SAN
DIEGO, Oct. 19, 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 third cohort of patients in the Phase 1b MAD study of RGSL8429 for the treatment of
ADPKD. Patients in the third cohort will receive 3 mg/kg of
RGLS8429 or placebo every other week for three months.
"I am very pleased with the positive safety data that we have
continued to see in this trial, and our advancement into the third
cohort is another important milestone in our mission to bring
RGLS8429 to patients living with ADPKD," said Jay Hagan, CEO of Regulus Therapeutics. "I look
forward to watching this cohort progress, as we prepare to share
top line data from the second cohort in the first quarter of
2024."
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
second cohort is being dosed at 2 mg/kg of RGLS8429 or placebo
every other week for three months. No safety issues were identified
in cohort 2 based on the review of all available blinded safety
data allowing advancement to cohort 3.
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 September 2023, Regulus announced top line
data from the first cohort of patients in the ongoing Phase
1b MAD study of RGLS8429 as well as
completion of enrollment in the second cohort. After review of all
available safety data, Regulus has advanced to the third cohort
where patients will receive 3 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, the expected timing
for initiating clinical studies, potentially achieving therapeutic
efficacy and clinical translation for ADPKD patients, the expected
timing for reporting topline data, the timing and future occurrence
of other preclinical and clinical activities and the expected
length of our cash runway. 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, the approach we
are taking to discover and develop drugs is novel and may never
lead to marketable products, preliminary or initial results may not
be indicative of future results, preclinical and clinical studies
may not be successful, risks related to regulatory review and
approval, risks related to our reliance on third-party
collaborators and other third parties, risks related to
intellectual property, 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 and our need for additional
capital. 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 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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