Podofilox

Summary

Podofilox (CHEMBL61) is an approved small-molecule antineoplastic agent (ATC D06BB04); indicated across 3 conditions including viral infectious disease and anogenital human papillomavirus infection.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: D06BB04
• Indications: 3 conditions
• Clinical trials: 2
• Chemistry: 414.4 Da · C22H22O8

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL61
Name	Podofilox
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	10607
ChEBI	CHEBI:50305
ATC	D06BB04
Molecular formula	C22H22O8
Molecular weight	414.4
InChIKey	YJGVMLPVUAXIQN-XVVDYKMHSA-N

SMILES: COC1=CC(=CC(=C1OC)OC)[C@H]2[C@@H]3[C@H](COC3=O)[C@H](C4=CC5=C(C=C24)OCO5)O

IUPAC name: (5R,5aR,8aR,9R)-5-hydroxy-9-(3,4,5-trimethoxyphenyl)-5a,6,8a,9-tetrahydro-5H-[2]benzofuro[5,6-f][1,3]benzodioxol-8-one

ChEBI definition: An organic heterotetracyclic compound that has a furonaphthodioxole skeleton bearing a 3,4,5-trimethoxyphenyl substituent. It is found in the roots and rhizomes of Podophyllum species and is used for the topical treatment of genital warts.

Pharmacological roles (ChEBI): antineoplastic agent, keratolytic drug, tubulin modulator, microtubule-destabilising agent, antimitotic.

Other ChEBI roles (chemical / environmental): plant metabolite.

Also known as: Condyline, Condylox, NSC-24818, Podofilox, Podophyllotoxin, Warticon, podophyllotoxin, podophylotoxin, podophyllotoxine, SID29215001, SID50103982, SID855658

Patent coverage: 10,534 distinct patent families (37,640 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 37,274 (99%) 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

Broader ChEMBL bioactivity targets: 22 (assay-derived). Sample: Survival motor neuron protein, Prelamin-A/C, NPC intracellular cholesterol transporter 1, Ras-related protein Rab-9A, Nuclear receptor subfamily 0 group B member 1, Glucocorticoid receptor, Tubulin, Tubulin, Tubulin, Tubulin.

Bioactivity

ChEMBL activities: 52 potent at pChembl ≥ 5 of 58 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
NR3C1	7.85	IC50	14	nM	CHEMBL_ACT_5156256
HIF1A	7.8	Potency	15.8	nM	CHEMBL_ACT_4130682
HIF1A	7.8	Potency	15.8	nM	CHEMBL_ACT_4518959
LMNA	7.55	Potency	28.2	nM	CHEMBL_ACT_3627429
LMNA	7.5	Potency	31.6	nM	CHEMBL_ACT_3654889
RAB9A	6.95	Potency	112.2	nM	CHEMBL_ACT_3858349
TUBB4A	6.58	IC50	260	nM	CHEMBL_ACT_18357192
P02550	6.46	IC50	350	nM	CHEMBL_ACT_1798097
P02550	6.46	IC50	350	nM	CHEMBL_ACT_2003723
P02550	6.46	IC50	350	nM	CHEMBL_ACT_2388435
P02550	6.46	IC50	350	nM	CHEMBL_ACT_316906
P02550	6.46	IC50	350	nM	CHEMBL_ACT_3372033
TUBB4A	6.46	IC50	350	nM	CHEMBL_ACT_3372168
P02550	6.46	IC50	350	nM	CHEMBL_ACT_6275459
P02550	6.46	IC50	350	nM	CHEMBL_ACT_6310447
SMN1	6.35	Potency	446.7	nM	CHEMBL_ACT_3862394
Q6B856	6.34	IC50	460	nM	CHEMBL_ACT_1064050
Q6B856	6.34	IC50	460	nM	CHEMBL_ACT_1076535
Q6B856	6.34	IC50	460	nM	CHEMBL_ACT_338329
P02550	6.34	IC50	460	nM	CHEMBL_ACT_357575
Q6B856	6.34	IC50	460	nM	CHEMBL_ACT_942143
Q6B856	6.34	IC50	460	nM	CHEMBL_ACT_945276
Q6B856	6.34	IC50	460	nM	CHEMBL_ACT_989569
NPC1	6.3	Potency	501.2	nM	CHEMBL_ACT_4724267
P02550	6.25	IC50	560	nM	CHEMBL_ACT_2228228
Q6B856	6.23	IC50	590	nM	CHEMBL_ACT_864792
P02550	6.22	Kd	600	nM	CHEMBL_ACT_2211929
CYP3A4	6.22	IC50	600	nM	CHEMBL_ACT_7730435
P15917	6.1	Potency	794.3	nM	CHEMBL_ACT_4358877
Q6B856	6.09	IC50	810	nM	CHEMBL_ACT_15040879

Target pathways

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

Indications & clinical

Indications

3 indications (3 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
viral infectious disease	4	MONDO:0005108	EFO:0000763
anogenital human papillomavirus infection	4	MONDO:0005647	EFO:0007147
squamous cell carcinoma	4	MONDO:0005096	EFO:0000707

Clinical trials

Total trials: 2.

Phase distribution

Phase	Trials
Not specified	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03561272	Not specified	COMPLETED	The Validation of a Novel Adherence Method for Oral Oncolytics
NCT04943809	Not specified	COMPLETED	Comparison of Visual Outcomes After Implantation of the POD FT and the POD F

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).