Piflufolastat

Summary

Piflufolastat (CHEMBL4299851) is an approved small molecule; indicated across 2 conditions including prostate adenocarcinoma and hepatocellular carcinoma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• Indications: 2 conditions
• Clinical trials: 4
• Chemistry: 442.4 Da · C18H23FN4O8

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4299851
Name	Piflufolastat
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	71532123
Molecular formula	C18H23FN4O8
Molecular weight	442.4
InChIKey	OLWVRJUNLXQDSP-RYUDHWBXSA-N

SMILES: C1=CC(=NC=C1C(=O)NCCCC[C@@H](C(=O)O)NC(=O)N[C@@H](CCC(=O)O)C(=O)O)F

IUPAC name: (2S)-2-[[(1S)-1-carboxy-5-[(6-fluoropyridine-3-carbonyl)amino]pentyl]carbamoylamino]pentanedioic acid

Also known as: Dcfpyl, Piflufolastat, PIFLUFOLASTAT

Parent form; salt/anhydrous children: CHEMBL4297334

Patent coverage: 111 distinct patent families (275 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 273 (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: 1 (assay-derived). Sample: Glutamate carboxypeptidase 2.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 2 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
FOLH1	8.81	IC50	1.56	nM	CHEMBL_ACT_19061738
FOLH1	7.81	Ki	15.6	nM	CHEMBL_ACT_28230719

Target pathways

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

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
prostate adenocarcinoma	2	MONDO:0005082	EFO:0000673
hepatocellular carcinoma	2	MONDO:0007256	EFO:0000182

Clinical trials

Total trials: 4.

Phase distribution

Phase	Trials
PHASE2	2
PHASE4	1
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05919329	PHASE4	RECRUITING	Evaluation of the Change in PSMA Expression in Prostate Cancer in Response to Hormonal Therapy
NCT04734184	PHASE3	UNKNOWN	A Prospective Study on 18F-DCFPyL PET/CT Imaging in Biochemical Recurrence of Prostate Cancer
NCT03585114	PHASE2	ACTIVE_NOT_RECRUITING	Prostate Cancer Monitoring Using [18F]DCFPyL and Blood Based Biomarkers
NCT04700332	PHASE2	RECRUITING	PyL in Patients With High Risk and Biochemically Recurrent Prostate 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).