Obatoclax

Summary

Obatoclax (CHEMBL408194) is a phase-3 clinical-stage small molecule targeting BCL2, BCL2L1, and MCL1; indicated across 2 conditions including acute myeloid leukemia and small cell lung carcinoma.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 3 (BCL2, BCL2L1, MCL1)
• Indications: 2 conditions
• Clinical trials: 2
• Chemistry: 317.4 Da · C20H19N3O

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL408194
Name	Obatoclax
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	11404337
Molecular formula	C20H19N3O
Molecular weight	317.4
InChIKey	CVCLJVVBHYOXDC-IAZSKANUSA-N

SMILES: CC1=CC(=C(N1)/C=C\2/C(=C/C(=C/3\C=C4C=CC=CC4=N3)/N2)OC)C

IUPAC name: (2Z)-2-[(5Z)-5-[(3,5-dimethyl-1H-pyrrol-2-yl)methylidene]-4-methoxypyrrol-2-ylidene]indole

Also known as: Obatoclax, obatoclax, OBATOCLAX

Parent form; salt/anhydrous children: CHEMBL2107358

Patent coverage: 1,130 distinct patent families (2,914 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 2,847 (98%) 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

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
BCL2	BCL2 apoptosis regulator	Antagonist	5.96	3.3%	P10415
BCL2L1	Bcl-2-like 1	Antagonist	5.33	86.5%	Q07817
MCL1	MCL1 apoptosis regulator, BCL2 family member	Antagonist	5.54	63.7%	Q07820

Broader ChEMBL bioactivity targets: 8 (assay-derived). Sample: Adenosine receptor A1, Bcl2-associated agonist of cell death, Induced myeloid leukemia cell differentiation protein Mcl-1, Bcl-2-like protein 1, Bcl-2-like protein 2, Apoptosis regulator Bcl-2, Bcl-2-like protein 10, Bcl-2-related protein A1.

Bioactivity

ChEMBL activities: 21 potent at pChembl ≥ 5 of 21 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
BCL2	6.7	Ki	200	nM	CHEMBL_ACT_29155650
BCL2	6.52	EC50	300	nM	CHEMBL_ACT_12100874
MCL1	6.3	EC50	500	nM	CHEMBL_ACT_12100873
BCL2A1	6.22	EC50	600	nM	CHEMBL_ACT_12100872
BCL2L1	6.05	EC50	900	nM	CHEMBL_ACT_12100871
BAD	6	Ki	1000	nM	CHEMBL_ACT_2150678
MCL1	6	Ki	1000	nM	CHEMBL_ACT_2150679
BCL2L2-PABPN1	6	Ki	1000	nM	CHEMBL_ACT_2150680
BCL2A1	6	Ki	1000	nM	CHEMBL_ACT_2150681
BCL2L10	6	Ki	1000	nM	CHEMBL_ACT_2150682
BCL2	5.96	IC50	1110	nM	CHEMBL_ACT_16623378
BCL2	5.96	IC50	1110	nM	CHEMBL_ACT_18390898
BCL2	5.96	Ki	1110	nM	CHEMBL_ACT_19235572
MCL1	5.7	Ki	2000	nM	CHEMBL_ACT_19235575
MCL1	5.54	IC50	2900	nM	CHEMBL_ACT_16623377
MCL1	5.54	IC50	2900	nM	CHEMBL_ACT_18390904
BCL2L1	5.33	IC50	4690	nM	CHEMBL_ACT_16623379
BCL2L1	5.33	IC50	4690	nM	CHEMBL_ACT_18390901
BCL2L1	5.33	Ki	4690	nM	CHEMBL_ACT_19235573
ADORA1	5.3	Ki	5000	nM	CHEMBL_ACT_19235576
BCL2L2-PABPN1	5.15	Ki	7010	nM	CHEMBL_ACT_19235574

Target pathways

Aggregated over 3 target gene(s): BCL2, BCL2L1, MCL1.

