Rubitecan

Summary

Rubitecan (CHEMBL77305) is a phase-3 clinical-stage small-molecule antineoplastic agent; indicated across 12 conditions including exocrine pancreatic carcinoma and cutaneous melanoma.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 12 conditions
• Clinical trials: 18
• Chemistry: 393.3 Da · C20H15N3O6

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL77305
Name	Rubitecan
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	472335
ChEBI	CHEBI:90225
Molecular formula	C20H15N3O6
Molecular weight	393.3
InChIKey	VHXNKPBCCMUMSW-FQEVSTJZSA-N

SMILES: CC[C@@]1(C2=C(COC1=O)C(=O)N3CC4=CC5=C(C=CC=C5[N+](=O)[O-])N=C4C3=C2)O

IUPAC name: (19S)-19-ethyl-19-hydroxy-8-nitro-17-oxa-3,13-diazapentacyclo[11.8.0.02,11.04,9.015,20]henicosa-1(21),2,4(9),5,7,10,15(20)-heptaene-14,18-dione

ChEBI definition: A pyranoindolizinoquinoline that is camptothecin in which the hydrogen at position 9 has been replaced by a nitro group. It is a prodrug for 9-aminocamptothecin.

Pharmacological roles (ChEBI): antineoplastic agent, EC 5.99.1.2 (DNA topoisomerase) inhibitor, prodrug.

Also known as: RFS 2000, RFS-2000, RFS2000, Rubitecan, SID50113013, RUBITECAN, SID144206150, rubitecan, C0088758

Patent coverage: 5,820 distinct patent families (22,927 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 21,921 (96%) 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: 7 (assay-derived). Sample: Nuclear receptor ROR-gamma, ATP-binding cassette sub-family C member 4, Acetylcholinesterase, Prostaglandin G/H synthase 1, Huntingtin, ATP-binding cassette sub-family C member 2, ATP-binding cassette sub-family C member 3.

Bioactivity

ChEMBL activities: 4 potent at pChembl ≥ 5 of 8 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
P51450	7.05	Potency	89.1	nM	CHEMBL_ACT_4777791
P51450	6.2	Potency	631	nM	CHEMBL_ACT_4072132
ACHE	5.44	AC50	3650	nM	CHEMBL_ACT_25142467
ABCC4	5.29	IC50	5100	nM	CHEMBL_ACT_18130907

Target pathways

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

Indications & clinical

Indications

12 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
exocrine pancreatic carcinoma	3	MONDO:0005192	EFO:0002618
cutaneous melanoma	2	MONDO:0005012	EFO:0000389
central nervous system neoplasm	2	MONDO:0006130	EFO:1000158
peritoneal neoplasm	2	MONDO:0006901	MONDO:0002087
breast neoplasm	2	MONDO:0021100	MONDO:0007254
ovarian cancer	2	MONDO:0008170	MONDO:0008170
lung neoplasm	2	MONDO:0021117	MONDO:0008903
endometrium neoplasm	2	MONDO:0021251	MONDO:0011962
colorectal neoplasm	2	MONDO:0005335	MONDO:0005575
neoplasm	1	MONDO:0005070	EFO:0000616

2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 18.

Phase distribution

Phase	Trials
PHASE2	12
PHASE3	3
PHASE1	2
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00005869	PHASE3	UNKNOWN	Nitrocamptothecin Compared With Gemcitabine in Treating Patients With Unresectable Locally Advanced or Metastatic Pancreatic Cancer
NCT00005870	PHASE3	UNKNOWN	Nitrocamptothecin Compared With Other Chemotherapy in Treating Patients With Recurrent or Refractory Cancer of the Pancreas
NCT00005871	PHASE3	UNKNOWN	Nitrocamptothecin or Fluorouracil in Treating Patients With Recurrent or Refractory Pancreatic Cancer
NCT00113256	PHASE2/PHASE3	TERMINATED	Orathecin + Gemcitabine Versus Placebo + Gemcitabine in Chemonaive Non-Resectable Pancreatic Cancer
NCT00005826	PHASE2	COMPLETED	Nitrocamptothecin in Treating Patients With Glioblastoma Multiforme
NCT00005872	PHASE2	COMPLETED	Nitrocamptothecin in Treating Patients With Recurrent Non-small Cell Lung Cancer
NCT00005873	PHASE2	COMPLETED	Nitrocamptothecin in Treating Patients With Locally Recurrent or Metastatic Breast Cancer
NCT00005874	PHASE2	COMPLETED	Nitrocamptothecin in Treating Patients With Soft Tissue Sarcomas
NCT00005875	PHASE2	COMPLETED	Nitrocamptothecin in Treating Patients With Metastatic Melanoma
NCT00005876	PHASE2	UNKNOWN	Nitrocamptothecin in Treating Patients With Locally Advanced or Metastatic Stomach Cancer That Cannot Be Removed During Surgery
NCT00005877	PHASE2	UNKNOWN	Nitrocamptothecin in Treating Patients With Advanced or Recurrent Colorectal Cancer
NCT00006026	PHASE2	COMPLETED	Nitrocamptothecin in Treating Patients With Metastatic Cancer of the Urinary Tract
NCT00006082	PHASE2	COMPLETED	Nitrocamptothecin in Treating Patients With Advanced Small Cell Lung Cancer
NCT00006230	PHASE2	COMPLETED	Nitrocamptothecin in Treating Patients With Advanced Ovarian Cancer
NCT00006267	PHASE2	WITHDRAWN	Nitrocamptothecin in Treating Patients With Recurrent or Metastatic Ovarian Epithelial or Primary Peritoneal Cancer
NCT00250068	PHASE2	COMPLETED	Study of Aerosolized Liposomal 9-Nitro-20 (S)- Camptothecin (L9NC)
NCT00006047	PHASE1	COMPLETED	Combination Therapy With Oral 9-Nitrocamptothecin & Oral Etoposide
NCT00113113	PHASE1	COMPLETED	Study of Rubitecan in Cancer Patients With Renal or Hepatic Dysfunction

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).