Setmelanotide

drug
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Also known as BIM-22493ImcivreeRM-493Setmelanotida

Summary

Setmelanotide (CHEMBL3301624) is an approved protein (ATC A08AA12) targeting MC1R, MC3R, and MC4R; indicated across 5 conditions including obesity disorder and bardet-biedl syndrome.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Protein
  • ATC class: A08AA12
  • Targets: 4 (MC1R, MC3R, MC4R…)
  • Indications: 5 conditions
  • Clinical trials: 25
  • Chemistry: 1117.3 Da · C49H68N18O9S2

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL3301624
NameSetmelanotide
TypeProtein
Max phase4
FDA approvedyes
PubChem CID11993702
ATCA08AA12
Molecular formulaC49H68N18O9S2
Molecular weight1117.3
InChIKeyHDHDTKMUACZDAA-PHNIDTBTSA-N

SMILES: C[C@@H]1C(=O)N[C@H](C(=O)N[C@@H](C(=O)N[C@H](C(=O)N[C@H](C(=O)N[C@@H](CSSC[C@@H](C(=O)N1)NC(=O)[C@H](CCCN=C(N)N)NC(=O)C)C(=O)N)CC2=CNC3=CC=CC=C32)CCCN=C(N)N)CC4=CC=CC=C4)CC5=CN=CN5

IUPAC name: (4R,7S,10S,13R,16S,19R,22R)-22-[[(2S)-2-acetamido-5-(diaminomethylideneamino)pentanoyl]amino]-13-benzyl-10-[3-(diaminomethylideneamino)propyl]-16-(1H-imidazol-5-ylmethyl)-7-(1H-indol-3-ylmethyl)-19-methyl-6,9,12,15,18,21-hexaoxo-1,2-dithia-5,8,11,14,17,20-hexazacyclotricosane-4-carboxamide

Also known as: BIM-22493, Imcivree, RM-493, Setmelanotida, Setmelanotide, SETMELANOTIDE, setmelanotide

Parent form; salt/anhydrous children: CHEMBL4650273

Patent coverage: 256 distinct patent families (679 SureChEMBL compound mentions), from 4 matched compound structure(s). One matched structure accounts for 550 (81%) 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).

GeneTargetActionpAffinityCancer dependencyUniProt
MC1RMC1 receptorAgonist8.411%Q01726
MC3RMC3 receptorAgonist80.1%P41968
MC4RMC4 receptorAgonist8.680%P32245
MC5RMC5 receptorAgonist6.370%P33032

Broader ChEMBL bioactivity targets: 5 (assay-derived). Sample: Melanocortin receptor 4, Melanocortin receptor 4, Melanocyte-stimulating hormone receptor, Melanocortin receptor 5, Melanocortin receptor 3.

Bioactivity

ChEMBL activities: 22 potent at pChembl ≥ 5 of 22 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
MC3R9.82EC500.15nMCHEMBL_ACT_24362763
MC3R9.82EC500.15nMCHEMBL_ACT_24697728
MC1R9.8EC500.16nMCHEMBL_ACT_24362735
MC1R9.8EC500.16nMCHEMBL_ACT_24697694
MC4R9.57EC500.27nMCHEMBL_ACT_25500524
MC4R9.57IC500.27nMCHEMBL_ACT_29222748
P705969.55EC500.28nMCHEMBL_ACT_25500525
MC4R9.41EC500.39nMCHEMBL_ACT_24362791
MC4R9.4EC500.4nMCHEMBL_ACT_24697759
MC4R8.68Ki2.1nMCHEMBL_ACT_29222747
MC1R8.41Ki3.9nMCHEMBL_ACT_29222751
MC4R8.39Ki4.07nMCHEMBL_ACT_24362875
MC4R8.37Ki4.27nMCHEMBL_ACT_24697663
MC1R8.34Ki4.57nMCHEMBL_ACT_24362819
MC1R8.34Ki4.57nMCHEMBL_ACT_24697601
MC3R8.28IC505.3nMCHEMBL_ACT_29222750
MC1R8.24IC505.8nMCHEMBL_ACT_29222752
MC3R8Ki10nMCHEMBL_ACT_29222749
MC3R7.88Ki13.18nMCHEMBL_ACT_24362847
MC3R7.88Ki13.18nMCHEMBL_ACT_24697632
MC5R5.73IC501862nMCHEMBL_ACT_24697790
MC5R5.72IC501905nMCHEMBL_ACT_24362903

Target pathways

Aggregated over 4 target gene(s): MC1R, MC3R, MC4R, MC5R.

