Melphalan Flufenamide

Summary

Melphalan Flufenamide (CHEMBL4303060) is an approved small molecule (ATC L01AA10); indicated across 2 conditions including neoplasm and plasma cell myeloma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L01AA10
• Indications: 2 conditions
• Clinical trials: 6
• Chemistry: 498.4 Da · C24H30Cl2FN3O3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4303060
Name	Melphalan Flufenamide
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	9935639
ATC	L01AA10
Molecular formula	C24H30Cl2FN3O3
Molecular weight	498.4
InChIKey	YQZNKYXGZSVEHI-VXKWHMMOSA-N

SMILES: CCOC(=O)[C@H](CC1=CC=C(C=C1)F)NC(=O)[C@H](CC2=CC=C(C=C2)N(CCCl)CCCl)N

IUPAC name: ethyl (2S)-2-[[(2S)-2-amino-3-[4-[bis(2-chloroethyl)amino]phenyl]propanoyl]amino]-3-(4-fluorophenyl)propanoate

Also known as: J 1 (PRODRUG), J-1, J-1 (PRODRUG), J1, J1 (PRODRUG), Melfalan flufenamida, Melphalan flufenamide, Melflufen, PRODRUG J 1, PRODRUG J-1, PRODRUG J1, MELPHALAN FLUFENAMIDE

Parent form; salt/anhydrous children: CHEMBL4297403

Patent coverage: 91 distinct patent families (220 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

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

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
neoplasm	4	MONDO:0005070	EFO:0000616
plasma cell myeloma	1	MONDO:0009693	EFO:0001378

Clinical trials

Total trials: 6.

Phase distribution

Phase	Trials
PHASE3	2
PHASE2	2
PHASE1/PHASE2	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03151811	PHASE3	TERMINATED	A Study of Melphalan Flufenamide (Melflufen)-Dex or Pomalidomide-dex for RRMM Patients Refractory to Lenalidomide
NCT04649060	PHASE3	TERMINATED	Study of Melflufen (Melphalan Flufenamide) in Combination With Daratumumab in Relapsed-Refractory Multiple Myeloma
NCT01897714	PHASE1/PHASE2	TERMINATED	Safety and Efficacy of Melflufen and Dexamethasone in Relapsed and/or Relapsed-Refractory Multiple Myeloma Patients
NCT03639610	PHASE2	TERMINATED	PK of Melphalan During Treatment With Melflufen and Dexamethasone in Patients With RRMM and Impaired Renal Function
NCT06682637	PHASE2	WITHDRAWN	Melflufen for Elderly Patients With Relapsed Myeloma
NCT04534322	Not specified	APPROVED_FOR_MARKETING	Expanded Access Program for Melphalan Flufenamide (Melflufen) in Triple Class Refractory Multiple Myeloma

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).