2-Hydroxylpropyl-beta-cyclodextrin

DB15146InvestigationalSmall molecule

Adrabetadex is under investigation in clinical trial NCT03887533 (Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1).

Mechanism: Pharmacologically active on Insulin and Transcription factor EB
Curator reviewed
Formula / weight: C₅₄H₉₄O₃₈ · 1351.308 g/mol (avg)
Also known as: 2-Hydroxypropyl-b-cyclodextrin · 2-HYDROXYPROPYL-BETA-CYCLODEXTRIN · 2-hydroxypropyl-β-cyclodextrin · Adrabetadex · HP-beta-CD
Code names: VTS-270

Resolves to

Compound

UNII8W6Q67R6NXCAS128446-35-5ChEMBLCHEMBL4297678

Clinical / RWD

RxNorm2105627

Structure

InChIKeyNJWCVQBHNBLNOZ-ZRMYETLXSA-NSMILES

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

Targets

P01308INSP19484TFEB

What you can answer from here — as of September 16, 2026

2Protein targetsEach mapped to UniProt, with action and pharmacological action

Listed above

11Clinical trialsPhase, status and sponsor resolved per trial

3+ReferencesStructured and connected to the statements they support

Which drugs share a target with 2-Hydroxylpropyl-beta-cyclodextrin, and which of those have an active Phase 3 trial?

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