Drug intelligence / Profile preview

PMT-307

Development stage
Preclinical
Modality
Small Molecules
01

Overview

PMT-307 (also known as HO-4450) is a bifunctional small molecule designed as a Paramagnetic Amyloid Ligand (PAL) for the potential treatment of Alzheimer's disease. It features a stilbene scaffold coupled with a six-membered nitroxyl ring. The molecule operates through a dual mechanism: it binds to and remodels the secondary structure of toxic amyloid-beta oligomers (AβOs), specifically reducing the fraction of antiparallel β-strands, and acts as a catalytic antioxidant by mimicking the activity of superoxide dismutase (SOD). This dual action aims to neutralize both the structural toxicity of amyloid aggregates and the associated oxidative stress in the brain. Preclinical studies in Alzheimer's disease mouse models have indicated modest therapeutic effects and protection against Aβ-induced cytotoxicity.

02

Targets

Misfolded SOD1Aβ (Amyloid-beta peptides and aggregates)

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