Macimorelin

Summary

Macimorelin (CHEMBL278623) is an approved small molecule (ATC V04CD06) targeting GHSR; indicated across 2 conditions including pituitary dwarfism.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: V04CD06
• Targets: 1 (GHSR)
• Indications: 2 conditions
• Clinical trials: 4
• Chemistry: 474.6 Da · C26H30N6O3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL278623
Name	Macimorelin
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	9804938
ATC	V04CD06
Molecular formula	C26H30N6O3
Molecular weight	474.6
InChIKey	UJVDJAPJQWZRFR-DHIUTWEWSA-N

SMILES: CC(C)(C(=O)N[C@H](CC1=CNC2=CC=CC=C21)C(=O)N[C@H](CC3=CNC4=CC=CC=C43)NC=O)N

IUPAC name: 2-amino-N-[(2R)-1-[[(1R)-1-formamido-2-(1H-indol-3-yl)ethyl]amino]-3-(1H-indol-3-yl)-1-oxopropan-2-yl]-2-methylpropanamide

Also known as: AEZS-130, Ard-07, ARD-07, ARD07, EP-01572, EP-1572, EP01572, EP1572, JMV 1843, JMV-1843, Macimorelin, Macimorelina

Parent form; salt/anhydrous children: CHEMBL2364617

Patent coverage: 84 distinct patent families (196 SureChEMBL compound mentions), from 1 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
GHSR	ghrelin receptor	Agonist	7.81	0.2%	Q92847

Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Mu-type opioid receptor, Growth hormone secretagogue receptor type 1.

Bioactivity

ChEMBL activities: 3 potent at pChembl ≥ 5 of 3 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
GHSR	7.64	IC50	22.9	nM	CHEMBL_ACT_1149212
GHSR	6.91	IC50	123	nM	CHEMBL_ACT_1149213
OPRM1	6.12	AC50	750	nM	CHEMBL_ACT_25158077

Target pathways

Aggregated over 1 target gene(s): GHSR.

Top Reactome pathways

2 total, by targets touching each:

Pathway	Targets	Genes
Peptide ligand-binding receptors	1	GHSR
G alpha (q) signalling events	1	GHSR

Dominant GO biological processes

GO term	Targets
G protein-coupled receptor signaling pathway	1
spermatogenesis	1
learning or memory	1
actin polymerization or depolymerization	1
adult feeding behavior	1
response to hormone	1
hormone-mediated signaling pathway	1
negative regulation of norepinephrine secretion	1
growth hormone secretion	1
response to food	1
negative regulation of appetite	1
positive regulation of appetite	1
response to follicle-stimulating hormone	1
response to estradiol	1
negative regulation of interleukin-1 beta production	1

Indications & clinical

Indications

1 disease in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
pituitary dwarfism	3	MONDO:0006909	EFO:1001109

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

Clinical trials

Total trials: 4.

Phase distribution

Phase	Trials
PHASE3	2
PHASE2	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02558829	PHASE3	COMPLETED	Validation of Macimorelin as a Test for Adult Growth Hormone Deficiency
NCT04786873	PHASE3	COMPLETED	A Research Study of How Well Macimorelin Works to Find Out if Children Have a Lack of Growth Hormone and How Safe it is
NCT01614990	PHASE2	COMPLETED	Pilot Clinical Trial of Repeated Doses of Macimorelin to Assess Safety and Efficacy in Patients With Cancer Cachexia
NCT03844217	Not specified	COMPLETED	Copeptin After an Oral Stimulation With Macimorelin in Healthy Volunteers

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.

