Drug intelligence / Profile preview

triptolide

Development stage
Phase 3
Lead developer
Minneamrita Therapeutics
Modality
Covalent Small Molecules → Small Molecules, Classical Binding 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

NF-κBSTAT3 (Signal Transducer and Activator of Transcription 3)ERCC3 (General transcription and DNA repair factor IIH helicase subunit XPB)

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