Arimoclomol

Summary

Arimoclomol (CHEMBL2107726) is an approved small molecule (ATC N07XX17); indicated across 4 conditions including inclusion body myositis and amyotrophic lateral sclerosis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: N07XX17
• Indications: 4 conditions
• Clinical trials: 11
• Chemistry: 313.78 Da · C14H20ClN3O3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2107726
Name	Arimoclomol
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	9568077
ATC	N07XX17
Molecular formula	C14H20ClN3O3
Molecular weight	313.78
InChIKey	SGEIEGAXKLMUIZ-XUVDNFPMSA-N

SMILES: C1CCN(CC1)C[C@H](CO/N=C(/C2=C[N+](=CC=C2)[O-])\Cl)O

IUPAC name: (3Z)-N-[(2R)-2-hydroxy-3-piperidin-1-ylpropoxy]-1-oxidopyridin-1-ium-3-carboximidoyl chloride

Also known as: Arimoclomol, BRX-220 FREE BASE, ARIMOCLOMOL

Parent form; salt/anhydrous children: CHEMBL4298149

Patent coverage: 20 distinct patent families (30 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

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

4 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
inclusion body myositis	3	MONDO:0007827	EFO:0007323
amyotrophic lateral sclerosis	3	MONDO:0004976	MONDO:0004976
Gaucher disease	2	MONDO:0018150	MONDO:0018150
Niemann-Pick disease type C	2	MONDO:0018982	MONDO:0018982

Clinical trials

Total trials: 11.

Phase distribution

Phase	Trials
PHASE2	4
PHASE2/PHASE3	3
PHASE3	3
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00706147	PHASE2/PHASE3	COMPLETED	Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis
NCT00769860	PHASE2/PHASE3	COMPLETED	Arimoclomol in Sporadic Inclusion Body Myositis
NCT02612129	PHASE2/PHASE3	COMPLETED	Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C
NCT03491462	PHASE3	COMPLETED	Arimoclomol in Amyotropic Lateral Sclerosis
NCT03836716	PHASE3	TERMINATED	Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial
NCT04049097	PHASE3	TERMINATED	Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
NCT00244244	PHASE2	COMPLETED	A Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in ALS
NCT00561366	PHASE2	WITHDRAWN	A Multicenter, Double-Blind Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With ALS
NCT02753530	PHASE2	COMPLETED	Study of Arimoclomol in Inclusion Body Myositis (IBM)
NCT03746587	PHASE2	TERMINATED	Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
NCT04316637	Not specified	AVAILABLE	Early Access Program With Arimoclomol in US Patients With NPC

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).