Blinded safety data observed so far are
encouraging
SAN
DIEGO, May 16, 2023 /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,
after review of all available safety data, that it will advance to
the second cohort of patients in the Phase 1b MAD study of RGSL8429 for the treatment of
ADPKD.
"We are pleased to see our Phase 1b MAD trial continue to advance with the
initiation of the second cohort of patients," said Jay Hagan, CEO of Regulus Therapeutics. "This is
another important step in our quest to develop a novel treatment
for ADPKD. We look forward to topline data from the first
cohort around the end of the third quarter of this year."
The Phase 1b MAD study is a
double-blind, placebo-controlled trial evaluating the safety,
tolerability, pharmacokinetics and pharmacodynamics (PK/PD) of
RGLS8429 in adult patients with ADPKD. The study will evaluate
RGLS8429 treatment across three different dose levels, including
measuring changes in polycystins, height-adjusted total kidney
volume (htTKV), cyst architecture, and overall kidney function. The
first cohort is being dosed at 1 mg/kg of RGLS8429 or placebo every
other week for three months. No issues were identified in cohort 1
based on the review of all available blinded safety data clearing
the way to advancement to cohort 2. The safety review incorporated
available data from all 12 patients in the first cohort including
several who had completed the dosing schedule. Patients
in the second cohort will receive 2 mg/kg of RGLS8429 or placebo
every other week for three months. The Company also recently
completed the 27-week chronic toxicity study of RGLS8429 in mice.
No RGLS8429-related toxicity, including CNS effects, was observed
at any dose level up to the top dose of 300 mg/kg administered
every other week.
More information about the MAD clinical trial is available at
clinicaltrials.gov (NCT05521191).
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, RGLS4326. Regulus
announced completion of the Phase 1 SAD study in September
2022. The Phase 1 SAD study demonstrated that RGLS8429 has a
favorable safety and PK profile. RGLS8429 was well-tolerated
with no serious adverse events reported and plasma exposure was
approximately linear across the four doses tested and is similar to
the PK data from the first-generation compound. In April, Regulus
announced completion of enrollment for the first cohort of patients
in the Phase 1b MAD study and with
the recent review of all available safety data, is advancing to the
second cohort where patients will receive 2 mg/kg of RGLS8429 or
placebo every other week for three months.
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 expected timing for initiating clinical
studies, the expected timing for reporting topline data, the safety
of RGLS8429 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 filed annual 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.