical studies.
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.
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 planned
initiation of clinical trials involving RGLS8429 for the treatment
of autosomal dominant polycystic kidney disease. 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.