- TRYNGOLZA shown to significantly reduce triglycerides and
substantially reduce acute pancreatitis events in adults with FCS;
a rare, highly debilitating and life-threatening disease
- Indicated for adults with FCS regardless of genetically
or clinically confirmed diagnosis
- TRYNGOLZA is the first of four independent launches planned
over the next three years, pending approvals
- Ionis to host webcast today at 6:45pm
ET
CARLSBAD, Calif., Dec. 19,
2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) announced today that the U.S. Food and Drug
Administration (FDA) has approved TRYNGOLZA™ (olezarsen) as an
adjunct to diet to reduce triglycerides in adults with familial
chylomicronemia syndrome (FCS), a rare, genetic form of severe
hypertriglyceridemia (sHTG) that can lead to potentially
life-threatening acute pancreatitis (AP). TRYNGOLZA is the
first-ever FDA-approved treatment that significantly and
substantially reduces triglyceride levels in adults with FCS
and provides clinically meaningful reduction in AP events when used
with an appropriate diet (≤20 grams of fat per day). TRYNGOLZA is
self-administered via an auto-injector once monthly.
Experience the full interactive Multichannel News Release here:
https://www.multivu.com/ionis-pharmaceuticals/9295551-en-tryngolza-olezarsen-fda-approval
"Today's FDA approval of TRYNGOLZA heralds the arrival of the
first-ever FCS treatment in the U.S. – a transformational moment
for patients and their families. For the first time, adults with
FCS can now access a treatment that substantially reduces
triglycerides and the risk of debilitating and potentially
life-threatening acute pancreatitis," said Brett P. Monia, Ph.D., chief executive officer,
Ionis. "We are proud of our long-standing partnership with the FCS
community and are grateful to the patients, families and
investigators who participated in our clinical studies, enabling
Ionis to make this new treatment a reality. The FDA approval of
TRYNGOLZA is also a pivotal moment for Ionis, representing our
evolution into a fully
integrated commercial-stage biotechnology company – a
goal we set out to achieve five years ago. With our rich pipeline
of potentially life-changing medicines, we expect TRYNGOLZA to
be the first in a steady cadence of innovative medicines we will
deliver independently to people living with serious diseases."
The FDA approval was based on positive data from the global,
multicenter, randomized, placebo-controlled, double-blind Phase 3
Balance clinical trial in adult patients with genetically
identified FCS and fasting triglyceride levels ≥880 mg/dL. In the
Balance study, TRYNGOLZA 80 mg demonstrated a statistically
significant placebo-adjusted mean reduction in triglyceride levels
of 42.5% from baseline to six months (p=0.0084). Reductions
from baseline to 12 months were further improved, with TRYNGOLZA
achieving a placebo-adjusted 57% mean reduction in
triglycerides. TRYNGOLZA also demonstrated a substantial,
clinically meaningful reduction in AP events over 12 months; one
patient (5%) experienced one episode of AP in the TRYNGOLZA group
compared with seven patients (30%) who experienced 11 total
episodes of AP in the placebo group.
TRYNGOLZA demonstrated a favorable safety profile. The most
common adverse reactions (incidence >5% of TRYNGOLZA-treated
patients and at a >3% higher frequency than placebo) were
injection site reactions (19% and 9%, respectively), decreased
platelet count (12% and 4%, respectively) and arthralgia (9% and
0%, respectively).
Results from the Phase 3 Balance study were previously published
in The New England Journal of Medicine (NEJM).
"With no treatment options previously available, we were limited
to relying only on extremely strict diet and lifestyle changes as
the sole preventative treatment option," said Alan Brown, M.D., FNLA, FACC, FAHA, clinical
professor of medicine, Rosalind Franklin
University of Medicine and Science; Balance trial
investigator. "The FDA approval of TRYNGOLZA is an important moment
for people living with FCS, their families and physicians who now,
for the first time, have a treatment that significantly lowers
triglycerides and decreases the risk of potentially
life-threatening acute pancreatitis events, as an adjunct to a
low-fat diet. I am excited to have a medicine I can prescribe to my
patients that has been shown to change the course of their
disease."
FCS is a rare, genetic, potentially life-threatening form of
sHTG that prevents the body from breaking down fats and severely
impairs the body's ability to remove triglycerides from the
bloodstream due to an impaired function of the enzyme lipoprotein
lipase (LPL). While healthy levels for adults are below 150 mg/dL,
people with FCS often have triglyceride levels of more than 880
mg/dL and often have a history of pancreatitis. Those living with
FCS have a high risk of potentially fatal AP, which is a painful
inflammation of the pancreas, and chronic health issues such as
fatigue and severe, recurrent abdominal pain. People living with
FCS can also experience psychological and financial stress, which
can significantly impact their quality of life. In the U.S., FCS is
estimated to impact up to approximately 3,000 people, the vast
majority of whom remain undiagnosed.
