Opevesostat

Summary

Opevesostat (CHEMBL5314533) is a phase-3 clinical-stage small molecule targeting CYP11A1; indicated across 3 conditions including metastatic prostate carcinoma and prostate adenocarcinoma.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (CYP11A1)
• Indications: 3 conditions
• Clinical trials: 10
• Chemistry: 418.5 Da · C21H26N2O5S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL5314533
Name	Opevesostat
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	135151902
Molecular formula	C21H26N2O5S
Molecular weight	418.5
InChIKey	LHVKCOBGLZGRQZ-UHFFFAOYSA-N

SMILES: CS(=O)(=O)N1CCC(CC1)COC2=COC(=CC2=O)CN3CC4=CC=CC=C4C3

IUPAC name: 2-(1,3-dihydroisoindol-2-ylmethyl)-5-[(1-methylsulfonylpiperidin-4-yl)methoxy]pyran-4-one

Also known as: Odm-208, ODM208, Opevesostat, OPEVESOSTAT

Patent coverage: 13 distinct patent families (52 SureChEMBL compound mentions), from 2 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

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
CYP11A1	CYP11A1	Inhibition	6.97	0.2%	P05108

Bioactivity

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

Target pathways

Aggregated over 1 target gene(s): CYP11A1.

Top Reactome pathways

3 total, by targets touching each:

Pathway	Targets	Genes
Pregnenolone biosynthesis	1	CYP11A1
Endogenous sterols	1	CYP11A1
Defective CYP11A1 causes AICSR	1	CYP11A1

Dominant GO biological processes

GO term	Targets
C21-steroid hormone biosynthetic process	1
glucocorticoid biosynthetic process	1
cholesterol metabolic process	1
sterol metabolic process	1
cortisol metabolic process	1
vitamin D metabolic process	1
cellular response to peptide hormone stimulus	1
steroid hormone biosynthetic process	1
alcohol metabolic process	1
lipid metabolic process	1
steroid biosynthetic process	1
steroid metabolic process	1
C21-steroid hormone metabolic process	1

Indications & clinical

Indications

3 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
metastatic prostate carcinoma	3	MONDO:0004956	EFO:0000196
prostate adenocarcinoma	3	MONDO:0005082	EFO:0000673

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

Clinical trials

Total trials: 10.

Phase distribution

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

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06136624	PHASE3	RECRUITING	Study of Opevesostat (MK-5684) Versus Alternative NHA in mCRPC (MK-5684-003)
NCT06136650	PHASE3	RECRUITING	A Study of Opevesostat (MK-5684) Versus Alternative Next-generation Hormonal Agent (NHA) in Metastatic Castration-resistant Prostate Cancer (mCRPC) Post One NHA (MK-5684-004)
NCT03436485	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Safety and Pharmacokinetics of ODM-208 in Patients With Metastatic Castration-resistant Prostate Cancer
NCT06353386	PHASE1/PHASE2	RECRUITING	Substudy 01A: Safety and Efficacy of Opevesostat (MK-5684)-Based Treatment Combinations or Opevesostat Alone in Participants With Metastatic Castration-resistant Prostate Cancer (mCRPC) (MK-5684-01A)
NCT06979596	PHASE2	RECRUITING	A Study of MK-5684 in People With Certain Solid Tumors (MK-5684-015/OMAHA-015)
NCT07548606	PHASE1	ACTIVE_NOT_RECRUITING	A Drug-Drug Interaction Study of Itraconazole and Opevesostat (MK-5684) in Healthy Adult Male Participants (MK-5684-017)
NCT06104449	PHASE1	COMPLETED	A Study of Opevesostat (MK-568)4 in Japanese Participants With Metastatic Castration-resistant Prostate Cancer (mCRPC) (MK-5684-005)
NCT06136598	PHASE1	COMPLETED	A Study of Opevesostat (MK-5684) in China Participants With Metastatic Castration-Resistant Prostate Cancer (mCRPC) (MK-5684-001)
NCT06633419	PHASE1	COMPLETED	A Drug-Drug Interaction Study of Carbamazepine and Opevesostat (MK-5684) in Healthy Adult Male Participants (MK-5684-012)
NCT06860243	PHASE1	COMPLETED	A Study to Evaluate Opevesostat (MK-5684) in Male Participants With Moderate Hepatic Impairment (MK-5684-009)

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

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

1 molecules share ≥1 primary target. Top 1 by shared-target count:

Molecule	Source	Status	Shared targets
AMINOGLUTETHIMIDE	ChEMBL	Phase 4 (approved)	CYP11A1