Drug intelligence / Profile preview

leelamine

Development stage
Preclinical
Lead developer
Cipher Pharmaceuticals
Modality
Small Molecules
Administration
Intraperitoneal
01

Overview

Leelamine is a naturally occurring tricyclic diterpene amine, primarily derived from the bark of pine trees (*Pinus* species). It has emerged as a potent anti-cancer agent, particularly in the context of melanoma, due to its unique ability to simultaneously inhibit multiple oncogenic signaling pathways, including PI3K/Akt, MAPK/Erk, and STAT3. Its primary mechanism of action involves the disruption of lysosomal cholesterol trafficking, which leads to the sequestration of cholesterol within lysosomes and the subsequent inhibition of signaling proteins that rely on cholesterol-rich membrane domains for activation. Preclinical studies have demonstrated that leelamine can significantly reduce tumor growth and vascularization while inducing apoptosis in melanoma cells, all while maintaining a favorable safety profile in animal models.

Other names
dehydroabietylamine
02

Targets

NPC1 (Niemann-Pick C1 protein binding inhibition)

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