Last reviewed 2026-04-23 · How we verify

ION582

ION582 is an antisense oligonucleotide that reduces production of apolipoprotein B (ApoB) to lower LDL cholesterol levels.

ION582 is an antisense oligonucleotide that reduces production of apolipoprotein B (ApoB) to lower LDL cholesterol levels. Used for Hypercholesterolemia; LDL cholesterol reduction in patients at high cardiovascular risk.

At a glance

Mechanism of action

ION582 works by binding to and degrading the mRNA that encodes apolipoprotein B, a critical structural protein required for the formation and secretion of LDL particles. By reducing ApoB production in the liver, the drug decreases circulating LDL cholesterol levels. This mechanism is distinct from statins and PCSK9 inhibitors, offering an alternative approach to lipid-lowering therapy.

Approved indications

• Hypercholesterolemia; LDL cholesterol reduction in patients at high cardiovascular risk

Common side effects

• Injection site reactions
• Liver enzyme elevations
• Flu-like symptoms

Key clinical trials

• REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome (PHASE3)
• HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome (PHASE1, PHASE2)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• ION582 CI brief — competitive landscape report
• ION582 updates RSS · CI watch RSS
• Ionis Pharmaceuticals, Inc. portfolio CI