Pentamidine

Summary

Pentamidine (CHEMBL55) is an approved small-molecule trypanocidal drug; indicated across 11 conditions including trypanosomiasis and cutaneous melanoma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• Indications: 11 conditions
• Clinical trials: 12
• Chemistry: 340.4 Da · C19H24N4O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL55
Name	Pentamidine
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	4735
ChEBI	CHEBI:45081
Molecular formula	C19H24N4O2
Molecular weight	340.4
InChIKey	XDRYMKDFEDOLFX-UHFFFAOYSA-N

SMILES: C1=CC(=CC=C1C(=N)N)OCCCCCOC2=CC=C(C=C2)C(=N)N

IUPAC name: 4-[5-(4-carbamimidoylphenoxy)pentoxy]benzenecarboximidamide

ChEBI definition: A diether consisting of pentane-1,5-diol in which both hydroxyl hydrogens have been replaced by 4-amidinophenyl groups. A trypanocidal drug that is used for treatment of cutaneous leishmaniasis and Chagas disease.

Pharmacological roles (ChEBI): trypanocidal drug, antifungal agent, NMDA receptor antagonist, anti-inflammatory agent, chemokine receptor 5 antagonist, EC 2.3.1.48 (histone acetyltransferase) inhibitor, calmodulin antagonist, S100 calcium-binding protein B inhibitor.

Other ChEBI roles (chemical / environmental): xenobiotic.

Also known as: GNF-Pf-3680, MB 800 FREE BASE, MB-800, MB-800 (AS ISETHIONATE), MB-800 FREE BASE, Mp-601205, NSC-9921, Pentacarinat, Pentam 300, Pentamidina, Pentamidine, RP 2512 FREE BASE

Parent form; salt/anhydrous children: CHEMBL935, CHEMBL361506, CHEMBL494163, CHEMBL543544

Patent coverage: 6,998 distinct patent families (27,049 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: 42 (assay-derived). Sample: Protein tyrosine phosphatase type IVA 2, Protein tyrosine phosphatase type IVA 1, Solute carrier family 22 member 2, Multidrug and toxin extrusion protein 1, Multidrug and toxin extrusion protein 2, Transmembrane protease serine 2, Plasminogen, Muscarinic acetylcholine receptor M4, Alpha-2A adrenergic receptor, Glutamate NMDA receptor; GRIN1/GRIN2A.

Bioactivity

ChEMBL activities: 60 potent at pChembl ≥ 5 of 78 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CHRM4	6.66	Ki	220	nM	CHEMBL_ACT_7719389
AOC1	6.54	Ki	290	nM	CHEMBL_ACT_14581414
PTP4A1	6.52	IC50	300	nM	CHEMBL_ACT_19453963
PTP4A2	6.52	IC50	300	nM	CHEMBL_ACT_19453964
PTP4A3	6.52	IC50	300	nM	CHEMBL_ACT_19453965
SLC47A1	6.39	IC50	410	nM	CHEMBL_ACT_12636120
HTR2A	6.33	Ki	468	nM	CHEMBL_ACT_7721456
P43140	6.31	Ki	491	nM	CHEMBL_ACT_7719247
ADRA2A	6.29	Ki	516	nM	CHEMBL_ACT_7719253
CHRM2	6.29	Ki	507	nM	CHEMBL_ACT_7719385
P15823	6.27	Ki	535	nM	CHEMBL_ACT_7719249
HRH3	6.19	AC50	643.2	nM	CHEMBL_ACT_25199306
GRIN1	6.14	IC50	720	nM	CHEMBL_ACT_14578584
MAOA	6.1	IC50	793	nM	CHEMBL_ACT_7719380
P15823	6.01	IC50	967	nM	CHEMBL_ACT_7719248
S100B	6	Kd	1000	nM	CHEMBL_ACT_1468033
ST14	5.94	Ki	1160	nM	CHEMBL_ACT_724145
SLC22A2	5.92	IC50	1200	nM	CHEMBL_ACT_12636174
TMPRSS2	5.92	IC50	1200	nM	CHEMBL_ACT_25068125
P43140	5.92	IC50	1212	nM	CHEMBL_ACT_7719246
P35439	5.88	IC50	1310	nM	CHEMBL_ACT_1072592
SLC6A2	5.88	AC50	1326	nM	CHEMBL_ACT_25145774
DRD3	5.87	Ki	1358	nM	CHEMBL_ACT_7719325
ADRA2A	5.86	IC50	1375	nM	CHEMBL_ACT_7719252
CHRM2	5.85	IC50	1425	nM	CHEMBL_ACT_7719384
CHRM2	5.8	AC50	1582	nM	CHEMBL_ACT_25195510
CHRM4	5.8	IC50	1580	nM	CHEMBL_ACT_7719388
HTR2A	5.79	IC50	1639	nM	CHEMBL_ACT_7721455
P35439	5.76	IC50	1740	nM	CHEMBL_ACT_1072593
HTR2C	5.76	Ki	1717	nM	CHEMBL_ACT_7721460

