Triptolide

Summary

Triptolide (CHEMBL463763) is a phase-3 clinical-stage small molecule; indicated across 5 conditions including autosomal dominant polycystic kidney disease and hiv infectious disease.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 5 conditions
• Clinical trials: 3
• Chemistry: 360.4 Da · C20H24O6

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL463763
Name	Triptolide
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	107985
Molecular formula	C20H24O6
Molecular weight	360.4
InChIKey	DFBIRQPKNDILPW-CIVMWXNOSA-N

SMILES: CC(C)[C@@]12[C@@H](O1)[C@H]3[C@@]4(O3)[C@]5(CCC6=C([C@@H]5C[C@H]7[C@]4([C@@H]2O)O7)COC6=O)C

IUPAC name: (1S,2S,4S,5S,7R,8R,9S,11S,13S)-8-hydroxy-1-methyl-7-propan-2-yl-3,6,10,16-tetraoxaheptacyclo[11.7.0.02,4.02,9.05,7.09,11.014,18]icos-14(18)-en-17-one

Also known as: NSC-163062, Triptolide, triptolide, SID26719814, SID26756783, SID49681800, SID438041, SID26756784, TRIPTOLIDE, C0164704

Patent coverage: 133 distinct patent families (258 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: 9 (assay-derived). Sample: Nuclear receptor ROR-gamma, Prelamin-A/C, 4’-phosphopantetheinyl transferase ffp, NPC intracellular cholesterol transporter 1, Ras-related protein Rab-9A, Nuclear factor NF-kappa-B complex, Prostaglandin G/H synthase 2, dCTP pyrophosphatase 1, Proteinase-activated receptor 2.

Bioactivity

ChEMBL activities: 10 potent at pChembl ≥ 5 of 15 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
NFKB1	8	IC50	10	nM	CHEMBL_ACT_25878327
DCTPP1	8	IC50	10	nM	CHEMBL_ACT_25878336
F2RL1	7.85	IC50	14	nM	CHEMBL_ACT_13465011
F2RL1	7.64	IC50	23	nM	CHEMBL_ACT_13465010
PTGS2	7.4	IC50	40	nM	CHEMBL_ACT_2324536
LMNA	7.05	Potency	89.1	nM	CHEMBL_ACT_3643123
NPC1	7.05	Potency	89.1	nM	CHEMBL_ACT_4744872
P51450	6.5	Potency	316.2	nM	CHEMBL_ACT_5011508
P51450	6.4	Potency	398.1	nM	CHEMBL_ACT_4090786
RAB9A	6	Potency	1000	nM	CHEMBL_ACT_3848774

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
autosomal dominant polycystic kidney disease	3	MONDO:0004691	EFO:1001496
HIV infectious disease	1	MONDO:0005109	EFO:0000764
non-small cell lung carcinoma	1	MONDO:0005233	EFO:0003060
lung neoplasm	1	MONDO:0021117	MONDO:0008903

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

Clinical trials

Total trials: 3.

Phase distribution

Phase	Trials
PHASE3	1
PHASE1/PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03403569	PHASE3	COMPLETED	Safety and Efficacy of Triptolide Wilfordii in New Onset HIV-1 Infection
NCT01817283	PHASE1/PHASE2	UNKNOWN	Impact on T Cell Immune Activation and Inflammation of Triptolide Woldifii in HIV-infected Immunological Non-responders
NCT05166616	PHASE1	ACTIVE_NOT_RECRUITING	Minnelide and Osimertinib for the Treatment of Advanced EGFR Mutated Non-Small Cell Lung Cancer

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).