Top Reactome pathways

10 total, by targets touching each:

PathwayTargetsGenes
Developmental Biology4MC1R, MC3R, MC4R, MC5R
Signal Transduction4MC1R, MC3R, MC4R, MC5R
Signaling by GPCR4MC1R, MC3R, MC4R, MC5R
Class A/1 (Rhodopsin-like receptors)4MC1R, MC3R, MC4R, MC5R
Peptide ligand-binding receptors4MC1R, MC3R, MC4R, MC5R
GPCR downstream signalling4MC1R, MC3R, MC4R, MC5R
G alpha (s) signalling events4MC1R, MC3R, MC4R, MC5R
GPCR ligand binding4MC1R, MC3R, MC4R, MC5R
MITF-M-regulated melanocyte development4MC1R, MC3R, MC4R, MC5R
Transcriptional and post-translational regulation of MITF-M expression and activity4MC1R, MC3R, MC4R, MC5R

Dominant GO biological processes

GO termTargets
adenylate cyclase-activating G protein-coupled receptor signaling pathway4
signal transduction4
G protein-coupled receptor signaling pathway4
G protein-coupled receptor signaling pathway, coupled to cyclic nucleotide second messenger3
regulation of feeding behavior3
phospholipase C-activating G protein-coupled receptor signaling pathway2
adenylate cyclase-modulating G protein-coupled receptor signaling pathway2
UV protection1
sensory perception of pain1
negative regulation of tumor necrosis factor production1
intracellular signal transduction1
melanin biosynthetic process1
pigmentation1
positive regulation of transcription by RNA polymerase II1
positive regulation of melanin biosynthetic process1

Indications & clinical

Indications

5 indications (1 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).

IndicationTrial phaseMONDOEFO
obesity disorder4MONDO:0011122EFO:0001073
Bardet-Biedl syndrome3MONDO:0015229MONDO:0015229
Prader-Willi syndrome2MONDO:0008300MONDO:0008300
morbid obesity1MONDO:0005139EFO:0001074
kidney failure1MONDO:0001106HP:0000083

Clinical trials

Total trials: 25.

Phase distribution

PhaseTrials
PHASE39
PHASE29
PHASE13
PHASE41
PHASE2/PHASE31
PHASE1/PHASE21
Not specified1

Top trials by phase / activity

NCTPhaseStatusTitle
NCT05046132PHASE4COMPLETEDStudy of Setmelanotide Effects on QTc (Corrected QT) Interval in Healthy Participants
NCT05093634PHASE3ACTIVE_NOT_RECRUITINGEMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway
NCT05774756PHASE3ACTIVE_NOT_RECRUITINGA Trial of Setmelanotide in Acquired Hypothalamic Obesity
NCT06596135PHASE3ENROLLING_BY_INVITATIONOpen-Label Extension Study of Setmelanotide
NCT06760546PHASE3RECRUITINGA Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)
NCT02896192PHASE3COMPLETEDSetmelanotide for the Treatment of Early-Onset Pro-Opiomelanocortin (POMC) Deficiency Obesity
NCT03287960PHASE3COMPLETEDSetmelanotide for the Treatment of Leptin Receptor (LEPR) Deficiency Obesity
NCT03651765PHASE2/PHASE3COMPLETEDLong Term Extension Trial of Setmelanotide
NCT03746522PHASE3COMPLETEDSetmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity
NCT04966741PHASE3COMPLETEDSetmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity
NCT05194124PHASE3COMPLETEDPhase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway
NCT06772597PHASE2ACTIVE_NOT_RECRUITINGA Study of Setmelanotide in Patients With Prader-Willi Syndrome
NCT07496463PHASE2NOT_YET_RECRUITINGSetmelanotide to Treat Obesity in a Patient With Pseudohypoparathyroidism Type 1a (PHP1a)
NCT01749137PHASE2COMPLETEDPhase 2 Study to Evaluate Safety and Efficacy of RM-493 in Obese Participants
NCT02041195PHASE1/PHASE2COMPLETEDPhase 1b/2a Study to Evaluate Safety and Efficacy of Setmelanotide in Obese Patients
NCT02311673PHASE2COMPLETEDPhase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome
NCT02507492PHASE2UNKNOWNRM-493 Treatment Trial in Proopiomelanocortin (POMC) Deficient Patients
NCT03013543PHASE2COMPLETEDSetmelanotide Phase 2 Treatment Trial in Participants With Rare Genetic Disorders of Obesity
NCT03262610PHASE2COMPLETEDSetmelanotide in a Single Patient With Partial Lipodystrophy
NCT04725240PHASE2COMPLETEDOpen-Label Study of Setmelanotide in Hypothalamic Obesity
NCT04963231PHASE2COMPLETEDDAYBREAK: A Study of Setmelanotide in Participants With Specific Gene Variants in the Melanocortin-4 Receptor (MC4R) Pathway
NCT01867437PHASE1COMPLETEDEffects of RM-493 on Energy Expenditure in Obese Individuals
NCT02431442PHASE1COMPLETEDStudy to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RM-493 Administered to Healthy, Obese, Non-diabetic Volunteers
NCT04348175PHASE1COMPLETEDA Phase 1 Study to Evaluate the Pharmacokinetics of Setmelanotide in Subjects With Varying Degrees of Renal Impairment
NCT05183802Not specifiedAPPROVED_FOR_MARKETINGAn Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)

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.

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.