Target pathways

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

Indications & clinical

Indications

11 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
trypanosomiasis	2	MONDO:0000940	DOID:10113
cutaneous melanoma	2	MONDO:0005012	EFO:0000389
leishmaniasis	2	MONDO:0011989	EFO:0005044
type 1 diabetes mellitus	2	MONDO:0005147	MONDO:0005147
colorectal neoplasm	2	MONDO:0005335	MONDO:0005575
exocrine pancreatic carcinoma	1	MONDO:0005192	EFO:0002618
hepatocellular carcinoma	1	MONDO:0007256	EFO:0000182
kidney disorder	1	MONDO:0005240	EFO:0003086
Hodgkins lymphoma	1	MONDO:0004952	EFO:0000183

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

Clinical trials

Total trials: 12.

Phase distribution

Phase	Trials
PHASE2	4
PHASE1/PHASE2	3
PHASE1	2
PHASE4	1
PHASE3	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07357103	PHASE4	NOT_YET_RECRUITING	Positioning Second-line Therapies for Pneumocystis Jirovecii Pneumonia (PCP Alternatives)
NCT01360762	PHASE3	COMPLETED	Prophylaxis of Visceral Leishmaniasis Relapses in HIV Co-infected Patients With Pentamidine: a Cohort Study
NCT00002524	PHASE2	COMPLETED	Combination Chemotherapy in Treating Patients With AIDS-Related Lymphoma
NCT00014911	PHASE2	COMPLETED	Islet Transplantation for Type 1 Diabetes
NCT00078559	PHASE1/PHASE2	COMPLETED	Combination Immunosuppressive Therapy to Prevent Kidney Transplant Rejection in Adults
NCT00729807	PHASE2	TERMINATED	0794GCC: Pentamidine in Treating Patients With Relapsed or Refractory Melanoma
NCT00803933	PHASE2	COMPLETED	Trial of DB289 for the Treatment of Stage I African Trypanosomiasis
NCT00809796	PHASE1/PHASE2	COMPLETED	A Safety Study of Pentamidine in Patients With Metastatic Colon Cancer Undergoing Standard Chemotherapy as Second-line and/or Third-line Treatment
NCT00810953	PHASE1/PHASE2	COMPLETED	A Safety Study Using Pentamidine in Patients With Pancreatic Cancer Undergoing Standard Therapy
NCT02210182	PHASE1	COMPLETED	A Phase I Clinical Study on a New Oral Pentamidine Formulation in Hepatocellular Carcinoma
NCT03730363	PHASE1	COMPLETED	Pentamidine + Salvage Chemo for Relapsed/Refractory Classical Hodgkin Lymphoma
NCT02669706	Not specified	COMPLETED	Intravenous Pentamidine for Pneumocystis Jirovecii Pneumonia

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 0 clinical and 3 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).