Ecopipam

Summary

Ecopipam (CHEMBL298406) is a phase-3 clinical-stage small molecule targeting DRD1 and DRD5; indicated across 5 conditions including tourette syndrome and pathological gambling.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 2 (DRD1, DRD5)
• Indications: 5 conditions
• Clinical trials: 16
• Chemistry: 313.8 Da · C19H20ClNO

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL298406
Name	Ecopipam
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	107930
Molecular formula	C19H20ClNO
Molecular weight	313.8
InChIKey	DMJWENQHWZZWDF-PKOBYXMFSA-N

SMILES: CN1CCC2=CC(=C(C=C2[C@@H]3[C@@H]1CCC4=CC=CC=C34)O)Cl

IUPAC name: (6aS,13bR)-11-chloro-7-methyl-5,6,6a,8,9,13b-hexahydronaphtho[1,2-a][3]benzazepin-12-ol

Also known as: Ecopipam, SCH 39166, SID144204748, SID170466462, ECOPIPAM

Parent form; salt/anhydrous children: CHEMBL1535402, CHEMBL1822916

Patent coverage: 113 distinct patent families (238 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 205 (86%) 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
DRD1	D1 receptor	Antagonist	8.3	0%	P21728
DRD5	D5 receptor	Antagonist	8.3	0%	P21918

Broader ChEMBL bioactivity targets: 6 (assay-derived). Sample: D(1B) dopamine receptor, D(1A) dopamine receptor, D(2) dopamine receptor, D(4) dopamine receptor, D(1A) dopamine receptor, D(2) dopamine receptor.

Bioactivity

ChEMBL activities: 15 potent at pChembl ≥ 5 of 15 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
DRD1	9.05	Ki	0.9	nM	CHEMBL_ACT_22411774
DRD1	8.92	Ki	1.2	nM	CHEMBL_ACT_25917637
DRD1	8.92	Ki	1.2	nM	CHEMBL_ACT_3092562
DRD1	8.92	Ki	1.2	nM	CHEMBL_ACT_3095592
DRD1	8.72	Ki	1.9	nM	CHEMBL_ACT_1047737
P18901	8.72	Ki	1.9	nM	CHEMBL_ACT_187671
DRD1	8.72	Ki	1.9	nM	CHEMBL_ACT_64028
DRD5	8.7	Ki	2	nM	CHEMBL_ACT_3092582
DRD5	8.7	Ki	2	nM	CHEMBL_ACT_3095618
P61169	6.29	Ki	514	nM	CHEMBL_ACT_187672
DRD2	6.01	Ki	980	nM	CHEMBL_ACT_3092597
DRD2	6.01	Ki	980	nM	CHEMBL_ACT_3095636
DRD2	5.91	Ki	1240	nM	CHEMBL_ACT_22411749
DRD4	5.26	Ki	5515	nM	CHEMBL_ACT_3092616
DRD4	5.26	Ki	5520	nM	CHEMBL_ACT_3095662

Target pathways

Aggregated over 2 target gene(s): DRD1, DRD5.

Top Reactome pathways

2 total, by targets touching each:

Pathway	Targets	Genes
Dopamine receptors	2	DRD1, DRD5
G alpha (s) signalling events	2	DRD1, DRD5

Dominant GO biological processes

GO term	Targets
response to amphetamine	2
adenylate cyclase-activating G protein-coupled receptor signaling pathway	2
adenylate cyclase-activating dopamine receptor signaling pathway	2
G protein-coupled dopamine receptor signaling pathway	2
mating behavior	2
transmission of nerve impulse	2
positive regulation of MAPK cascade	2
sensitization	2
phospholipase C-activating dopamine receptor signaling pathway	2
long-term synaptic depression	2
cellular response to catecholamine stimulus	2
adenylate cyclase-activating adrenergic receptor signaling pathway	2
synaptic transmission, dopaminergic	2
signal transduction	2
G protein-coupled receptor signaling pathway	2

Indications & clinical

Indications

5 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
Tourette syndrome	3	MONDO:0007661	EFO:0004895
pathological gambling	2	MONDO:0011662	EFO:0004699
restless legs syndrome	1	MONDO:0005391	EFO:0004270
Lesch-Nyhan syndrome	1	MONDO:0010298	MONDO:0010298

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

Clinical trials

Total trials: 16.

Phase distribution

Phase	Trials
PHASE2	5
PHASE1	5
PHASE3	2
PHASE1/PHASE2	2
PHASE2/PHASE3	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06021522	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Evaluate Long-term Safety of Ecopipam Tablets in Children, Adolescents and Adults With Tourette’s Disorder
NCT01751802	PHASE3	TERMINATED	Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
NCT02909088	PHASE2/PHASE3	UNKNOWN	Efficacy and Tolerability of Ecopipam in Adults With Childhood Onset Fluency Disorder (Stuttering).
NCT01215357	PHASE2	COMPLETED	Clinical Study to Determine if Ecopipam Can Reduce Urges to Gamble
NCT01244633	PHASE1/PHASE2	COMPLETED	Ecopipam Treatment of Tourette Syndrome
NCT02102698	PHASE2	COMPLETED	Ecopipam Treatment of Tourette’s Syndrome in Subjects 7-17 Years
NCT03218969	PHASE1/PHASE2	COMPLETED	Treatment of Restless Leg Syndrome (RLS) Augmentation With Ecopipam, a D1 Specific Antagonist
NCT04007991	PHASE2	COMPLETED	Ecopipam Tablets to Study Tourette’s Syndrome in Children and Adolescents
NCT04114539	PHASE2	COMPLETED	Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents - Open Label Extension
NCT04492956	PHASE2	COMPLETED	Effects of Ecopipam or Placebo in Adults With Stuttering (Speak Freely)
NCT01065558	PHASE1	COMPLETED	Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease
NCT04764851	PHASE1	TERMINATED	Drug-Drug Interaction Study to Evaluate the Effects of Ecopipam on the Pharmacokinetics of Multiple Substrates
NCT05334108	PHASE1	COMPLETED	Drug-Drug Interaction Study to Evaluate the Effects of Ecopipam on the Pharmacokinetics of Multiple Substrates for Drug Metabolism and Transport
NCT06194864	PHASE1	COMPLETED	Drug Interaction Study
NCT06669091	PHASE1	COMPLETED	Food-effect Study of Ecopipam Pharmacokinetics
NCT07093541	Not specified	AVAILABLE	Expanded Access Program for Treatment With Ecopipam for Tourette’s Disorder

