Tominersen

Summary

Tominersen (CHEMBL4297581) is a phase-3 clinical-stage oligonucleotide; indicated across 5 conditions including huntington disease and pyoderma gangrenosum.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligonucleotide
• Indications: 5 conditions
• Clinical trials: 5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297581
Name	Tominersen
Type	Oligonucleotide
Max phase	3

Also known as: Ionis httrx, Isis 443139, ISIS-443139 FREE ACID, RG-6042 FREE ACID, Rg6042, RG6042, RG6042 FREE ACID, Ro7234292, Tominersen, TOMINERSEN

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

5 indications (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
Huntington disease	3	MONDO:0007739	MONDO:0007739
pyoderma gangrenosum	1	MONDO:0018824	EFO:0006835
hidradenitis suppurativa	1	MONDO:0006559	EFO:1000710
anti-neutrophil cytoplasmic antibody-associated vasculitis	1	MONDO:0015492	MONDO:0015492

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 5.

Phase distribution

Phase	Trials
PHASE3	2
PHASE2	2
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03761849	PHASE3	COMPLETED	A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington’s Disease
NCT03842969	PHASE3	COMPLETED	An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington’s Disease Participants Who Participated in Prior Roche and Genentech Sponsored Studies
NCT05686551	PHASE2	ACTIVE_NOT_RECRUITING	GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington’s Disease
NCT03342053	PHASE2	COMPLETED	A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington’s Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139)
NCT04000594	PHASE1	COMPLETED	A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington’s 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).