Ulefnersen

Summary

Ulefnersen (CHEMBL5095349) is a phase-3 clinical-stage oligonucleotide; indicated across 1 condition including amyotrophic lateral sclerosis.

At a glance

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

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL5095349
Name	Ulefnersen
Type	Oligonucleotide
Max phase	3

Also known as: ION-363, ION363, Ulefnersen, ULEFNERSEN

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 disease in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
amyotrophic lateral sclerosis	1	MONDO:0004976	MONDO:0004976

Clinical trials

Total trials: 1.

Phase distribution

Phase	Trials
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04768972	PHASE3	ACTIVE_NOT_RECRUITING	FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

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).