SAN
DIEGO, May 6, 2024 /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, the Company initiated
screening in the of the fourth cohort of patients in the Phase
1b MAD study of RGSL8429 for the
treatment of ADPKD. Patients in the fourth cohort will receive an
open label fixed dose of 300 mg of RGLS8429 every other week for
three months. The study protocol has been amended to include up to
30 subjects in the fourth cohort in order to further examine
potential impact on total kidney volume in patients with APDKD.
"After sharing positive topline data from cohort 2 that further
validates RGLS8429's potential efficacy in ADPKD, we are moving to
the fourth and final cohort of the Phase 1b MAD study," said Jay
Hagan, CEO of Regulus Therapeutics. "This open label, fixed
dose cohort will provide essential insight and data as we begin to
think about study design for our potentially pivotal Phase 2 trial.
Our team is looking forward to sharing further updates as they
become available, including topline data from the third cohort in
the middle 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 weight-based dose levels,
including measuring changes in urinary polycystins PC1 and PC2,
height-adjusted total kidney volume (htTKV), cyst architecture, and
overall kidney function. PC1 and PC2 are the protein products of
the PKD1 and PKD2 genes respectively and have been shown to
inversely correlate with disease severity. The Company amended the
protocol to include a fourth cohort of up to 30 subjects who will
receive a fixed dose of 300 mg of RGLS8429 administered every other
week for three months.
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 clear improvements in kidney function, size, and
other measures of disease severity in preclinical models. 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. In the Phase 1b MAD study
Regulus announced both topline data from the first cohort of
patients in September 2023 and from
the second cohort of patients in March
2024. Regulus announced completion of enrollment in the
third cohort in January 2024 with
patients receiving 3 mg/kg of RGLS8429 or placebo every other week
for three months. Topline data from the third cohort is anticipated
in mid-2024.
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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SOURCE Regulus Therapeutics Inc.