Drug intelligence / Profile preview

EPI-589

Development stage
Phase 2
Lead developer
PTC Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

EPI-589 is an orally available, small-molecule, novel redox-active agent with high blood-brain barrier permeability. It is designed to reduce oxidative stress and enhance mitochondrial energy metabolism by acting as a substrate for quinone-oxidoreductases and increasing glutathione (GSH) levels. EPI-589 has demonstrated potent protective effects against oxidative stress-induced cell death in preclinical models, including ALS patient-derived cells and animal models. The drug was originally developed by BioElectron Technology Corporation (formerly Edison Pharmaceuticals) for the treatment of inflammatory neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and Parkinson’s disease. Sumitomo Pharma holds licensing rights in Japan and North America, while PTC Therapeutics acquired BioElectron in 2019. Clinical trials have shown that EPI-589 is well tolerated at doses up to 1,500 mg per day in healthy adults and may slow disease progression in ALS patients[1][4][5][6].

Other names
R-troloxamide quinone
02

Targets

NeuraminidaseNQO1

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