Mizoribine

Summary

Mizoribine (CHEMBL245019) is a phase-3 clinical-stage small molecule; indicated across 3 conditions including rheumatoid arthritis and nephrotic syndrome.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 3 conditions
• Clinical trials: 5
• Chemistry: 259.22 Da · C9H13N3O6

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL245019
Name	Mizoribine
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	104762
Molecular formula	C9H13N3O6
Molecular weight	259.22
InChIKey	HZQDCMWJEBCWBR-UUOKFMHZSA-N

SMILES: C1=NC(=C(N1[C@H]2[C@@H]([C@@H]([C@H](O2)CO)O)O)O)C(=O)N

IUPAC name: 1-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-hydroxyimidazole-4-carboxamide

Also known as: Bredinin, Mizoribina, Mizoribine, NSC-289637, SID11532986, SID50106568, SID90340602, SID50106569, MIZORIBINE, SID170466027, MIZORIBINE (BREDININ), MizoribineÊ

Patent coverage: 3,681 distinct patent families (14,790 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: 6 (assay-derived). Sample: Lysine-specific demethylase 4E, Survival motor neuron protein, RecQ-like DNA helicase BLM, Lysosomal alpha-glucosidase, Aldehyde dehydrogenase 1A1, Huntingtin.

Bioactivity

ChEMBL activities: 5 potent at pChembl ≥ 5 of 7 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
BLM	8.55	Potency	2.8	nM	CHEMBL_ACT_4749927
BLM	8.55	Potency	2.8	nM	CHEMBL_ACT_4933916
SMN1	7.45	Potency	35.5	nM	CHEMBL_ACT_3888395
HTT	6.25	Potency	562.3	nM	CHEMBL_ACT_3763289
GAA	5.05	Potency	8844	nM	CHEMBL_ACT_4922464

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
rheumatoid arthritis	3	MONDO:0008383	EFO:0000685
nephrotic syndrome	3	MONDO:0005377	EFO:0004255
lupus nephritis	3	MONDO:0005556	EFO:0005761

Clinical trials

Total trials: 5.

Phase distribution

Phase	Trials
PHASE3	3
PHASE4	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05293704	PHASE4	UNKNOWN	An Open Study on the Preventive Effect of Early Mizoribine Conversion on BKV Nephropathy in Renal Transplant Recipients
NCT06114953	PHASE4	UNKNOWN	Comparative Efficacy of Mizoribine With Mycophenolate Mofetil for Living Related Kidney Transplantation Recipients
NCT02256150	PHASE3	COMPLETED	A Study to Evaluate the Efficacy and Safety of Mizoribine in the Treatment of Lupus Nephritis
NCT02257697	PHASE3	COMPLETED	A Study to Evaluate the Efficacy and Safety of Mizoribine in the Treatment of Refractory Nephrotic Syndrome
NCT02373202	PHASE3	COMPLETED	A Study Assessing the Safety and Efficacy of Sarilumab Added to Non-MTX DMARDs or as Monotherapy in Japanese Patients With Active Rheumatoid Arthritis (SARIL-RA-HARUKA)

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).