Drug intelligence / Profile preview

triptolide

Development stage
Phase 3
Lead developer
Minneamrita Therapeutics
Modality
Classical Binding Small Molecules → Small Molecules, Covalent Small Molecules → Small Molecules, Nucleic Acid-Directed Small Molecules → Small Molecules
Administration
Oral, Intravenous, Topical
01

Overview

Triptolide is a small molecule diterpene triepoxide isolated from the Chinese medicinal herb Tripterygium wilfordii (Thunder God Vine). It exhibits potent immunosuppressive and anti-inflammatory properties and has demonstrated significant antitumor activity in preclinical models. Triptolide acts through multiple mechanisms including inhibition of transcription factors such as NF-κB (notably RELA/p65 and RELB), suppression of heat shock protein 70 (HSP70), disruption of super-enhancer networks in cancer cells, modulation of immune cell function, and interference with RANKL-mediated NF-κB signaling. It also inhibits osteoclast differentiation and bone resorption by increasing IL-10 and TGF-β1 production via regulatory T cells. Triptolide is under investigation for various indications including cancer (notably pancreatic ductal adenocarcinoma), autoimmune diseases like rheumatoid arthritis, polycystic kidney disease, Crohn's disease, HIV infection, bone-related disorders, and others[1][3][4][5][6][7].

Other names
triptolidediterpene triepoxideactive ingredient of Tripterygium wilfordii (Thunder God Vine)
02

Targets

STAT3 (Signal Transducer and Activator of Transcription 3)ERCC3 (General transcription and DNA repair factor IIH helicase subunit XPB)HSP70 (Heat shock protein 70)PRDX5 (Peroxiredoxin-5)RELB (Nuclear factor kappa-light-chain-enhancer of activated B cells subunit RelB)RELA (Nuclear Factor Kappa B Subunit p65)

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