Ronopterin

Summary

Ronopterin (CHEMBL1230245) is a phase-3 clinical-stage small molecule; indicated across 2 conditions including brain injury and kidney disorder.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 2 conditions
• Clinical trials: 3
• Chemistry: 240.26 Da · C9H16N6O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1230245
Name	Ronopterin
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	5287516
Molecular formula	C9H16N6O2
Molecular weight	240.26
InChIKey	NDSDGUULXHNXGA-RPDRRWSUSA-N

SMILES: C[C@@H]([C@@H]([C@H]1CNC2=NC(=NC(=C2N1)N)N)O)O

IUPAC name: (1R,2S)-1-[(6R)-2,4-diamino-5,6,7,8-tetrahydropteridin-6-yl]propane-1,2-diol

Also known as: Ronopterin, Ronopterin, (6r)-, Ronopterina, Ronopterine, VAS-203, Vas203, VAS203, RONOPTERIN

Patent coverage: 34 distinct patent families (93 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

2 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
brain injury	3	MONDO:0043510	MONDO:0043510
kidney disorder	1	MONDO:0005240	EFO:0003086

Clinical trials

Total trials: 3.

Phase distribution

Phase	Trials
PHASE3	1
PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02794168	PHASE3	COMPLETED	Efficacy of VAS203 (Ronopterin) in Patients With Moderate and Severe Traumatic Brain Injury
NCT02012582	PHASE2	COMPLETED	Phase 2 a Study to Assess Safety and Pharmacokinetics of VAS203 in Patients With Traumatic Brain Injury
NCT02992236	PHASE1	COMPLETED	Effects of the NO-synthase Inhibitor VAS203 on Renal Function in Healthy Volunteers

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).