2-HYDROXYPROPYL-BETA-CYCLODEXTRIN

Summary

2-Hydroxypropyl-Beta-Cyclodextrin (CHEMBL577294) is a phase-3 clinical-stage oligosaccharide; indicated across 3 conditions including niemann-pick disease type c and herpes labialis.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligosaccharide
• Indications: 3 conditions
• Clinical trials: 4
• Chemistry: 1541.5 Da · C63H112O42

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL577294
Name	2-HYDROXYPROPYL-BETA-CYCLODEXTRIN
Type	Oligosaccharide
Max phase	3
FDA approved	no
PubChem CID	14049689
Molecular formula	C63H112O42
Molecular weight	1541.5
InChIKey	ODLHGICHYURWBS-LKONHMLTSA-N

SMILES: CC(COC[C@@H]1[C@@H]2[C@@H]([C@H]([C@H](O1)O[C@@H]3[C@H](O[C@@H]([C@@H]([C@H]3O)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)COCC(C)O)COCC(C)O)COCC(C)O)COCC(C)O)COCC(C)O)O)O)O

IUPAC name: (1S,3R,5R,6S,8R,10R,11S,13R,15R,16S,18R,20R,21S,23R,25R,26S,28R,30R,31S,33R,35R,36R,37R,38R,39R,40R,41R,42R,43R,44R,45R,46R,47R,48R,49R)-5,10,15,20,25,30,35-heptakis(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-36,37,38,39,40,41,42,43,44,45,46,47,48,49-tetradecol

Also known as: Hpbcd, Hydroxyl-propyl-beta-cyclodextrin, Hydroxypropyl .beta.-cyclodextrin, Hydroxypropyl bcd, Hydroxypropyl betacyclodextrin, Hydroxypropyl betadex, Hydroxypropyl-bcyclodextrin, Hydroxypropylbetadex, Hydroxypropyl-beta-cyclodextrin, HYDROXYPROPYL-BETA-CYCLODEXTRIN

Patent coverage: 2,493 distinct patent families (4,490 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

Broader ChEMBL bioactivity targets: 7 (assay-derived). Sample: 5-hydroxytryptamine receptor 2B, Tyrosine-protein kinase Fyn, Amine oxidase [flavin-containing] A, Sodium-dependent noradrenaline transporter, 5-hydroxytryptamine receptor 2C, Sodium-dependent dopamine transporter, Androgen receptor.

Bioactivity

ChEMBL activities: 12 potent at pChembl ≥ 5 of 12 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
HTR2B	7.4	Ki	40	nM	CHEMBL_ACT_7643939
HTR2B	7.2	IC50	63	nM	CHEMBL_ACT_7643938
MAOA	6.78	IC50	167	nM	CHEMBL_ACT_7642520
HTR2C	6.08	Ki	833	nM	CHEMBL_ACT_7643941
SLC6A2	5.9	IC50	1270	nM	CHEMBL_ACT_7641078
SLC6A2	5.9	Ki	1260	nM	CHEMBL_ACT_7641079
SLC6A3	5.87	Ki	1341	nM	CHEMBL_ACT_7642469
P15207	5.84	Ki	1440	nM	CHEMBL_ACT_7643961
HTR2C	5.8	IC50	1590	nM	CHEMBL_ACT_7643940
SLC6A3	5.77	IC50	1687	nM	CHEMBL_ACT_7642468
P15207	5.67	IC50	2160	nM	CHEMBL_ACT_7643960
FYN	5.52	IC50	3043	nM	CHEMBL_ACT_7643922

Target pathways

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

Indications & clinical

Indications

3 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
Niemann-Pick disease type C	3	MONDO:0018982	MONDO:0018982
herpes labialis	2	MONDO:0043653	EFO:1001347
Alzheimer disease	2	MONDO:0004975	MONDO:0004975

Clinical trials

Total trials: 4.

Phase distribution

Phase	Trials
PHASE3	2
PHASE1/PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04860960	PHASE3	ACTIVE_NOT_RECRUITING	Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
NCT00942448	PHASE3	COMPLETED	Evaluation of the Efficacy and Safety of Diclofenac HPBCD 25, 50 mg/ml in the Treatment of Post-surgical Pain Following Dental Surgery
NCT02912793	PHASE1/PHASE2	COMPLETED	Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
NCT02939547	PHASE1	COMPLETED	Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

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).