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

MoleculeSourceStatusShared targets
AFAMELANOTIDEChEMBL + PubChemPhase 4 (approved)MC1R, MC3R, MC4R, MC5R
BREMELANOTIDEChEMBLPhase 4 (approved)MC1R, MC3R, MC4R, MC5R
INTERMEDINEChEMBLPhase 2MC1R, MC3R, MC4R, MC5R
AMIODARONEChEMBLPhase 4 (approved)MC3R, MC4R, MC5R
ASTEMIZOLEChEMBLPhase 4 (approved)MC3R, MC4R, MC5R
CHLORPROMAZINEChEMBLPhase 4 (approved)MC3R, MC4R, MC5R
COLISTINChEMBLPhase 4 (approved)MC3R, MC4R, MC5R
FLUPHENAZINEChEMBLPhase 4 (approved)MC3R, MC4R, MC5R
MICONAZOLEChEMBLPhase 4 (approved)MC3R, MC4R, MC5R
SERTRALINEChEMBLPhase 4 (approved)MC3R, MC4R, MC5R
TAMOXIFENChEMBLPhase 4 (approved)MC3R, MC4R, MC5R
CLOZAPINEChEMBL + PubChemPhase 4 (approved)MC3R, MC4R
CRIZOTINIBChEMBL + PubChemPhase 4 (approved)MC3R, MC4R
DESLORATADINEChEMBL + PubChemPhase 4 (approved)MC3R, MC4R
IvacaftorChEMBL + PubChemPhase 4 (approved)MC3R, MC4R
LinagliptinChEMBL + PubChemPhase 4 (approved)MC3R, MC4R
PIMOZIDEChEMBL + PubChemPhase 4 (approved)MC3R, MC4R
PYRAZINAMIDEChEMBL + PubChemPhase 4 (approved)MC3R, MC4R
TEGASERODChEMBL + PubChemPhase 4 (approved)MC3R, MC4R
AMITRIPTYLINEChEMBLPhase 4 (approved)MC3R, MC4R
AMLODIPINEChEMBLPhase 4 (approved)MC3R, MC4R
ANTAZOLINEChEMBLPhase 4 (approved)MC3R, MC4R
BEPRIDILChEMBLPhase 4 (approved)MC3R, MC4R
BOSUTINIBChEMBLPhase 4 (approved)MC3R, MC4R
CARVEDILOLChEMBLPhase 4 (approved)MC3R, MC4R
CHLORHEXIDINEChEMBLPhase 4 (approved)MC3R, MC4R
CLEMASTINEChEMBLPhase 4 (approved)MC3R, MC4R
CLOMIPRAMINEChEMBLPhase 4 (approved)MC3R, MC4R
DULOXETINEChEMBLPhase 4 (approved)MC3R, MC4R
ECONAZOLEChEMBLPhase 4 (approved)MC3R, MC5R
FENOTEROLChEMBLPhase 4 (approved)MC3R, MC4R
FLUSPIRILENEChEMBLPhase 4 (approved)MC3R, MC4R
GRAMICIDINChEMBLPhase 4 (approved)MC4R, MC5R
HALOPERIDOLChEMBLPhase 4 (approved)MC3R, MC4R
IMIPRAMINEChEMBLPhase 4 (approved)MC3R, MC4R
LIDOCAINEChEMBLPhase 4 (approved)MC3R, MC4R
MEFLOQUINEChEMBLPhase 4 (approved)MC3R, MC4R
MITOXANTRONEChEMBLPhase 4 (approved)MC3R, MC4R
MODAFINILChEMBLPhase 4 (approved)MC3R, MC4R
NAFARELINChEMBLPhase 4 (approved)MC3R, MC4R
PROCHLORPERAZINEChEMBLPhase 4 (approved)MC3R, MC4R
PROMETHAZINEChEMBLPhase 4 (approved)MC3R, MC4R
RIFAXIMINChEMBLPhase 4 (approved)MC3R, MC4R
RIMONABANTChEMBLPhase 4 (approved)MC3R, MC4R
ROSIGLITAZONEChEMBLPhase 4 (approved)MC3R, MC4R
TERFENADINEChEMBLPhase 4 (approved)MC3R, MC4R
THIORIDAZINEChEMBLPhase 4 (approved)MC3R, MC4R
TRIFLUOPERAZINEChEMBLPhase 4 (approved)MC3R, MC4R
PL-8177ChEMBLPhase 2MC1R, MC4R
AbirateronePubChemApprovedMC3R, MC4R
Aclidinium BromidePubChemApprovedMC3R, MC4R
AcyclovirPubChemApprovedMC3R, MC4R
AllopurinolPubChemApprovedMC3R, MC4R
AlogliptinPubChemApprovedMC3R, MC4R
AmiloridePubChemApprovedMC3R, MC4R
AnagrelidePubChemApprovedMC3R, MC4R
AprepitantPubChemApprovedMC3R, MC4R
AtazanavirPubChemApprovedMC3R, MC4R
Betamethasone ValeratePubChemApprovedMC3R, MC4R
BosentanPubChemApprovedMC3R, MC4R