- Pelacarsen is a potentially first-in-class treatment
specifically targeting elevated lipoprotein(a) (Lp(a)), an
independent, inherited and causal risk factor for cardiovascular
disease
- There are currently no approved pharmacological
therapies to effectively lower Lp(a), which cannot be
effectively addressed by diet and other lifestyle changes
- Topline results expected 2025
CARLSBAD, Calif., July 20,
2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) today announced that Novartis has completed patient
enrollment of the pivotal Phase 3 Lp(a) HORIZON cardiovascular
outcomes study of pelacarsen, with 8,325 study participants. Lp(a)
HORIZON is evaluating the safety and efficacy of pelacarsen in
reducing cardiovascular events in patients with cardiovascular
disease and elevated levels of Lp(a). Topline data from the study
are expected in 2025. Novartis licensed pelacarsen from Ionis in
2019.
Pelacarsen, formerly IONIS-APO(a)-LRx, is an
investigational antisense medicine designed to inhibit the
production of apolipoprotein(a) in the liver to reduce elevated
lipoprotein(a) or Lp(a) levels, an independent, inherited and
causal risk factor for cardiovascular disease (CVD) and calcific
aortic valve stenosis. High Lp(a) levels, which are associated with
significant risk of coronary heart disease, cannot be reduced with
lifestyle modifications or with existing lipid-lowering therapies.
Pelacarsen uses Ionis'
Ligand-Conjugated Antisense (LICA)
technology platform.
"Completing enrollment of this pivotal outcomes study brings us
closer to the day when this potentially transformative treatment is
available to patients," said Sotirios "Sam" Tsimikas, M.D., senior
vice president, clinical development and cardiovascular franchise
leader at Ionis, who specializes in Lp(a). "There are no approved
pharmacological therapies to effectively lower Lp(a) for the more
than eight million patients living with cardiovascular disease and
elevated levels of Lp(a) worldwide. We are grateful to the
patients, investigators and site staff who are participating to
make the Phase 3 Lp(a) HORIZON study possible."
Data from a Phase 2 study showed pelacarsen reduced Lp(a) levels
below the recommended threshold of risk for CVD events (<50
mg/dL, <125 nmol/L) in 98% of participants with the dose being
used in the Lp(a) HORIZON study.
Additional information about Lp(a) HORIZON (NCT04023552) may be
found at ClinicalTrials.gov.
About Lp(a) HORIZON
Lp(a) HORIZON is a global, multicenter, double-blind,
placebo-controlled pivotal Phase 3 study conducted by
Novartis. The study 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 enrolled 8,325
participants. Topline results are expected in 2025.
The primary objectives of the study 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 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)
levels in the blood can vary greatly between individuals primarily
due to genetic variations and do not correlate with LDL-C 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). Elevated Lp(a) is a genetically determined
condition that is not responsive to lifestyle changes, therefore
patients are unable to adequately control their Lp(a) levels
through improved diet or increased physical activity. Elevated
Lp(a) is recognized as an independent, genetic cause of coronary
artery disease, heart attack, stroke, peripheral arterial disease
and aortic stenosis. Currently, there is no effective drug therapy
to specifically and robustly lower elevated levels of Lp(a).
About Pelacarsen
Pelacarsen, licensed by Novartis for exclusive worldwide
development, manufacturing 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).
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 cardiovascular and neurological
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision of
becoming a leading, fully integrated biotechnology company.
To learn more about Ionis visit www.ionispharma.com and follow
us on Twitter @ionispharma.
Ionis' Forward-looking
Statements
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 Dec. 31, 2021 and the most
recent Form 10-Q quarterly filing, which are on file with the
Securities and Exchange Commission. Copies of these and other
documents are available from the Company.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our," and "us" refers to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a trademark of Ionis
Pharmaceuticals, Inc.
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SOURCE Ionis Pharmaceuticals, Inc.