Adrabetadex

Summary

Adrabetadex (CHEMBL4297678) is a phase-3 clinical-stage oligosaccharide; indicated across 1 condition including niemann-pick disease type c.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligosaccharide
• Indications: 1 condition
• Clinical trials: 7
• Chemistry: 1351.3 Da · C54H94O38

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297678
Name	Adrabetadex
Type	Oligosaccharide
Max phase	3
FDA approved	no
PubChem CID	138059665
Molecular formula	C54H94O38
Molecular weight	1351.3
InChIKey	NJWCVQBHNBLNOZ-ZRMYETLXSA-N

SMILES: CC(COC[C@@H]1[C@@H]2C[C@H]([C@H](O1)O[C@@H]3[C@H](O[C@@H]([C@@H]([C@H]3O)OCC(C)O)O[C@@H]4[C@H](O[C@@H]([C@@H]([C@H]4O)O)O[C@@H]5[C@H](O[C@@H]([C@@H]([C@H]5O)O)O[C@@H]6[C@H](O[C@@H]([C@@H]([C@H]6O)O)O[C@@H]7[C@H](O[C@@H]([C@@H]([C@H]7O)O)O[C@@H]8[C@H](O[C@H](O2)[C@@H]([C@H]8O)O)COCC(C)O)CO)COCC(C)O)CO)CO)CO)O)O

IUPAC name: (1S,3R,5R,6S,8R,10R,11S,13S,15R,16S,18R,20R,21S,23R,25R,26S,28R,30R,31S,33R,35R,36R,37R,38R,39R,40R,41R,42R,43R,44R,45R,47R,48S,49R)-5,20,30,35-tetrakis(hydroxymethyl)-49-(2-hydroxypropoxy)-10,15,25-tris(2-hydroxypropoxymethyl)-2,4,7,9,12,14,17,19,22,24,27,29,32,34-tetradecaoxaoctacyclo[31.2.2.23,6.28,11.213,16.218,21.223,26.228,31]nonatetracontane-36,37,38,39,40,41,42,43,44,45,47,48-dodecol

Also known as: Adrabetadex, Kleptose hpb, VT-270, Vts-270, VTS-270, Vts270, ADRABETADEX

Patent coverage: 53 distinct patent families (191 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

No target linkage available.

Bioactivity

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

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

1 indication (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
Niemann-Pick disease type C	3	MONDO:0018982	MONDO:0018982

Clinical trials

Total trials: 7.

Phase distribution

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

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03643562	PHASE3	TERMINATED	Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
NCT03879655	PHASE2/PHASE3	TERMINATED	Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
NCT04958642	PHASE2/PHASE3	TERMINATED	Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
NCT03471143	PHASE1/PHASE2	COMPLETED	Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
NCT03687476	PHASE2	WITHDRAWN	Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease
NCT03887533	PHASE1/PHASE2	TERMINATED	Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1
NCT01747135	PHASE1	COMPLETED	Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease

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

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).