SAN DIEGO, Oct. 12, 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
the prioritization of its promising, next-generation candidate,
RGLS8429, for the treatment of Autosomal Dominant Polycystic Kidney
Disease (ADPKD). The Company recently completed the dosing
period of the in-life portion of the IND-enabling toxicity studies
for RGLS8429; anticipates submitting an Investigational New Drug
application (IND); and subject to U.S. Food and Drug Administration
(FDA) clearance, initiating a phase 1 study in the second quarter
of 2022.
Based on discussions with FDA and data from the second cohort of
patients in the Phase 1b trial of
first-generation RGLS4326 in ADPKD, the Company believes that a
strategic prioritization of RGLS8429 represents a more judicious
use of its resources, rather than continuing the development of
RGLS4326 based on likely limitations on dose and duration of
therapy.
"In light of our discussions with FDA and early analysis of data
from the second cohort of our Phase 1b trial in ADPKD, we have determined that
advancing our next-generation compound RGLS8429 is more compelling
than further development of RGLS4326," commented Jay Hagan, CEO of Regulus. "The extensive work
and investment we have made in RGLS4326 will directly inform the
advancement of RGLS8429, and we believe will make this transition
both expeditious and productive. This prioritization of RGLS8429 is
supported both by robust data in preclinical models, where we have
seen clear improvements in kidney function, size, and other
measures of disease severity, as well as the compound's superior
pharmacologic profile."
With the completion of the dosing phase of the IND-enabling
toxicity studies, the Company believes RGLS8429 has demonstrated a
superior profile, including absence of the off-target CNS effects
that were seen with RGLS4326 at the top doses tested in chronic
preclinical toxicology studies as well as equal potency to RGLS4326
for its molecular target (miR-17) in both in-vitro and in-vivo
efficacy studies. The Company expects to have a pre-IND meeting
with the FDA for RGLS8429 later this year and is on track for an
IND submission and, subject to FDA clearance of the IND, initiation
of a Phase 1 clinical study in the second quarter of 2022.
Leveraging the learnings from the first-generation compound,
coupled with the superior pharmacological profile of RGLS8429 in
animal models, the Company's Phase 1 plans include a single dose
escalation study in healthy volunteers to enable a multi-dose
escalation study in ADPKD patients around the dose levels where
robust clinical biomarker effects were demonstrated with RGLS4326.
The Company anticipates reporting top-line biomarker data in the
first cohort of RGLS8429 treated patients in early 2023.
Conference Call Information
The Company will host a
conference call and live audio webcast on Tuesday, October 12,
2021 at 5:00 p.m. Eastern Daylight Time. To access
the call, please dial (877) 257-8599 (domestic) or (970) 315-0459
(international) and refer to conference ID 7733988. To access
the telephone replay of the call, dial (855) 859-2056 (domestic) or
(404) 537-3406 (international), passcode ID 7733988. The
webcast and telephone replay will be archived on the Company's
website at www.regulusrx.com following the call.
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 press release 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.
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SOURCE Regulus Therapeutics Inc.