- Preliminary data are expected in the second
half of 2022
SAN
DIEGO, March 24, 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 initiation of studies evaluating a library of
oligonucleotides designed to block miR-155 for the potential to
treat Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig's disease) under a collaboration
agreement with the laboratories of Oleg
Butovsky, Ph.D., and Howard L.
Weiner, M.D., at Brigham and Women's Hospital (BWH). The
agreement, effective since September
2021, encompasses the investigation of the biologic effects
of miR-155 inhibitors in both in vitro and in vivo models of
ALS.
Dr. Butovsky is an Associate Professor of Neurology at
Harvard Medical School and a Senior
Scientist at BWH. Dr. Weiner is the Robert L. Kroc Professor
of Neurology at the Harvard Medical
School, Director and Founder of the Partners Multiple
Sclerosis Center and Co-Director of the Center for Neurologic
Diseases at BWH. The study plan focuses on the evaluation of
the Regulus-designed compounds that inhibit miR-155 activity in
microglia cell-based assays and in animal models of the disease, to
identify potential candidates to advance into further studies.
Results from these initial studies will be used to guide the design
of future experiments in additional animal models of the disease
and characterize their properties. Combined, these data will be
used in an effort to identify anti-miR-155 lead compounds
exhibiting robust pharmacologic profiles and enable their further
preclinical therapeutic evaluation and initial testing in subjects
with ALS.
"In addition to our focus on genetic kidney disease, Regulus has
a growing research interest in the opportunity to apply our
technology to significant unmet needs in CNS disease. We are
excited by the initiation of this research collaboration with Drs.
Butovsky and Weiner, who are leading ALS researchers at Brigham and
Women's Hospital, affiliated with Harvard
Medical School," commented Denis
Drygin, Ph.D., CSO of Regulus Therapeutics. "ALS is a
devastating, progressive neurodegenerative disease with a major
unmet medical need. We look forward to the preliminary data, which
are expected in the second half of 2022."
Prior research published by BWH demonstrates that miR-155 plays
an important role in the pathogenesis of ALS and supports this
target as a potential therapeutic approach to address this
devastating disease. Researchers at BWH identified a
disease-associated microglia signature in ALS. In animal models of
ALS, miR-155 was found upregulated in microglia and associated with
disease progression. Importantly, miR-155 was also elevated in the
spinal cord of patients with ALS. Blocking miR-155 in animal models
through either genetic deletion or with compounds that blocked
miR-155 ameliorated disease. Anti-miR-155 treatment suppressed
disease-associated cytotoxic microglia, and resulted in robust
improvement in clinically relevant disease endpoints, including
prolongation of survival.
"This collaboration with Regulus will build upon our body of
research in ALS," said Tracy
Batchelor, chairman of Neurology at BWH, "We are pleased to
work with a company that shares our mission to understand the
pathophysiology of ALS with the objective of developing novel
therapies to treat this devastating disease. "
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
preclinical programs, including the potential identification and
development of therapeutic candidates in ALS. 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 year-end and quarterly
report on Form 10-K. 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.