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Bio-e

Targeting mitochondrial pathologies to treat CNS diseases and cancer

The Bio-e 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 Bio-e 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 mitochondrial disease and CNS pathology. Two registration-directed studies are ongoing with vatiquinone: a Phase 2/3 trial in refractory mitochondrial epilepsy and a Phase 3 trial in Friedreich ataxia. In addition, PTC is conducting a clinical trial in amyotrophic lateral sclerosis (ALS) for utreloxastat.

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.

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Gene Therapy

Targeted genetic therapies to treat rare CNS disorders

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Nonsense Suppression

Bypassing translation mutations to enable protein production

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Alternative Splicing

Modulating splicing to control protein production