168 molecules share ≥1 primary target. Top 100 by shared-target count:

Molecule	Source	Status	Shared targets
ATAZANAVIR	ChEMBL + PubChem	Phase 4 (approved)	GHSR
CINACALCET	ChEMBL + PubChem	Phase 4 (approved)	GHSR
CRIZOTINIB	ChEMBL + PubChem	Phase 4 (approved)	GHSR
DIHYDROERGOTAMINE	ChEMBL + PubChem	Phase 4 (approved)	GHSR
LINAGLIPTIN	ChEMBL + PubChem	Phase 4 (approved)	GHSR
TEGASEROD	ChEMBL + PubChem	Phase 4 (approved)	GHSR
AMIODARONE	ChEMBL	Phase 4 (approved)	GHSR
ARIPIPRAZOLE	ChEMBL	Phase 4 (approved)	GHSR
ASENAPINE	ChEMBL	Phase 4 (approved)	GHSR
ASTEMIZOLE	ChEMBL	Phase 4 (approved)	GHSR
ATRACURIUM	ChEMBL	Phase 4 (approved)	GHSR
AZELASTINE	ChEMBL	Phase 4 (approved)	GHSR
BAZEDOXIFENE	ChEMBL	Phase 4 (approved)	GHSR
BENFLUOREX	ChEMBL	Phase 4 (approved)	GHSR
BENPERIDOL	ChEMBL	Phase 4 (approved)	GHSR
BEPRIDIL	ChEMBL	Phase 4 (approved)	GHSR
BICALUTAMIDE	ChEMBL	Phase 4 (approved)	GHSR
BOSUTINIB	ChEMBL	Phase 4 (approved)	GHSR
BROMOCRIPTINE	ChEMBL	Phase 4 (approved)	GHSR
BROMPERIDOL	ChEMBL	Phase 4 (approved)	GHSR
BUCLIZINE	ChEMBL	Phase 4 (approved)	GHSR
CARVEDILOL	ChEMBL	Phase 4 (approved)	GHSR
CHLORHEXIDINE	ChEMBL	Phase 4 (approved)	GHSR
CHLORPROMAZINE	ChEMBL	Phase 4 (approved)	GHSR
CHLORPROTHIXENE	ChEMBL	Phase 4 (approved)	GHSR
CLEMASTINE	ChEMBL	Phase 4 (approved)	GHSR
CLOFAZIMINE	ChEMBL	Phase 4 (approved)	GHSR
CLOMIPRAMINE	ChEMBL	Phase 4 (approved)	GHSR
DARIFENACIN	ChEMBL	Phase 4 (approved)	GHSR
DAUNORUBICIN	ChEMBL	Phase 4 (approved)	GHSR
DESERPIDINE	ChEMBL	Phase 4 (approved)	GHSR
DESIPRAMINE	ChEMBL	Phase 4 (approved)	GHSR
DIBUCAINE	ChEMBL	Phase 4 (approved)	GHSR
DICYCLOMINE	ChEMBL	Phase 4 (approved)	GHSR
DIETHYLSTILBESTROL	ChEMBL	Phase 4 (approved)	GHSR
DILTIAZEM	ChEMBL	Phase 4 (approved)	GHSR
DOMPERIDONE	ChEMBL	Phase 4 (approved)	GHSR
DULOXETINE	ChEMBL	Phase 4 (approved)	GHSR
EBASTINE	ChEMBL	Phase 4 (approved)	GHSR
FEDRATINIB	ChEMBL	Phase 4 (approved)	GHSR
FENTANYL	ChEMBL	Phase 4 (approved)	GHSR
FLUNITRAZEPAM	ChEMBL	Phase 4 (approved)	GHSR
FLURAZEPAM	ChEMBL	Phase 4 (approved)	GHSR
FLUSPIRILENE	ChEMBL	Phase 4 (approved)	GHSR
HALOPERIDOL	ChEMBL	Phase 4 (approved)	GHSR
IDARUBICIN	ChEMBL	Phase 4 (approved)	GHSR
