–
Olezarsen PDUFA date set for December 19,
2024 for treatment of familial chylomicronemia syndrome
–
–
Phase 3 enrollment completed in CORE, CORE2 and ESSENCE
evaluating olezarsen for the treatment of severe
hypertriglyceridemia, with results expected in 2H 2025 –
CARLSBAD, Calif., June 25,
2024 /PRNewswire/ -- Ionis Pharmaceuticals,
Inc. (Nasdaq: IONS) today announced that the U.S. Food and
Drug Administration (FDA) has accepted for Priority Review the New
Drug Application (NDA) for olezarsen, an investigational
RNA-targeted medicine for the treatment of adults with familial
chylomicronemia syndrome (FCS). The FDA has set an action date of
December 19, 2024 and indicated they
are not currently planning an advisory committee meeting for
olezarsen.
"FCS is a debilitating, rare, genetic disease that causes
significant physical, emotional and financial burden with no
approved treatment options in the U.S.," said Brett Monia, Ph.D., chief executive officer of
Ionis. "The Priority Review underscores the urgent need people
living with FCS have for a medicine that may help lower
triglyceride levels and reduce incidence of life-threatening acute
pancreatitis events. We look forward to partnering closely with the
FDA during the review process as we work to bring this potentially
breakthrough medicine to patients before the end of 2024.
Separately, we are pleased with the progress of our now fully
enrolled Phase 3 program investigating olezarsen in the much more
common severe hypertriglyceridemia population."
Priority Review is a designation for medicines that have the
potential to provide significant improvements in the treatment,
prevention or diagnosis of a serious disease, with the expectation
of the FDA taking action within six months, compared to 10 months
under standard review. The application to the FDA was based on
positive results from Balance, a global, multicenter, randomized,
double-blind, placebo-controlled Phase 3 study. The study results
were presented at the 2024 American College of
Cardiology (ACC) Annual Meeting and published simultaneously
in The New England Journal of Medicine (NEJM).
The U.S. FDA granted olezarsen Fast Track designation for the
treatment of FCS in January
2023, as well as Orphan Drug
designation and Breakthrough Therapy designation in
February 2024. Ionis is planning
additional regulatory filings for the treatment of FCS in the
European Union this year.
In addition to FCS, Ionis is evaluating olezarsen for the
treatment of severe hypertriglyceridemia (sHTG) in three Phase 3
clinical trials – CORE, CORE2 and ESSENCE, all three of which
completed enrollment in the first half of 2024. CORE and CORE2
enrolled more than 1,000 sHTG patients with fasting triglyceride
levels ≥500 mg/dL; ESSENCE enrolled more than 1,400 high
triglyceride patients with fasting triglyceride levels ≥150 mg/dL
to <500 mg/dL who were diagnosed and/or at risk for
atherosclerotic cardiovascular disease as well as patients with
fasting triglycerides ≥500 mg/dL. sHTG is a common,
life-threatening condition that can result in severe health
complications, including potentially fatal acute pancreatitis (AP).
Data are expected in the second half of 2025.
About Olezarsen
Olezarsen is an RNA-targeted
investigational LIgand Conjugated Antisense
(LICA) medicine being evaluated for people at risk of disease due
to elevated triglyceride levels, including those with familial
chylomicronemia syndrome (FCS) and severe hypertriglyceridemia
(sHTG). Olezarsen is designed to lower the body's production of
apoC-III, a protein produced in the liver that regulates
triglyceride metabolism in the blood.
Olezarsen is an investigational medicine that has not been
reviewed or approved for the treatment of any disease by any
regulatory authority.
About FCS
FCS is a rare, genetic disease characterized by extremely
elevated triglyceride levels. It is caused by impaired function of
the enzyme lipoprotein lipase (LPL). Because of limited LPL
production or function, people with FCS cannot effectively break
down chylomicrons, lipoprotein particles that are 90%
triglycerides. FCS is estimated to impact one to 13 people per
million in the U.S. People living with FCS are at high risk of
acute pancreatitis (AP) in addition to other chronic health issues
such as fatigue and severe, recurrent abdominal pain. People living
with FCS are sometimes unable to work, adding to the burden of
disease.
Currently, there are no U.S. FDA-approved therapies for the
treatment of FCS and people with FCS are generally nonresponsive to
standard triglyceride lowering therapies. People living with this
condition currently rely solely on nutrition management through
extremely restrictive and difficult to manage diets to navigate the
health risks associated with FCS.
About sHTG
sHTG is a disease categorized by triglyceride levels of 500
mg/dL and above. It develops due to primary (genetic) and secondary
causes including diet and lifestyle, other medical conditions and
certain medications. More than three million people are currently
estimated to live with sHTG in the U.S. People living with
sHTG are at high risk of pancreatitis and damage to the pancreas,
as such, reducing the risk of pancreatitis is a critically
important reason to treat sHTG. People with sHTG are also at risk
of heart, brain and blood vessel damage.
There remains a need for additional therapeutic options for the
treatment of sHTG. Endocrinology and cardiovascular treatment
guidelines recommend treating triglyceride levels of ≥500 mg/dL to
reduce the risk of pancreatitis. Multiple treatment guidelines
recommend lifestyle interventions to reduce triglyceride levels in
people with sHTG and the addition of a triglyceride lowering
medicine such as a fibrate or omega-3 fatty acid if needed to
reduce fasting triglycerides to appropriate levels. However,
these interventions are not sufficient for all people with sHTG,
for whom there remains a significant need for new treatments that
may help further reduce triglyceride levels and the risk of
pancreatitis.
About Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines that bring
better futures to people with serious diseases. Ionis currently has
five marketed medicines and a leading pipeline in neurology,
cardiology, and other areas of high patient need. As the pioneer in
RNA-targeted medicines, Ionis continues to drive innovation in RNA
therapies in addition to advancing new approaches in gene editing.
A deep understanding of disease biology and industry-leading
technology propels our work, coupled with a passion and urgency to
deliver life-changing advances for patients. To learn more about
Ionis, visit Ionis.com and follow us on X (Twitter) and
LinkedIn.
Ionis Forward-looking
Statements
This press release includes forward-looking statements regarding
olezarsen, Ionis' business, and the therapeutic and commercial
potential of Ionis' commercial medicines, additional medicines in
development and technologies. 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 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 forward-looking statements.
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. Except as required by
law, we undertake no obligation to update any forward-looking
statements for any reason. 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 Dec. 31, 2023, and most recent Form 10-Q, which
are on file with the SEC. Copies of these and other documents are
available at www.Ionis.com.
Ionis Pharmaceuticals® is a registered trademark of Ionis
Pharmaceuticals, Inc.
Ionis Investor Contact:
D. Wade Walke, Ph.D.
info@ionis.com 760-603-2331
Ionis Media Contact:
Hayley Soffer
media@ionis.com 760-603-4679
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