CARLSBAD, Calif., Aug. 2, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (NASDAQ: IONS) today announced that
pelacarsen, formerly known as AKCEA-APO(a)-LRx, and
licensed by Novartis (NYSE: NVS) for exclusive worldwide
development and commercialization, continues to
advance in the clinic as the potential first-in-class treatment for
lipoprotein(a) or Lp(a)-driven cardiovascular disease. The Novartis
pivotal Phase 3 cardiovascular outcomes study of pelacarsen, Lp(a)
HORIZON (NCT04023552), has reached 50% enrollment with a
target goal of 7,680 trial participants.
Pelacarsen is an investigational antisense medicine that uses
Ionis' proprietary Ligand Conjugated Antisense
(LICA) technology platform. It is designed to inhibit the
production of apolipoprotein(a) in the liver to target elevated
Lp(a) levels, an independent genetic risk factor for cardiovascular
diseases (CVD), that are determined at birth and cannot be
controlled with diet or exercise. High Lp(a) levels are associated
with significant risk of cardiovascular disease, including heart
attacks and strokes. There are no approved pharmacological
therapies to effectively lower Lp(a).
"We are pleased by Novartis progress in advancing the Lp(a)
HORIZON study and enrolling nearly 4,000 study participants around
the world. Pelacarsen represents a potential first-in-class
treatment to address a significant unmet need with the potential to
become the new standard of care for Lp(a)-driven cardiovascular
disease," said Sotirios "Sam" Tsimikas, M.D., senior vice
president, clinical development and cardiovascular franchise leader
at Ionis, who specializes in Lp(a). "Ionis' vision is that
pelacarsen emerges as an effective therapy to improve
cardiovascular outcomes through normalizing Lp(a) levels, as there
are an estimated eight million people globally living with elevated
Lp(a) and cardiovascular disease."
Data from a Phase 2 study published in the New England
Journal of Medicine showed pelacarsen provided potent
dose-dependent reductions of Lp(a) compared to placebo, with a
favorable safety and tolerability profile in patients who had
elevated Lp(a) levels and established CVD. These data support the
potential of antisense-mediated reduction of Lp(a) with
pelacarsen.
Ionis earned a $25 million
milestone payment from Novartis for achieving 50% enrollment in the
pivotal Phase 3 study. In 2017, Novartis entered a collaboration
agreement with Ionis for pelacarsen. In February 2019, Novartis exercised an option to
license the rights to develop and commercialize pelacarsen for
targeted cardiovascular therapy for $150
million. Under the terms of the agreement, Novartis is
exclusively responsible for worldwide development and
commercialization of pelacarsen and Ionis is eligible to receive up
to $675 million in regulatory and
sales milestones. Ionis is also eligible to receive tiered
royalties in the mid-teens to low 20% range on net sales of
pelacarsen.
Additional information about Lp(a) HORIZON may be found at
www.ClinicalTrials.gov (NCT04023552).
About Lp(a) HORIZON
Lp(a) HORIZON is a pivotal, global multicenter, double-blind,
placebo-controlled pivotal Phase 3 study conducted by
Novartis. The trial is designed to support an indication for the
reduction of cardiovascular risk in patients with established CVD
and elevated Lp(a) with 80 mg of pelacarsen administered monthly
via subcutaneous administration. The study has a targeted
enrollment of 7,680 participants. The estimated study completion
date is in 2024.
The primary objectives of the trial are to demonstrate
superiority compared to placebo in reducing the risk of
expanded MACE (major adverse cardiac events such as cardiovascular
death, non-fatal myocardial infarction, non-fatal stroke and urgent
coronary re-vascularization requiring hospitalization) in the
overall study population with established CVD and Lp(a) ≥ 70 mg/dL,
and in the population with Lp(a) ≥ 90 mg/dL.
About pelacarsen
Pelacarsen, licensed by Novartis for exclusive worldwide
development and commercialization, is an investigational antisense
medicine designed to reduce apolipoprotein(a) production in the
liver to offer a direct approach for reducing circulating
lipoprotein(a), or Lp(a), an atherogenic, pro-inflammatory and
thrombogenic lipoprotein that induces additional cardiovascular
risk independent of LDL-cholesterol, in patients already treated
with LDL-C-lowering therapies (such as statins or PCSK9
inhibitors). Elevated Lp(a) is recognized as an independent,
genetic cause of coronary artery disease, heart attack, stroke and
peripheral arterial disease. Currently, there is no effective drug
therapy to specifically and robustly lower elevated levels of
Lp(a). Lp(a) levels are determined at birth and, therefore,
lifestyle modification, including diet and exercise, does not
impact Lp(a) levels. Even patients with LDL-C lowered to target
levels (<70 mg/dL) remain at high-risk of cardiovascular events
if they have high levels of Lp(a).
Pelacarsen is being developed for patients who are at
significant risk of CVD because of their elevated Lp(a).
About Lp(a)
Lp(a) is a lipoprotein particle assembled in the liver that
consists of an LDL-C-like particle and apolipoprotein(a). Lp(a) is
considered a key driver for cardiovascular disease due to its
association with an increased risk of coronary heart disease. There
is evidence that elevated Lp(a) levels may contribute directly to
coronary artery disease, heart attacks, strokes and peripheral
artery disease. Lp(a) levels in the blood can vary greatly between
individuals primarily due to genetic variations and do not
correlate with LDL-C levels. Because elevated Lp(a) is a
genetically determined condition that is not responsive to
lifestyle changes, patients are unable to adequately control their
Lp(a) levels through improved diet or increased physical activity.
Moreover, current therapies are not able to normalize Lp(a) levels
in patients who have high Lp(a). Although Lp(a) can be measured by
a variety of reliable and readily available simple tests, the lack
of drugs to effectively lower Lp(a) has made treating patients with
Lp(a)-driven cardiovascular disease difficult.
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
Ionis' technologies, pelacarsen 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 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 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. 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
Company.
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SOURCE Ionis Pharmaceuticals, Inc.