– First presentation of pivotal results
showing significant reduction in triglycerides and substantial
reduction in acute pancreatitis events in patients with rare,
life-threatening disease, for which there are no approved treatment
options in U.S. –
CARLSBAD, Calif., March 25,
2024 /PRNewswire/ -- Ionis Pharmaceuticals,
Inc. (Nasdaq: IONS) announced today that it will present
positive Phase 3 Balance results in patients with familial
chylomicronemia syndrome (FCS) for the company's lead independent,
investigational medicine, olezarsen, at the 2024 American College
of Cardiology (ACC) Annual Meeting in Atlanta, Georgia.
The oral presentation will take place on April 7, 2024 at 10:08am
ET during the session on Prevention and Health Promotion and
the Year in Review. Ionis previously announced positive
topline results. As previously shared, monthly dosing of olezarsen
80 mg met the primary endpoint with a statistically significant
reduction in triglyceride levels at six months, demonstrated robust
reductions in apolipoprotein C-III (apoC-III) and marked reductions
in acute pancreatitis (AP) events versus placebo. Olezarsen also
demonstrated a favorable safety and tolerability profile.
In addition, a late-breaking abstract entitled "Efficacy and
Safety of Olezarsen in Patients with Hypertriglyceridemia and High
Cardiovascular Risk: Primary Results of the Bridge-TIMI 73a Trial"
will be presented on April 7, 2024 at
8:30am ET during the session on
Late-Breaking Clinical Trials II.
About the Balance Study
The global, multicenter, randomized, double-blind,
placebo-controlled Phase 3 Balance study (NCT04568434) enrolled 66
patients aged 18 and older with confirmed FCS. Patients in the
study received background therapies including statins, fibrates and
omega-3 fatty acids. Patients were randomized in a 1:1:1 ratio to
receive olezarsen 80 mg or 50 mg or placebo via subcutaneous
injection once every four weeks for 53 weeks. 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.
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). Olezarsen is designed to inhibit
the body's production of apoC-III, a protein produced in the liver
that regulates triglyceride metabolism in the blood.1,2
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. In addition to FCS, Ionis is
evaluating olezarsen for the treatment of severe
hypertriglyceridemia (sHTG) in Phase 3 clinical trials.
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).3 Because of limited
LPL production or function, people with FCS cannot effectively
break down chylomicrons, lipoprotein particles that are 90%
triglycerides.3,4 FCS is estimated to impact one to 13
people per million in the U.S.5,6,7 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.3,8,9 People living with FCS are
sometimes unable to work, adding to the burden of
disease.9
Currently, there are no U.S. FDA-approved therapies for the
treatment of FCS and standard triglyceride lowering therapies are
generally ineffective in patients with FCS.10,11 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.11,12
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 Ionispharma.com and follow us on X (Twitter)
and LinkedIn.
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.ionispharma.com.
Ionis Pharmaceuticals® is a registered trademark of Ionis
Pharmaceuticals, Inc.
1Alexander VJ, et al. Eur Heart J
2019;40(33):2785-2796.
2Tardif JC, et al. Eur Heart J
2022;43(14):1401-1412.
3Gaudet D, et al. N Engl J Med. 2014;371:2200-2206.
4Ginsberg HN, et al. Eur Heart
J. 2021;42:4791-4806.
5Pallazola VA, et al. Eur J Prev Cardiol
2020;27(19):2276-8.
6Warden BA, et al. J Clin Lipidol 2020;14(2):201-6.
7Tripathi M, et al. Endocr Pract 2021;27(1):71-6.
8Bashir B, et al. Metabolites. 2023;(5):621.
9Davidson M, et al. J Clin Lipidol.
2018;12(4):898-907.e2.
10Gouni-Berthold I. J Endocr Soc. 2019;4(2):bvz035.
11Paquette M, et al. Atherosclerosis.
2019;283:137-142.
12Williams L, et al. J Clin Lipidol. 2018;4:908-919.
View original content to download
multimedia:https://www.prnewswire.com/news-releases/positive-olezarsen-phase-3-data-in-familial-chylomicronemia-syndrome-to-be-presented-at-2024-american-college-of-cardiology-acc-annual-meeting-302098131.html
SOURCE Ionis Pharmaceuticals, Inc.