"As a rare and difficult to diagnose disease, FCS has a profound
impact on the lives of patients and families. Many people living
with FCS have experienced severe pain their whole lives – sometimes
so intense they require lengthy hospitalization stays – and
struggle through life with daily fatigue, nausea, brain fog and
stomach pain," said Lindsey Sutton
Bryan, co-founder and co-president, FCS Foundation. "Until
now, our treatment options have been limited, relying on diet alone
to try to manage triglyceride levels and keep acute pancreatitis
attacks at bay. For the first time, adults with FCS have seen their
hope for a treatment become a reality."
TRYNGOLZA will be available in the U.S. before year end.
Ionis is committed to helping people access the medicines they
are prescribed and will offer a suite of services designed to meet
the unique needs of the FCS community through Ionis Every
Step™. As part of Ionis Every Step,
patients and healthcare providers will have access to services
throughout the treatment journey provided by dedicated Patient
Education Managers and Ionis Every Step Case Managers, including
insurance and affordability support, as well as services and
resources, such as disease and nutrition education. Visit
TRYNGOLZA.com for more information.
TRYNGOLZA was reviewed by the FDA under Priority Review and had
previously been granted Fast Track designation for the treatment of
FCS, Orphan Drug designation and Breakthrough Therapy designation.
Olezarsen is undergoing review in the European Union and regulatory
filings in other countries are planned. Olezarsen is currently
being evaluated in three Phase 3 clinical trials – CORE, CORE2 and
ESSENCE – for the treatment of sHTG. Olezarsen has not been
reviewed or approved for the treatment of sHTG by regulatory
authorities.
Webcast
Ionis will hold a webcast today at 6:45pm
ET to discuss the FDA approval. Interested parties may access
the webcast here. A webcast replay will be available for a limited
time.
About TRYNGOLZA™ (olezarsen)
TRYNGOLZA™ (olezarsen)
was approved by the U.S. Food and Drug Administration as an adjunct
to diet to reduce triglycerides in adults with familial
chylomicronemia syndrome (FCS). TRYNGOLZA is an RNA-targeted
medicine designed to lower the body's production of apoC-III, a
protein produced in the liver that is a key regulator of
triglyceride metabolism. It is the only treatment currently
indicated in the U.S. for FCS, a potentially life-threatening
disease. For more information about TRYNGOLZA, visit
TRYNGOLZA.com.
IMPORTANT SAFETY
INFORMATION
CONTRAINDICATIONS
TRYNGOLZA is
contraindicated in patients with a history of serious
hypersensitivity to TRYNGOLZA or any of the excipients in
TRYNGOLZA. Hypersensitivity reactions requiring medical
treatment have occurred.
WARNINGS AND PRECAUTIONS
Hypersensitivity
Reactions
Hypersensitivity reactions (including symptoms of
bronchospasm, diffuse erythema, facial swelling, urticaria, chills
and myalgias) have been reported in patients treated with
TRYNGOLZA. Advise patients on the signs and symptoms of
hypersensitivity reactions and instruct patients to promptly seek
medical attention and discontinue use of TRYNGOLZA if
hypersensitivity reactions occur.
ADVERSE REACTIONS
The most common adverse reactions
(incidence >5% of TRYNGOLZA-treated patients and >3% higher
frequency than placebo) were injection site reactions, decreased
platelet count and arthralgia.
Please see full Prescribing Information for
TRYNGOLZA.
About Familial Chylomicronemia Syndrome (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 up to approximately 3,000 people 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.
About the Balance Study
Balance is a global,
multicenter, randomized, double-blind, placebo-controlled Phase 3
study evaluating the efficacy and safety of olezarsen in patients
with FCS at six and 12 months. The primary endpoint was the percent
change from baseline in fasting triglyceride levels at six months
compared to placebo. Secondary endpoints included percent changes
in triglyceride levels at 12 months, percent changes in other lipid
parameters and adjudicated acute pancreatitis event rates over the
treatment period. Following treatment and the end-of-trial
assessments, patients were eligible to enter an open-label
extension study to continue receiving olezarsen once every four
weeks.
About Ionis Pharmaceuticals, Inc.
For three decades,
Ionis has invented medicines that bring better futures to people
with serious diseases. Ionis has discovered and developed six
marketed medicines for serious diseases, including breakthrough
medicines for neurologic and cardiovascular diseases. Ionis has 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), LinkedIn and Instagram.
Ionis Forward-Looking Statements
This press release
includes forward-looking statements regarding Ionis' business and
the therapeutic and commercial potential of TRYNGOLZA, Ionis'
technologies and other 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 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® and TRYNGOLZA™ are trademarks
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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SOURCE Ionis Pharmaceuticals, Inc.