Eprenetapopt

Summary

Eprenetapopt (CHEMBL3186011) is a phase-3 clinical-stage small molecule; indicated across 6 conditions including myelodysplastic syndrome and ovarian cancer; with CIViC clinical evidence for 4 variant-indication associations (e.g. TP53 Mutation in myelodysplastic syndrome).

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 6 conditions
• Clinical trials: 11
• Precision-oncology evidence (CIViC): 4 variant–indication associations
• Chemistry: 199.25 Da · C10H17NO3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3186011
Name	Eprenetapopt
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	52918385
Molecular formula	C10H17NO3
Molecular weight	199.25
InChIKey	BGBNULCRKBVAKL-UHFFFAOYSA-N

SMILES: COCC1(C(=O)C2CCN1CC2)CO

IUPAC name: 2-(hydroxymethyl)-2-(methoxymethyl)-1-azabicyclo[2.2.2]octan-3-one

Also known as: Apr 246, Apr-246, APR-246, Eprenetapopt, PRIMA-1Met, SID174007096, EPRENETAPOPT

Patent coverage: 181 distinct patent families (404 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 403 (100%) 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

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

6 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
myelodysplastic syndrome	3	MONDO:0018881	EFO:0000198
ovarian cancer	2	MONDO:0008170	MONDO:0008170
mantle cell lymphoma	2	MONDO:0018876	EFO:1001469
melanoma	1	MONDO:0005105	EFO:0000756
carcinoma of esophagus	1	MONDO:0019086	EFO:0002916
neoplasm	1	MONDO:0005070	EFO:0000616

Clinical trials

Total trials: 11.

Phase distribution

Phase	Trials
PHASE1/PHASE2	5
PHASE2	3
PHASE1	2
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03745716	PHASE3	COMPLETED	APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS)
NCT02098343	PHASE1/PHASE2	COMPLETED	p53 Suppressor Activation in Recurrent High Grade Serous Ovarian Cancer, a Phase Ib/II Study of Systemic Carboplatin Combination Chemotherapy With or Without APR-246
NCT02999893	PHASE1/PHASE2	TERMINATED	A Study of APR-246 in Oesophageal Cancer
NCT03072043	PHASE1/PHASE2	COMPLETED	Phase 1b/2 Safety and Efficacy of APR-246 w/Azacitidine for tx of TP53 Mutant Myeloid Neoplasms
NCT03268382	PHASE2	COMPLETED	p53 Activation in Platinum-Resistant High Grade Serous Ovarian Cancer, a Study of PLD With APR-246
NCT03391050	PHASE1/PHASE2	TERMINATED	A Study of APR-246 in Combination With Dabrafenib in Resistant Patients With BRAF V600 Mutant Melanoma
NCT03588078	PHASE1/PHASE2	UNKNOWN	Study of the Safety and Efficacy of APR-246 in Combination With Azacitidine
NCT03931291	PHASE2	COMPLETED	APR-246 in Combination With Azacitidine for TP53 Mutated AML (Acute Myeloid Leukemia) or MDS (Myelodysplastic Syndromes) Following Allogeneic Stem Cell Transplant
NCT04990778	PHASE2	WITHDRAWN	Venetoclax and Eprenetapopt for the Treatment of Relapsed of Refractory Mantle Cell Lymphoma
NCT00900614	PHASE1	COMPLETED	Safety Study of APR-246 in Patients With Refractory Hematologic Cancer or Prostate Cancer
NCT04214860	PHASE1	COMPLETED	APR-246 in Combination With Venetoclax and Azacitidine in TP53-Mutant Myeloid Malignancies

Clinical evidence (CIViC)

Variant × indication × effect (4 predictive associations from 4 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
TP53 Mutation	Myelodysplastic Syndrome	Sensitivity/Response	Azacitidine + Eprenetapopt	CIViC B	EID12029
TP53 Mutation	Cancer	Sensitivity/Response	Eprenetapopt + Pembrolizumab	CIViC B	EID12033
TP53 Mutation	Ovarian Cancer	Sensitivity/Response	Eprenetapopt + Cisplatin	CIViC D	EID12010
TP53 Mutation	Ovarian Cancer	Sensitivity/Response	Eprenetapopt + Doxorubicin	CIViC D	EID12011

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).