SAN
DIEGO, Oct. 8, 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
it has completed enrollment of 26 patients in the fourth cohort in
the Phase 1b MAD study of RGLS8429
for the treatment of ADPKD.
"The completion of enrollment of our final cohort marks an
important milestone as we near the end of our Phase 1b study. We have been pleased to see positive
results from each of the prior three cohorts, showing continued
evidence of a mechanistic dose response based on increase in
urinary polycystins (PC1 and PC2), across cohorts. Additionally,
exploratory imaging analysis has shown mean reductions in height
adjusted total kidney volume (htTKV), a key measurement which we
will be evaluating in the fourth cohort and in the potentially
pivotal Phase 3 trial," said Jay
Hagan, CEO of Regulus. "We remain on track for an End of
Phase 1 meeting with the FDA by the end of 2024, and we plan to
share topline data from a substantial number of patients in cohort
four in early 2025."
The Phase 1b MAD study is a double-blind,
placebo-controlled, weight-based dosing and an open-label fixed
dose trial evaluating the safety, tolerability, pharmacokinetics
and pharmacodynamics (PK/PD) of RGLS8429 in adult patients with
ADPKD. The study is evaluating RGLS8429 treatment across three
different weight-based dose levels (cohort 1-3) and one fixed dose
level (cohort 4), 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 and have
been shown to inversely correlate with disease severity. Patients
in the fourth cohort are receiving 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 topline data from the first cohort of patients in
September 2023, from the second
cohort of patients in March 2024 and
from the third cohort of patients in June
2024. Regulus completed enrollment in the fourth cohort of
patients in October 2024. Patients in
the fourth cohort are receiving a 300 mg fixed dose of RGLS8429
administered 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 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 and preclinical pipeline, the potential that
RGLS8429 may be eligible for an Accelerated Approval pathway,
potentially achieving therapeutic efficacy and clinical translation
for patients, the expected timing for reporting interim or
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, the risk that the
approach we are taking to discover and develop drugs is novel and
may never lead to marketable products, that preliminary or topline
results are based on a preliminary analysis of key efficacy and
safety data, and such data may change following a more
comprehensive review of the data related to the clinical trial and
may not be indicative of future results, the FDA has not designated
RGLS8429 for an Accelerated Approval pathway and such designation
may not lead to a faster development, regulatory review or approval
process and does not increase the likelihood that RGLS8429 will
receive marketing approval, the risk that 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, the risk that additional
toxicology data may be negative, and risks related to our ability
to successfully secure and deploy 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' quarterly report on Form 10-Q
available on the Company's website or at www.sec.gov. 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.