Ionis Provides Update on Development Program Evaluating PCSK9 Antisense Drug for the Treatment of Hypercholesterolemia

Ionis Provides Update on Development Program Evaluating PCSK9 Antisense Drug for the Treatment of Hypercholesterolemia

  • ION449 (AZD8233) met the primary endpoint in phase 2b SOLANO study for patients with hypercholesterolemia

  • ION449 will not proceed to Phase 3 development based on predefined criteria

CARLSBAD, Calif., September 23, 2022 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that key results from the 2b SOLANO A study in patients with hypercholesterolemia demonstrated that 60 mg of ION449 (AZD8233) given monthly resulted in a statistically significant reduction of 62.3% (p<0.001) in low-density lipoprotein cholesterol (LDL- C) after 28 weeks compared to placebo, thus meeting the primary endpoints of the study. effectiveness endpoint. ION449 was generally safe and well tolerated in this study. However, these results did not meet the pre-specified efficacy criteria and AstraZeneca decided not to advance ION449 (AZD8233) into Phase 3 development for hypercholesterolemia. AstraZeneca continues to analyze the results of the SOLANO study to determine next steps for the program.

(PRNewsfoto/Ionis Pharmaceuticals, Inc.)

“While the reductions in LDL-C observed in patients with high-risk hypercholesterolemia on maximal statin therapy were both statistically significant and robust, these results did not meet AstraZeneca’s target product profile criteria for invest in a large phase 3 development program,” said Eugene Schneider, MD, Executive Vice President, Director of Clinical Development at Ionis. “AstraZeneca continues to be a valued collaborator and we look forward to working with them to advance several important programs.”


SOLANO (NCT04964557) was a randomized, double-blind, placebo-controlled parallel phase 2b study conducted on 411 participants with LDL-C hyperlipidaemia ≥ 70 mg/dL and < 190 mg/dL on a maximum 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.

About ION449

ION449 (AZD8233) administered once a month subcutaneously, is an investigational drug that uses advanced Ionis technologies LIthinking-VSconjugate Antisense technology platform (LICA). 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 people with lifelong LDL-C reduction due to reduced PCSK9 function have a significantly reduced risk of cardiovascular disease. Pharmacological inhibition of PCSK9 significantly reduces LDL-C. ION449 is designed to reduce hepatic production of PCSK9 and lower plasma LDL-C levels, thereby reducing the risk of cardiovascular disease. AstraZeneca licensed ION449 from Ionis in 2015.

About Hypercholesterolemia

Hypercholesterolemia, or high levels of LDL-C 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% of them not achieving their treatment goals despite taking a statin at high intensity. 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 the leader in RNA-targeted therapy, opening new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed drugs and a leading 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 to become a leading, fully integrated biotechnology company.

To learn more about Ionis, visit and follow us on Twitter @ionispharma.

Ionis Forward-Looking Statements

This press release contains 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’ objectives, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. These 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 related to our commercial products and pharmaceuticals. our pipeline, and in particular those inherent in the process of discovering, developing and commercializing safe and effective drugs for human therapeutic use, and in the effort to build a business around these drugs. Ionis’ forward-looking statements also involve assumptions which, if they never materialize or prove to be incorrect, 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, such statements are based solely on facts and factors currently known to Ionis. Accordingly, you are cautioned not to rely on these forward-looking statements. These and other risks relating to Ionis’ programs are further described in Ionis’ Annual Report on Form 10-K for the fiscal year ended. December 31, 2021, and the most recent quarterly filing of Form 10-Q, which are filed with the Securities and Exchange Commission. Copies of these and other documents are available from the Company.

In this press release, unless the context otherwise requires, “Ionis”, “Company”, “we”, “us” and “our” refer to Ionis Pharmaceuticals and its subsidiaries.

Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc.



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SOURCEIonis Pharmaceuticals, Inc.

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