ION582

Phase 3Small molecule

Ionis Pharmaceuticals

ION582 is one of 12 small molecule programs targeting Angelman syndrome. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Angelman SyndromeActive, not recruiting

Angelman SyndromePhase 3

Active, not recruiting

Competition

Competitive Landscape

16 competitors in Angelman Syndrome

#DrugCompanyPhaseTrials

Lodosyn

Gaboxadol

1 3

Minocin

TTriax

Phase 2

2 4

Bendopa

MMerz Pharmaceuticals GmbH

Phase 2

1 5

GTX-102

Ultragenyx

Phase 3

4 6

OV101

HHealx AI

Phase 3

2 7

ION582

Ionis Pharmaceuticals

Phase 3

2 8

Levodopa/carbidopa

Orion Corporation, Orion Pharma

Phase 1

1 9

NNZ-2591

NNeuren Pharmaceuticals Limited

Phase 2

1 10

Cystadane

RRECORDATI GROUP

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q1 2029

Phase 1/2Recruiting

Angelman Syndrome

NCT05127226

Q2 2030

Phase 3Recruiting

Angelman Syndrome

NCT06914609

Clinical

Trials by Indication[1]

Angelman syndrome2 trials

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

Recruiting· Ionis Pharmaceuticals logo

Ionis Pharmaceuticals·Phase 1/2·NCT05127226·70 enrolled

Phase 3 Study of the Efficacy and Safety of ION582 in Children and Adults With Angelman Syndrome

Recruiting· Ionis Pharmaceuticals logo

Ionis Pharmaceuticals·Phase 3·NCT06914609·158 enrolled

Sources

References

[1]
ClinicalTrials.gov — 2 trial records for ION582. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — ION582 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for ION582. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Ionis Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: Ionis Pharmaceuticals

ION582

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2029

Q2 2030

Trials by Indication[1]

Angelman syndrome2 trials

References

Drug Info

Quick Facts

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