CARLSBAD, Calif., Nov. 2, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted
therapies, announced today the initiation of CORE, the second Phase
3 clinical study of olezarsen, formerly known as
IONIS-APOCIII-LRx. The CORE study is evaluating
olezarsen in people with severe hypertriglyceridemia (triglyceride
levels ≥ 500 mg/dL). Severe hypertriglyceridemia is a
life-threatening condition associated with high levels of
apoC-III and chylomicronemia, which lead to higher rates of
acute pancreatitis, higher risk of cardiovascular disease, and has
a high morbidity rate.
"Severe hypertriglyceridemia is a common condition that affects
millions of people around the world, including more than 3 million
in the US," said Sotirios "Sam" Tsimikas, M.D., senior vice
president, clinical development and cardiovascular franchise leader
at Ionis. "Initiating the CORE study is a major step in realizing
the full potential of olezarsen to treat the range of conditions
related to elevated triglycerides associated with high levels of
the apoC-llI protein."
Olezarsen is an investigational antisense medicine that uses
Ionis' LIgand-Conjugated Antisense, or LICA,
technology. It is designed to inhibit the production of apoC-III
for patients who are at risk for cardiometabolic disease and acute
pancreatitis due to elevated triglyceride levels. ApoC-III is a
protein produced in the liver that regulates triglyceride
metabolism in the blood.
CORE is a global, double-blind, randomized, placebo-controlled,
registrational, Phase 3 study in patients with severe
hypertriglyceridemia. It is designed to compare olezarsen to
placebo in patients with triglyceride levels equal to or greater
than 500 mg/dL who are on the current available standard of care
therapies for elevated triglycerides. The primary endpoint of the
study is the percent change in fasting triglycerides from baseline
at month 6. Secondary endpoints include: percent change from
baseline in triglycerides at month 12; proportion of patients who
achieve fasting triglycerides less than 500 mg/dL, 880mg/dL and/or
1000 mg/dL and percent change from baseline in other atherogenic
lipids at months 6 and 12; and adjudicated acute pancreatitis event
In a Phase 2 clinical study, results showed that olezarsen met
its primary and key secondary endpoints with significant reductions
in triglyceride and apoC-III levels, and a favorable safety and
tolerability profile in the treatment of patients with
hypertriglyceridemia (≥200 to ≤500 mg/dL) who have established
cardiovascular disease or are at risk for cardiovascular disease.
In addition, 91% of subjects achieved a normal triglyceride level
of <150 mg/dL.
The broad olezarsen development program aims to enhance the full
profile of olezarsen and enroll more than 1500 patients. CORE is
one of several clinical studies aimed at evaluating olezarsen's
potential to treat diseases caused by high triglycerides. BALANCE,
a Phase 3 clinical study evaluating olezarsen in people with
familial chylomicronemia syndrome or FCS, is on track for a data
readout in 2023.
For more information on the CORE clinical study (NCT05079919)
please visit www.clinicaltrials.gov.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been the leader in
RNA-targeted therapy, pioneering new markets and changing standards
of care with its novel antisense technology. Ionis currently has
three marketed medicines and a premier late-stage pipeline
highlighted by industry-leading neurological and cardiometabolic
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision of
becoming one of the most successful biotechnology companies.
To learn more about Ionis, visit www.ionispharma.com and
follow us on twitter @ionispharma.
Ionis' Forward-looking Statement
This press release includes forward-looking statements regarding
Ionis' business, and the therapeutic and commercial potential of
olezarsen and products in development. Any statement describing
Ionis' goals, expectations, financial or other projections,
intentions or beliefs is a forward-looking statement and should be
considered an at-risk statement. Such statements are subject to
certain risks and uncertainties, including those related to the
impact COVID-19 could have on our business, and including but not
limited to those related to our commercial products and the
medicines in our pipeline, and particularly those inherent in the
process of discovering, developing and commercializing medicines
that are safe and effective for use as human therapeutics, and in
the endeavor of building a business around such medicines. Ionis'
forward-looking statements also involve assumptions that, if they
never materialize or prove correct, could cause its results to
differ materially from those expressed or implied by such
Although Ionis' forward-looking statements reflect the good
faith judgment of its management, these statements are based only
on facts and factors currently known by Ionis. As a result, you are
cautioned not to rely on these forward-looking statements. These
and other risks concerning Ionis' programs are described in
additional detail in Ionis' annual report on Form 10-K for the year
ended December 31, 2020, and the most
recent Form 10-Q quarterly filing, which are on file with the SEC.
Copies of these and other documents are available from the
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SOURCE Ionis Pharmaceuticals, Inc.