Ionis Provides Update on Development Program Evaluating Antisense Drug PCSK9 for the Treatment of Hypercholesterolemia | DNA RNA and Cells
Ionis Provides Update on Development Program Evaluating PCSK9 Antisense Drug for the Treatment of Hypercholesterolemia
Category: DNA RNA and Cells
Posted on Friday, September 23, 2022 9:48 AM
- ION449 (AZD8233) met the primary endpoint of the Phase 2b SOLANO study in patients with hypercholesterolemia
- ION449 will not proceed to Phase 3 development based on predefined criteria
CARLSBAD, California, United States I September 23, 2022 I Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that topline results from the Phase 2b SOLANO study in patients with hypercholesterolemia demonstrated that 60 mg of ION449 (AZD8233) administered monthly resulted in to achieve a statistically significant reduction of 62.3% (p
“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 an extensive Phase 3 development program,” said Eugene Schneider, MD, 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 several important programs.”
SOLANO (NCT04964557) was a phase 2b randomized, parallel, double-blind, placebo-controlled study in 411 participants with hyperlipidemia LDL-C ≥ 70 mg/dL and
ION449 (AZD8233) administered once a month subcutaneously, is an investigational drug that uses advanced Ionis technologies LIghent-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.
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 www.ionispharma.com and follow us on Twitter @ionispharma.
THE SOURCE: Ionis Pharmaceuticals