Phenformin

Summary

Phenformin (CHEMBL170988) is an approved small-molecule antineoplastic agent (ATC A10BA01); indicated across 2 conditions including diabetes mellitus and melanoma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: A10BA01
• Indications: 2 conditions
• Clinical trials: 1
• Chemistry: 205.26 Da · C10H15N5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL170988
Name	Phenformin
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	8249
ChEBI	CHEBI:8064
ATC	A10BA01
Molecular formula	C10H15N5
Molecular weight	205.26
InChIKey	ICFJFFQQTFMIBG-UHFFFAOYSA-N

SMILES: C1=CC=C(C=C1)CCN=C(N)N=C(N)N

IUPAC name: 1-(diaminomethylidene)-2-(2-phenylethyl)guanidine

ChEBI definition: A member of the class of biguanides that is biguanide in which one of the terminal nitrogen atoms is substituted by a 2-phenylethyl group. It was used as an anti-diabetic drug but was later withdrawn from the market due to potential risk of lactic acidosis.

Pharmacological roles (ChEBI): antineoplastic agent, geroprotector, hypoglycemic agent.

Also known as: Fenformina, Phenformin, Phenformine, SID11112448, SID124882390, phenformin, PHENFORMINE, SID174006812, PHENFORMIN, PHENFORMIN HYDROCHLORIDE

Parent form; salt/anhydrous children: CHEMBL1528839, CHEMBL2348409

Patent coverage: 11,471 distinct patent families (37,492 SureChEMBL compound mentions), from 7 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: 15 (assay-derived). Sample: Prelamin-A/C, Solute carrier family 22 member 2, 5-hydroxytryptamine receptor 2B, Alpha-2A adrenergic receptor, Alpha-2B adrenergic receptor, Thyrotropin receptor, Sodium-dependent noradrenaline transporter, Alpha-1A adrenergic receptor, Sodium-dependent dopamine transporter, cGMP-inhibited 3’,5’-cyclic phosphodiesterase 3A.

Bioactivity

ChEMBL activities: 11 potent at pChembl ≥ 5 of 19 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
LMNA	6.4	Potency	398.1	nM	CHEMBL_ACT_3667972
P35439	5.63	AC50	2321	nM	CHEMBL_ACT_25120426
HIF1A	5.2	Potency	6310	nM	CHEMBL_ACT_4128811
HIF1A	5.2	Potency	6310	nM	CHEMBL_ACT_4520633
ADRA2A	5.17	AC50	6711	nM	CHEMBL_ACT_25155882
TSHR	5.1	Potency	7943	nM	CHEMBL_ACT_3926301
TSHR	5.1	Potency	7943	nM	CHEMBL_ACT_4751500
HTR2B	5.05	AC50	8905	nM	CHEMBL_ACT_25164052
SLC22A1	5	IC50	10000	nM	CHEMBL_ACT_11000953
CYP2D6	5	Potency	10000	nM	CHEMBL_ACT_5010541
CYP2D6	5	AC50	10000	nM	CHEMBL_ACT_5986828

Target pathways

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

Indications & clinical

Indications

2 indications (1 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
diabetes mellitus	4	MONDO:0005015	EFO:0000400
melanoma	1	MONDO:0005105	EFO:0000756

Clinical trials

Total trials: 1.

Phase distribution

Phase	Trials
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03026517	PHASE1	COMPLETED	Clinical Trial of Phenformin in Combination With BRAF Inhibitor + MEK Inhibitor for Patients With BRAF-mutated Melanoma

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

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