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Ferroptosis & Inflammation

Targeting oxidative stress and inflammation pathways to treat CNS diseases

The Ferroptosis & Inflammation platform is based on a family of enzyme targets – oxidoreductases – which are critical to the generation and regulation of energy that’s key to disease pathology in the central nervous system (CNS) and other organ systems. The platform utilizes expertise in electron-transfer chemistry to modulate key biological processes beyond the reach of current drug development approaches.

The lead compounds from the Ferroptosis & Inflammation platform, vatiquinone (formerly PTC743) and utreloxastat (formerly PTC857), target the enzyme 15-lipoxygenase – a key enzymatic hub that regulates the inflammation and oxidative stress that underpin CNS pathology. 

Vatiquinone has been evaluated in over 400 patients with duration of exposure up to 10 years and has been reported to be safe and well-tolerated.