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

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.

338 molecules share ≥1 primary target. Top 60 by shared-target count:

Molecule	Source	Status	Shared targets
CLOZAPINE	ChEMBL + PubChem	Phase 4 (approved)	DRD1, DRD5
DIHYDROERGOTAMINE	ChEMBL + PubChem	Phase 4 (approved)	DRD1, DRD5
OLANZAPINE	ChEMBL + PubChem	Phase 4 (approved)	DRD1, DRD5
PALIPERIDONE	ChEMBL + PubChem	Phase 4 (approved)	DRD1, DRD5
AMITRIPTYLINE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
AMOXAPINE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
APOMORPHINE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
ARIPIPRAZOLE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
BREXPIPRAZOLE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
CABERGOLINE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
CARIPRAZINE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
CHLORPROMAZINE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
CHLORPROTHIXENE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
DOPAMINE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
DOXEPIN	ChEMBL	Phase 4 (approved)	DRD1, DRD5
FLUPHENAZINE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
HALOPERIDOL	ChEMBL	Phase 4 (approved)	DRD1, DRD5
IMIPRAMINE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
KETOTIFEN	ChEMBL	Phase 4 (approved)	DRD1, DRD5
LOXAPINE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
MAPROTILINE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
PROMAZINE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
RISPERIDONE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
ROPINIROLE	ChEMBL	Phase 4 (approved)	DRD1, DRD5
TEGASEROD	ChEMBL	Phase 4 (approved)	DRD1, DRD5
NIGULDIPINE	ChEMBL	Phase 2	DRD1, DRD5
PENFLURIDOL	ChEMBL	Phase 2	DRD1, DRD5
PHENYLTOLOXAMINE	ChEMBL	Phase 2	DRD1, DRD5
RACLOPRIDE	ChEMBL	Phase 2	DRD1, DRD5
RAZPIPADON	ChEMBL	Phase 2	DRD1, DRD5
RITANSERIN	ChEMBL	Phase 2	DRD1, DRD5
TETRAHYDROPALMATINE	ChEMBL	Phase 2	DRD1, DRD5
Belzutifan	PubChem	Approved	DRD1, DRD5
Pramipexole	PubChem	Approved	DRD1, DRD5
Tiotropium Bromide Monohydrate	PubChem	Approved	DRD1, DRD5
DESLORATADINE	ChEMBL + PubChem	Phase 4 (approved)	DRD1
GENTIAN VIOLET	ChEMBL + PubChem	Phase 4 (approved)	DRD1
PIMAVANSERIN	ChEMBL + PubChem	Phase 4 (approved)	DRD1
REGORAFENIB	ChEMBL + PubChem	Phase 4 (approved)	DRD1
TAFAMIDIS	ChEMBL + PubChem	Phase 4 (approved)	DRD1
TAFENOQUINE	ChEMBL + PubChem	Phase 4 (approved)	DRD1
ACETOPHENAZINE	ChEMBL	Phase 4 (approved)	DRD1
ALECTINIB	ChEMBL	Phase 4 (approved)	DRD1
ALPROSTADIL	ChEMBL	Phase 4 (approved)	DRD1
AMIODARONE	ChEMBL	Phase 4 (approved)	DRD1
AMLODIPINE	ChEMBL	Phase 4 (approved)	DRD1
ASENAPINE	ChEMBL	Phase 4 (approved)	DRD1
ASTEMIZOLE	ChEMBL	Phase 4 (approved)	DRD1
AZELASTINE	ChEMBL	Phase 4 (approved)	DRD1
BAZEDOXIFENE	ChEMBL	Phase 4 (approved)	DRD1
BENPERIDOL	ChEMBL	Phase 4 (approved)	DRD1
BENZIODARONE	ChEMBL	Phase 4 (approved)	DRD1
BENZTROPINE	ChEMBL	Phase 4 (approved)	DRD1
BENZYDAMINE	ChEMBL	Phase 4 (approved)	DRD1
BEXAROTENE	ChEMBL	Phase 4 (approved)	DRD1
BITHIONOL	ChEMBL	Phase 4 (approved)	DRD1
BOSUTINIB	ChEMBL	Phase 4 (approved)	DRD1
BROMOCRIPTINE	ChEMBL	Phase 4 (approved)	DRD1
BROMODIPHENHYDRAMINE	ChEMBL	Phase 4 (approved)	DRD1
BROMPHENIRAMINE	ChEMBL	Phase 4 (approved)	DRD1