ION449 (AZD8233) met the primary endpoint in phase 2 B SOLANO Study for patients with hypercholesterolemia
ION449 will not advance to Phase 3 of development based on predetermined criteria
CARLSBAD, CALIFORNIA., September 23, 2022 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced top-line results from the phase 2 B SOLANO Study in patients with hypercholesterolemia showed that monthly administration of 60 mg ION449 (AZD8233) produced a statistically significant reduction in low-density lipoprotein-cholesterol (LDL-C) levels by 62.3% (p<0.001) at 28 weeks to placebo and thus met the primary value of the study efficacy endpoint. ION449 was generally safe and well tolerated in this study. However, these results did not meet pre-established efficacy criteria and AstraZeneca decided not to advance ION449 (AZD8233) to Phase 3 development for hypercholesterolemia. AstraZeneca continues to analyze the results of the SOLANO study to determine the next steps for the program.
“While the LDL-C reductions observed in high-risk hypercholesterolemic patients receiving maximal 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, MD, Executive Vice President, Chief Clinical Development Officer at Ionis. “AstraZeneca continue to be a valued partner and we look forward to working with them to advance several important programs.”
SOLANO (NCT04964557) was a randomized, parallel, double-blind, placebo-controlled phase 2 B Study of 411 participants with hyperlipidemia LDL-C ≥ 70 mg/dL and < 190 mg/dL on maximally tolerated statin and/or ezetimibe. The primary objectives of this study were to evaluate the efficacy, safety and tolerability of ION449 (AZD8233) compared to placebo.
ION449 (AZD8233), which is given subcutaneously once a month, is an investigational drug that uses Ionis’ advanced drug LIgandCconjugated Antisense (LICA) technology platform. It is designed to lower plasma levels of the proprotein convertase subtilisin/kexin type 9 (PCSK9). PCSK9 is significantly involved in the regulation of LDL cholesterol. Genetic studies have shown that individuals with lifetime lowered LDL cholesterol levels have a significantly reduced risk of cardiovascular disease due to reduced function of PCSK9. Pharmacological inhibition of PCSK9 significantly lowers LDL-C. ION449 is designed to reduce liver production of PCSK9 and lower plasma levels of LDL-C, thereby reducing the risk of cardiovascular disease. AstraZeneca licensed ION449 from Ionis in 2015.
Hypercholesterolaemia, or elevated levels of LDL-C in the blood, is a major 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, of whom approximately 50% fail to meet their treatment goals despite taking a high-intensity statin. It is estimated that one in six patients with LDL-C >100 mg/dL will experience a second major adverse cardiovascular event within approximately three years.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been at the forefront of RNA-targeted therapy, pioneering new markets and changing the standard of care with its novel antisense technology. Ionis currently has three marketed drugs and a world-class late-stage pipeline highlighted by industry-leading cardiovascular and neurology franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, fueling our vision to become 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 contains forward-looking statements about Ionis’ business and the therapeutic and commercial potential of Ionis’ technologies, ION449 and other products in development. Any statement that describes Ionis’ goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered a statement that involves risk. Such statements are subject to certain risks and uncertainties, including those related to the impact that COVID-19 may have on our business and including but not limited to those relating to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing, and commercializing drugs that are safe and effective for use as human therapeutics, and seeking to build a business around such drugs. Ionis’ forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause results to differ materially from those expressed or implied by such forward-looking statements.
Although Ionis’ forward-looking statements reflect management’s good faith judgment, these statements are based only on facts and factors currently known to Ionis. Therefore, you are cautioned not to rely on these forward-looking statements. These and other risks related to Ionis’ programs are described in more detail in Ionis’ Annual Report on Form 10-K for the current year December 31, 2021, and most recent quarterly filing of Form 10-Q on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company.
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SOURCE Ionis Pharmaceuticals, Inc.