The upcoming presentations of clinical and non-clinical data from its cardiovascular pipeline, including several of its investigational antisense medicines for cardiovascular disease, at the American Heart Association (AHA) Scientific Sessions, to be held virtually Nov. 13-15, 2021.

New data from the Phase 1 multiple ascending dose study for ION449 (also known as AZD8233) will be featured in an oral presentation. ION449, an investigational antisense medicine that uses Ionis’ proprietary Ligand-Conjugated Antisense technology (LICA), is designed to reduce the production of PCSK9 and lower the plasma level of low-density lipoprotein-cholesterol (LDL-C) in patients who are at risk of cardiovascular disease due to high levels of LDL-C. 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. Data to be presented include results from a study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending subcutaneous doses of ION449 in patients with dyslipidemia. ION449 is being developed by AstraZeneca as part of a collaboration between Ionis and AstraZeneca.

Ionis will also present data on the effect of olezarsen, formerly known as IONIS-APOCIII-LRx, on a variety of lipoprotein fractions measured by Nuclear Magnetic Resonance (NMR) in patients with cardiovascular disease and hypertriglyceridemia. The apolipoprotein C-III (apoC-III) protein is an independent cardiovascular risk factor that regulates triglyceride metabolism in the blood. Olezarsen is designed to inhibit the production of apoC-III in patients who are at risk for cardiovascular disease due to elevated triglyceride levels. Olezarsen is an investigational antisense medicine that was also developed using Ionis’ advanced LICA technology.  Olezarsen is currently being evaluated in two phase 3 clinical studies as part of Ionis’ broad development program. The CORE study is evaluating olezarsen’s potential to treat diseases caused by severe hypertriglyceridemia. BALANCE is a study evaluating olezarsen in people with familial chylomicronemia syndrome or FCS.

In addition, Ionis will host a sponsored symposium, “Hidden in Plain Sight: ATTR-CM in Overlooked Patient Populations” to discuss the prevalence of cardiomyopathy in Blacks and African Americans while addressing biological and genetic differences.

The following is a schedule of the events and presentations of Ionis programs and collaborations.

Saturday, Nov. 138:00- 5:30 AM ET (on demand)
Scientific Session: Unraveling Dyslipidemias: Learning from Basic Science to Improve Treatment. Sotirios “Sam” Tsimikas, M.D., senior vice president and franchise leader of cardiovascular programs at Ionis, who specializes in lipoprotein(a), will present “Basic Science Evidence Based Approaches to Lp(a) Disorders.” Lp(a) is a lipoprotein particle assembled in the liver that consists of an LDL-C-like particle and apolipoprotein(a). Lp(a) is considered a key driver for cardiovascular disease due to its association with an increased risk of coronary heart disease. This educational session was planned by the Committee on Scientific Sessions Program and aims to cover the “best of 2020” cutting edge research that have advanced cardiovascular science and approaches to treatment.

Sunday, Nov.14, 7:30-9:00 p.m. ET
Symposium: Hidden in Plain Sight: ATTR-CM in Overlooked Patient Populations
Transthyretin amyloid cardiomyopathy, or ATTR-CM, is a serious and underdiagnosed type of amyloidosis that primarily affects the heart and leads to heart failure. Ionis will host a symposium focused on the prevalence of ATTR-CM in Blacks and African Americans and in subpopulations with heart failure with preserved ejection fraction (HFpEF), addressing biological and genetically inherited differences, implicit bias, and healthcare challenges that may disproportionately impact the diagnosis and treatment of those groups. Panel includes Dr. Mathew MaurerColumbia University; Dr. Alanna Morris, Emory University; Dr. Kevin AlexanderStanford University; and Dr. Keyur ShahVirginia Commonwealth University.

The following are presentations that will be available on the AHA Scientific Sessions’ website.

Saturday, Nov. 13, 20218:00 AM – 5:30 PM
Safety, pharmacokinetics and pharmacodynamics of multiple ascending doses of AZD8233 targeting PCSK9, in patients with dyslipidemia (Oral presentation 268)
FAT’s What It’s All About! Lipid, Lipid Lowering Agents and PCI

Saturday, Nov. 13, 20218:00 AM – 5:30 PM (ePoster VMP33)
Effect of olezarsen on lipoprotein size and particle numbers measured by NMR in patients with hypertriglyceridemia: Results of the olezarsen trial 
New Mechanisms and Therapeutic Developments in Lipid Metabolism

Saturday, Nov. 13, 20218:00-5:30 PM (on demand)
George Lyman Duff Memorial Lecture: Interventional Hepatic ApoC-III Knockdown Improves Atherosclerotic Plaque Stability and Remodeling via Lowering Remnant Lipoproteins

Saturday, Nov. 13, 20212:30-3:30 PM (ePoster P1104)
Relationship Between Circulating Levels of Angiotensinogen and Hypertension—The Multi-Ethnic Study of Atherosclerosis

Saturday, Nov. 13, 202111:00 AM – 12:00 PM (ePoster P108/1006)
Total lipoprotein lipase deficiency does not impact atherosclerosis development in low density lipoprotein receptor deficient mice
Lipids and Lipoproteins in Atherosclerosis