Ionis provides update on development program evaluating PCSK9 antisense medicine for the treatment of hypercholesterolemia
- ION449 (AZD8233) met primary endpoint in Phase 2b
SOLANOstudy for patients with hypercholesterolemia
- ION449 will not advance into Phase 3 development based on pre-specified criteria
"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
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 years.
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
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