Lonidamine

Summary

Lonidamine (CHEMBL1257030) is a phase-3 clinical-stage small-molecule antispermatogenic agent (ATC L01XX07) targeting MPC2 and MPC1L; indicated across 2 conditions including neoplasm and benign prostatic hyperplasia.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• ATC class: L01XX07
• Targets: 2 (MPC2, MPC1L)
• Indications: 2 conditions
• Clinical trials: 2
• Chemistry: 321.2 Da · C15H10Cl2N2O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1257030
Name	Lonidamine
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	39562
ChEBI	CHEBI:50138
ATC	L01XX07
Molecular formula	C15H10Cl2N2O2
Molecular weight	321.2
InChIKey	WDRYRZXSPDWGEB-UHFFFAOYSA-N

SMILES: C1=CC=C2C(=C1)C(=NN2CC3=C(C=C(C=C3)Cl)Cl)C(=O)O

IUPAC name: 1-[(2,4-dichlorophenyl)methyl]indazole-3-carboxylic acid

ChEBI definition: A member of the class of indazoles that is 1H-indazole that is substituted at positions 1 and 3 by 2,4-dichlorobenzyl and carboxy groups, respectively.

Pharmacological roles (ChEBI): antispermatogenic agent, antineoplastic agent, geroprotector, EC 2.7.1.1 (hexokinase) inhibitor.

Also known as: Doridamina, Lonidamina, Lonidamine, NSC-741419, NSC-758419, SID11111381, SID11114165, SID11532966, SID50104921, SID85231114, SID90341155, SID124880557

Patent coverage: 9,381 distinct patent families (34,131 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 30,174 (88%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
MPC2	mitochondrial pyruvate carrier 2	Inhibition	5.34	1%	O95563
MPC1L	mitochondrial pyruvate carrier 1 like	Inhibition	5.34	0%	P0DKB6

Broader ChEMBL bioactivity targets: 11 (assay-derived). Sample: Nuclear receptor ROR-gamma, Peripheral myelin protein 22, Leucine aminopeptidase, Thyrotropin receptor, Beta-lactamase, Cytochrome P450 2D6, Cytochrome P450 1A2, Cytochrome P450 2C9, Cytochrome P450 3A4, Hypoxia-inducible factor 1-alpha.

Bioactivity

ChEMBL activities: 6 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
P00811	6.25	Potency	562.3	nM	CHEMBL_ACT_4673010
CYP3A4	5.6	Potency	2512	nM	CHEMBL_ACT_5002049
CYP3A4	5.6	Potency	2512	nM	CHEMBL_ACT_5066953
CYP3A4	5.6	AC50	2512	nM	CHEMBL_ACT_6018514
NPSR1	5.5	Potency	3162	nM	CHEMBL_ACT_4924763
Q8IL11	5.13	IC50	7370	nM	CHEMBL_ACT_4683041

Target pathways

Aggregated over 2 target gene(s): MPC2, MPC1L.

Top Reactome pathways

3 total, by targets touching each:

Pathway	Targets	Genes
Aerobic respiration and respiratory electron transport	2	MPC1L, MPC2
Metabolism	2	MPC1L, MPC2
Pyruvate metabolism	2	MPC1L, MPC2

Dominant GO biological processes

GO term	Targets
pyruvate import into mitochondria	2
pyruvate decarboxylation to acetyl-CoA	1
positive regulation of insulin secretion involved in cellular response to glucose stimulus	1

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
neoplasm	3	MONDO:0005070	EFO:0000616
benign prostatic hyperplasia	3	MONDO:0010811	EFO:0000284

Clinical trials

Total trials: 2.

Phase distribution

Phase	Trials
PHASE3	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00435448	PHASE3	TERMINATED	Study of the Efficacy and Safety of Lonidamine for the Treatment of Symptomatic Benign Prostatic Hyperplasia
NCT00237536	PHASE2	TERMINATED	Study of the Efficacy and Safety of Lonidamine for the Treatment of Symptomatic Benign Prostatic Hyperplasia

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

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

1 molecules share ≥1 primary target. Top 1 by shared-target count:

Molecule	Source	Status	Shared targets
MITOGLITAZONE	ChEMBL	Phase 2	MPC2