SAN DIEGO, Dec. 1, 2021 /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 a
presentation at the first annual Rare & Genetic Kidney Disease
Drug Development Summit.
In the presentation, Regulus summarized its ongoing efforts to
therapeutically target microRNAs (miRs), small non-coding RNAs that
play an important role in the regulation of gene expression and are
known to be involved in the pathogenesis of numerous genetic kidney
diseases representing significant unmet medical needs. The Company
believes that targeting over-expression of miRs with antagonists
(anti-miRs) and normalizing the expression of miRs presents a
promising therapeutic strategy. Targeting of miR-21 and miR-17 is
currently being investigated for the treatment of Alport Syndrome
and Autosomal Dominant Polycystic Kidney Disease (ADPKD),
respectively.
ADPKD is caused by mutations of either Pkd1 (in 85% of cases) or
Pkd2 genes, where reduced function of their encoded proteins
polycystin-1 (PC1) or polycystin-2 (PC2) leads to formation and
proliferation of multiple fluid-filled renal cysts, and results in
loss of kidney function over time. The levels of urinary PC1
and PC2 are reduced in ADPKD patients and inversely correlate with
ADPKD disease severity, thereby serving as potential biomarkers for
disease severity and progression. PC1 and PC2 levels have been
shown to be mediated by direct repression of Pkd1 and Pkd2 genes by
miR-17, which is overexpressed in ADPKD. Today's presentation
summarized data from the first cohort of ADPKD patients in the
Phase 1b clinical trial of RGLS4326,
the Company's first-generation selective inhibitor of miR-17 for
the treatment of ADPKD. The presentation also summarized some of
the evidence for the role of miRs in the pathogenesis of other
genetic kidney diseases, including Focal Segmental
Glomerulosclerosis (FSGS) and IgA nephropathy (Berger's
disease).
In the first cohort, the mean increases in PC1 and PC2 at the
end of study treatment compared to baseline levels were 58%
(p=.0004) and 38% (p=.026), respectively. These data demonstrate
clinical evidence that inhibition of miR-17 by treatment with
RGLS4326 increased both PC1 and PC2. Along with other clinical data
presented earlier this year, these results provide clinical proof
of the mechanism for targeting miR-17 for the treatment of ADPKD.
The Company recently announced the strategic prioritization of its
second generation miR-17 inhibitor, RGLS8429, which is supported by
strong preclinical data and a superior pharmacologic profile to
that of RGLS4326. The Company is scheduled to have a pre-IND
meeting with the U.S. Food and Drug Administration (FDA) for
RGLS8429 in December and is on track for an IND submission and
initiation of clinical development in the second quarter of 2022,
subject to FDA clearance of the IND.
The details of the presentation are below:
Presentation Title: Exploring the Role of MicroRNA
in Genetic Kidney Disease Pathologies
Presenter: Denis Drygin, Ph.D., Chief Scientific
Officer, Regulus Therapeutics
Presentation Date and Time: Wednesday, December 1,
2021, 5:30 AM PDT
A copy of the presentation is
available at www.regulusrx.com/publications/.
About ADPKD
ADPKD, caused by the 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.
About RGLS8429
RGLS8429 is a novel, second 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, as well as a superior
pharmacologic profile have been demonstrated. Regulus has
nominated RGLS8429 as a clinical candidate for the treatment of
ADPKD.
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 sufficiency of the preclinical
data required to support clinical studies, the expected timing for
submitting an IND and initiating a Phase 1 clinical study, 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.
View original content to download
multimedia:https://www.prnewswire.com/news-releases/regulus-therapeutics-announces-presentation-at-the-first-annual-rare--genetic-kidney-disease-drug-development-summit-301434889.html
SOURCE Regulus Therapeutics Inc.