Tiopronin

Summary

Tiopronin (CHEMBL1314) is an approved small molecule (ATC G04BX16); indicated across 3 conditions including subarachnoid hemorrhage and drug-induced liver injury.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: G04BX16
• Indications: 3 conditions
• Clinical trials: 4
• Chemistry: 163.2 Da · C5H9NO3S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1314
Name	Tiopronin
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	5483
ATC	G04BX16
Molecular formula	C5H9NO3S
Molecular weight	163.2
InChIKey	YTGJWQPHMWSCST-UHFFFAOYSA-N

SMILES: CC(C(=O)NCC(=O)O)S

IUPAC name: 2-(2-sulfanylpropanoylamino)acetic acid

Also known as: Acadione, Captimer, NSC-760416, Thiola, Thiola ec, Tiopronin, Tiopronina, Tiopronine, SID29215361, TIOPRONIN, SID144205207, tiopronin

Patent coverage: 2,397 distinct patent families (7,415 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

Broader ChEMBL bioactivity targets: 8 (assay-derived). Sample: Tyrosyl-DNA phosphodiesterase 1, Microtubule-associated protein tau, Prelamin-A/C, Prostaglandin G/H synthase 2, Aldehyde dehydrogenase 1A1, Prostaglandin G/H synthase 1, Angiotensin-converting enzyme, Metallo-beta-lactamase type 2.

Bioactivity

ChEMBL activities: 5 potent at pChembl ≥ 5 of 10 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
TDP1	6	Potency	1000	nM	CHEMBL_ACT_3927681
PTGS2	5.8	AC50	1600	nM	CHEMBL_ACT_25166822
P12822	5.72	IC50	1900	nM	CHEMBL_ACT_639854
P12822	5.72	IC50	1900	nM	CHEMBL_ACT_702555
Q63921	5.48	AC50	3300	nM	CHEMBL_ACT_25175043

Target pathways

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

Indications & clinical

Indications

3 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
subarachnoid hemorrhage	2	MONDO:0005099	EFO:0000713
drug-induced liver injury	2	MONDO:0005359	EFO:1000905
cystinuria	2	MONDO:0009067	MONDO:0009067

Clinical trials

Total trials: 4.

Phase distribution

Phase	Trials
PHASE2	3
PHASE4	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02125721	PHASE4	COMPLETED	Effect of Increasing Doses of Cystine Binding Thiol Drugs on Cystine Capacity in Patients With Cystinuria
NCT01095731	PHASE2	COMPLETED	The Effects of Tiopronin on 3-Aminopropanal Level & Neurologic Outcome After Aneurysmal Subarachnoid Hemorrhage
NCT02734966	PHASE2	COMPLETED	Efficacy and Safety Study of Magnesium Isoglycyrrhizinate Injection in Subjects With Acute Drug-induced Liver Injury
NCT03663855	PHASE2	COMPLETED	Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria

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).