Top Reactome pathways

47 total, by targets touching each:

Pathway	Targets	Genes
Cytokine Signaling in Immune system	3	BCL2, BCL2L1, MCL1
Immune System	3	BCL2, BCL2L1, MCL1
Signaling by Interleukins	3	BCL2, BCL2L1, MCL1
Interleukin-4 and Interleukin-13 signaling	3	BCL2, BCL2L1, MCL1
Apoptosis	2	BCL2, BCL2L1
Intrinsic Pathway for Apoptosis	2	BCL2, BCL2L1
BH3-only proteins associate with and inactivate anti-apoptotic BCL-2 members	2	BCL2, BCL2L1
Signal Transduction	2	BCL2, BCL2L1
Disease	2	BCL2L1, MCL1
Innate Immune System	2	BCL2, BCL2L1
Nucleotide-binding domain, leucine rich repeat containing receptor (NLR) signaling pathways	2	BCL2, BCL2L1
Cellular responses to stress	2	BCL2, BCL2L1
Programmed Cell Death	2	BCL2, BCL2L1
Diseases of signal transduction by growth factor receptors and second messengers	2	BCL2L1, MCL1
Inflammasomes	2	BCL2, BCL2L1
The NLRP1 inflammasome	2	BCL2, BCL2L1
Cellular responses to stimuli	2	BCL2, BCL2L1
Cellular response to chemical stress	2	BCL2, BCL2L1
KEAP1-NFE2L2 pathway	2	BCL2, BCL2L1
Nuclear events mediated by NFE2L2	2	BCL2, BCL2L1
NFE2L2 regulating tumorigenic genes	2	BCL2, BCL2L1
Activation of BAD and translocation to mitochondria	1	BCL2
Activation of BH3-only proteins	1	BCL2
Developmental Biology	1	BCL2
Infectious disease	1	BCL2L1
RAF/MAP kinase cascade	1	BCL2L1
MAPK family signaling cascades	1	BCL2L1
MAPK1/MAPK3 signaling	1	BCL2L1
ESR-mediated signaling	1	BCL2
Signaling by Nuclear Receptors	1	BCL2

Dominant GO biological processes

GO term	Targets
release of cytochrome c from mitochondria	3
apoptotic process	3
intrinsic apoptotic signaling pathway in response to DNA damage	3
negative regulation of autophagy	3
response to cytokine	3
positive regulation of apoptotic process	3
negative regulation of apoptotic process	3
extrinsic apoptotic signaling pathway in absence of ligand	3
negative regulation of anoikis	3
negative regulation of extrinsic apoptotic signaling pathway in absence of ligand	3
regulation of apoptotic process	3
regulation of intracellular signal transduction	3
regulation of apoptotic signaling pathway	3
ovarian follicle development	2
DNA damage response	2

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
acute myeloid leukemia	2	MONDO:0018874	EFO:0000222
small cell lung carcinoma	1	MONDO:0008433	EFO:0000702

Clinical trials

Total trials: 2.

Phase distribution

Phase	Trials
PHASE1/PHASE2	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00682981	PHASE1/PHASE2	COMPLETED	A Phase I/II Study of Carboplatin and Etoposide With or Without Obatoclax in Extensive-stage Small Cell Lung Cancer (ES-SCLC)
NCT00684918	PHASE2	COMPLETED	Study of Obatoclax in Previously Untreated Acute Myeloid Leukemia (AML)

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.

19 molecules share ≥1 primary target. Top 19 by shared-target count:

Molecule	Source	Status	Shared targets
VENETOCLAX	ChEMBL	Phase 4 (approved)	BCL2, BCL2L1, MCL1
EPIGALOCATECHIN GALLATE	ChEMBL	Phase 3	BCL2, BCL2L1, MCL1
GOSSYPOL	ChEMBL	Phase 3	BCL2, BCL2L1, MCL1
NAVITOCLAX	ChEMBL	Phase 3	BCL2, BCL2L1, MCL1
METHYSERGIDE	ChEMBL	Phase 4 (approved)	BCL2L1, MCL1
SONROTOCLAX	ChEMBL	Phase 3	BCL2, BCL2L1
APOMORPHINE	ChEMBL	Phase 4 (approved)	MCL1
BITHIONOL	ChEMBL	Phase 4 (approved)	MCL1
COUMARIN	ChEMBL	Phase 4 (approved)	MCL1
HEXACHLOROPHENE	ChEMBL	Phase 4 (approved)	MCL1
IXABEPILONE	ChEMBL	Phase 4 (approved)	BCL2
LIOTHYRONINE	ChEMBL	Phase 4 (approved)	MCL1
OMEPRAZOLE	ChEMBL	Phase 4 (approved)	MCL1
PROCHLORPERAZINE	ChEMBL	Phase 4 (approved)	MCL1
ASARETOCLAX	ChEMBL	Phase 2	BCL2L1
CHLORCYCLIZINE	ChEMBL	Phase 2	BCL2
FORMONONETIN	ChEMBL	Phase 2	BCL2
HYMECROMONE	ChEMBL	Phase 2	MCL1
LACUTOCLAX	ChEMBL	Phase 2	BCL2