Drug intelligence / Profile preview

ingenol mebutate

Development stage
Phase 4
Lead developer
LEO Pharma
Modality
Peptides, Small Molecules
Administration
Topical
01

Overview

Ingenol mebutate is a small molecule topical agent originally derived from the sap of the *Euphorbia peplus* plant. It was developed for the treatment of actinic keratosis, a precancerous skin condition caused by sun exposure. The drug acts as a cytotoxic agent, rapidly inducing cell death in abnormal keratinocytes through both direct necrosis and immune-mediated mechanisms. Its mechanism involves activation and modulation of protein kinase C isoforms (notably PKC-delta and PKC-alpha), leading to downstream effects on cellular signaling pathways such as Ras/Raf/MAPK and p38 activation, as well as AKT inhibition. Ingenol mebutate also triggers neutrophil-mediated inflammation and antibody-dependent cell death targeting residual dysplastic cells[1][2][5][6][8]. The product was marketed under the brand name Picato but has been discontinued in several markets including the United States.

Brand names
Picato
Other names
ingenol mebutate
02

Targets

PKC (Protein kinase C family)PRKCA (Protein kinase C alpha)SLC25A20 (Carnitine/acylcarnitine translocase)PRKCD (Protein kinase C delta)

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