Zilganersen

Summary

Zilganersen (CHEMBL5095433) is a phase-3 clinical-stage oligonucleotide; indicated across 1 condition including alexander disease.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligonucleotide
• Indications: 1 condition
• Clinical trials: 2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL5095433
Name	Zilganersen
Type	Oligonucleotide
Max phase	3

Also known as: ION-1166998 (free acid), ION-1166998 FREE ACID, Zilganersen, ZILGANERSEN

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

1 indication (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
Alexander disease	3	MONDO:0008752	MONDO:0008752

Clinical trials

Total trials: 2.

Phase distribution

Phase	Trials
PHASE3	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04849741	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
NCT07487389	Not specified	AVAILABLE	Zilganersen Expanded Access Program for Individuals With Alexander Disease

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).