INDOCYANINE GREEN ACID FORM	ChEMBL	Phase 4 (approved)	GHSR
LANSOPRAZOLE	ChEMBL	Phase 4 (approved)	GHSR
LASOFOXIFENE	ChEMBL	Phase 4 (approved)	GHSR
LIOTHYRONINE	ChEMBL	Phase 4 (approved)	GHSR
LOFEPRAMINE	ChEMBL	Phase 4 (approved)	GHSR
MECLIZINE	ChEMBL	Phase 4 (approved)	GHSR
MEFLOQUINE	ChEMBL	Phase 4 (approved)	GHSR
NAFARELIN	ChEMBL	Phase 4 (approved)	GHSR
NEBIVOLOL	ChEMBL	Phase 4 (approved)	GHSR
NERATINIB	ChEMBL	Phase 4 (approved)	GHSR
NIMESULIDE	ChEMBL	Phase 4 (approved)	GHSR
NITAZOXANIDE	ChEMBL	Phase 4 (approved)	GHSR
NOSCAPINE	ChEMBL	Phase 4 (approved)	GHSR
OSIMERTINIB	ChEMBL	Phase 4 (approved)	GHSR
PASIREOTIDE	ChEMBL	Phase 4 (approved)	GHSR
PERHEXILINE	ChEMBL	Phase 4 (approved)	GHSR
PIMOZIDE	ChEMBL	Phase 4 (approved)	GHSR
PONATINIB	ChEMBL	Phase 4 (approved)	GHSR
PRENYLAMINE	ChEMBL	Phase 4 (approved)	GHSR
PROCHLORPERAZINE	ChEMBL	Phase 4 (approved)	GHSR
PROPAFENONE	ChEMBL	Phase 4 (approved)	GHSR
PYRVINIUM	ChEMBL	Phase 4 (approved)	GHSR
RIFAXIMIN	ChEMBL	Phase 4 (approved)	GHSR
RIMONABANT	ChEMBL	Phase 4 (approved)	GHSR
RITONAVIR	ChEMBL	Phase 4 (approved)	GHSR
ROTIGOTINE	ChEMBL	Phase 4 (approved)	GHSR
SAQUINAVIR	ChEMBL	Phase 4 (approved)	GHSR
SERTINDOLE	ChEMBL	Phase 4 (approved)	GHSR
SERTRALINE	ChEMBL	Phase 4 (approved)	GHSR
SUNITINIB	ChEMBL	Phase 4 (approved)	GHSR
TAMOXIFEN	ChEMBL	Phase 4 (approved)	GHSR
TELOTRISTAT ETHYL	ChEMBL	Phase 4 (approved)	GHSR
TERFENADINE	ChEMBL	Phase 4 (approved)	GHSR
THIORIDAZINE	ChEMBL	Phase 4 (approved)	GHSR
THIOTHIXENE	ChEMBL	Phase 4 (approved)	GHSR
TIOCONAZOLE	ChEMBL	Phase 4 (approved)	GHSR
TRIFLUOPERAZINE	ChEMBL	Phase 4 (approved)	GHSR
VANDETANIB	ChEMBL	Phase 4 (approved)	GHSR
VILANTEROL	ChEMBL	Phase 4 (approved)	GHSR
VINDESINE	ChEMBL	Phase 4 (approved)	GHSR
BENIDIPINE	ChEMBL	Phase 3	GHSR
GHRELIN	ChEMBL	Phase 3	GHSR
MELITRACEN	ChEMBL	Phase 3	GHSR
NAFAMOSTAT	ChEMBL	Phase 3	GHSR
OTILONIUM BROMIDE	ChEMBL	Phase 3	GHSR
PIRIBEDIL	ChEMBL	Phase 3	GHSR
PYRONARIDINE	ChEMBL	Phase 3	GHSR
ULIMORELIN	ChEMBL	Phase 3	GHSR
YOHIMBINE	ChEMBL	Phase 3	GHSR
APRINDINE	ChEMBL	Phase 2	GHSR
BENZETHONIUM	ChEMBL	Phase 2	GHSR
BOPINDOLOL	ChEMBL	Phase 2	GHSR
CAPROMORELIN	ChEMBL	Phase 2	GHSR
CAROVERINE	ChEMBL	Phase 2	GHSR