SAN
DIEGO, June 21, 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 granted orphan
drug designation (ODD) to RGLS8429 for the treatment of Autosomal
Dominant Polycystic Kidney Disease (ADPKD).
"Patients living with ADPKD currently have limited treatment
options and approximately half of patients develop end-stage renal
disease by age 60 requiring dialysis or transplantation," commented
Jay Hagan, CEO of Regulus
Therapeutics. "ADPKD is a disease of high unmet need affecting
nearly 160,000 Americans. We look forward to advancing
RGLS8429 through the clinic with the goal of improving future
treatment options for patients in need."
The FDA's Office of Orphan Products Development grants orphan
designation status to drugs and biologics that are intended for the
safe and effective treatment, diagnosis or prevention of rare
diseases, or conditions that affect fewer than 200,000 people in
the U.S. Orphan designation status is intended to facilitate drug
development for rare diseases and may provide several benefits to
drug developers, including financial incentives, to support
clinical development and the potential for up to seven years of
market exclusivity in the U.S. upon regulatory approval.
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 is currently conducting a Phase
1 single-ascending dose study in healthy volunteers to assess
safety, tolerability, and pharmacokinetics of RGLS8429.
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 potential outcome of clinical development.
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.