- ION449 (AZD8233) met primary endpoint in Phase 2b SOLANO study
for patients with hypercholesterolemia
- ION449 will not advance into Phase 3 development based on
CARLSBAD, Calif., Sept. 23,
2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) today announced that topline results from the Phase
2b SOLANO study in patients with
hypercholesterolemia demonstrated that 60mg of ION449 (AZD8233)
administered monthly achieved a statistically significant 62.3%
(p<0.001) reduction in low-density lipoprotein cholesterol
(LDL-C) levels after 28 weeks compared to placebo, meeting the
study's primary efficacy endpoint. ION449 was generally safe and
well tolerated in this study. However, these results did not
achieve pre-specified efficacy criteria and AstraZeneca has decided
not to advance ION449 (AZD8233) into Phase 3 development for
hypercholesterolemia. AstraZeneca is continuing to analyze the
results from the SOLANO study to
determine its next steps for the program.
"While the LDL-C reductions seen in high-risk
hypercholesterolemia patients on maximum statin therapy were both
statistically significant and robust, these results did not meet
AstraZeneca's target product profile criteria to invest in a broad
Phase 3 development program," said Eugene
Schneider, M.D., executive vice president, chief clinical
development officer at Ionis. "AstraZeneca continues to be a valued
collaborator and we look forward to working with them to advance
multiple important programs."
SOLANO (NCT04964557) was a
randomized parallel, double-blind, placebo-controlled Phase
2b study in 411 participants with
hyperlipidemia LDL-C ≥70 mg/dL and <190mg/dL on maximum
tolerated statin and/or ezetimibe. The primary objectives of this
study were to assess the efficacy, safety and tolerability of
ION449 (AZD8233) as compared with placebo.
ION449 (AZD8233) dosed once monthly via subcutaneous
administration, is an investigational medicine that uses Ionis'
advanced LIgand-Conjugated Antisense (LICA)
technology platform. It is designed to reduce plasma levels of
proprotein convertase subtilisin/kexin type 9 (PCSK9). PCSK9 is
integrally involved in the regulation of LDL-cholesterol. Genetic
studies have shown that individuals with life-long reduction of
LDL-C due to reduced function of PCSK9 have substantially reduced
risk of cardiovascular disease. Pharmacological inhibition of PCSK9
substantially lowers LDL-C. ION449 is designed to reduce the liver
production of PCSK9 and lower the plasma level of LDL-C and thus
reduce the risk of cardiovascular disease. AstraZeneca licensed
ION449 from Ionis in 2015.
Hypercholesterolemia, or elevated LDL-C levels in the blood, is
an important risk factor for cardiovascular disease, the leading
cause of death worldwide. There is a significant unmet medical need
for stronger LDL-C lowering therapies in secondary prevention
patients with approximately 50% who are not meeting their treatment
goals despite taking a high-intensity statin. It is estimated that
one in six patients with LDL-C >100mg/dL will suffer a second
major adverse cardiovascular event over approximately three
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, ION449 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,
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