Drug intelligence / Profile preview

MDL72527

Development stage
Preclinical
Lead developer
Sanofi
Modality
Small Molecules
01

Overview

MDL72527 is a small molecule experimental drug that acts as a potent and selective irreversible inhibitor of polyamine oxidases, specifically targeting spermine oxidase (SMOX) and polyamine oxidase (PAOX). By inhibiting these enzymes, it blocks the catabolism of polyamines such as spermine and spermidine. This inhibition reduces the production of toxic byproducts like hydrogen peroxide and acrolein-conjugated proteins, which are implicated in cellular damage. Preclinical studies have demonstrated neuroprotective effects, lysosomotropic activity (accumulation in lysosomes), anticancer properties through apoptosis stimulation in cancer cells, and significant reduction of retinal neuro-glial injury and vascular permeability in models of diabetic retinopathy and ischemic retinopathy. The compound was originally developed by Aventis SA.

Other names
N1,N4-bis(2,3-butadienyl)-1,4-butanediamine
02

Targets

PAOX (N1-acetylpolyamine oxidase)SMOX (Spermine oxidase)

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