SAN
DIEGO, June 27, 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
the dosing of the first patient in the second cohort of the Phase
1b MAD study of RGSL8429 for the
treatment of ADPKD.
"Following the positive safety review of patients in the first
cohort, we were excited to initiate screening for the second cohort
last month," said Jay Hagan, CEO of
Regulus Therapeutics. "Now with dosing of the first patient in the
second cohort, our Phase 1b MAD trial
is progressing well. We are on track to deliver topline data from
the first cohort around the end of the third quarter of this year.
This is another important milestone in our effort to develop a
novel treatment for patients with ADPKD."
About the Phase 1b MAD Clinical
Trial
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.
Patients in the second cohort will receive 2 mg/kg of RGLS8429 or
placebo every other week for three months. The first cohort was
dosed at 1 mg/kg of RGLS8429 or placebo every other week for three
months. All available blinded safety data for the second cohort
will be reviewed prior to proceeding to the third cohort.
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, has advanced to the
second cohort where patients 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.
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, potentially achieving therapeutic efficacy and clinical
translation for ADPKD patients the expected reporting of
preliminary data and the timing thereof, 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, 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, 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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