Pannexin 1 – a novel target for pediatric orphan epilepsy

Epilepsy is a group of syndromes with high unmet medical need, especially in children as frequent seizures affect the immature brain and its development. In some orphan syndromes, drug resistance is 70% with dramatic consequences for patients and their families.

Novel treatments must prevent the negative effects of seizures, but also minimize off-target effects on brain development and cognitive functions.

Our solution is the specific blockade of the activated form of the Pannexin 1 (Panx1) channel using a reformulated form of probenecid: PTI5803.

This compound, a first-in-class drug, inhibits seizures preclinically in >80% of cases without hampering cognitive functions.

Designed to be adapted to children (from 6 months of age) and to improve the bioavailability, PTI5803 is a highly specific breakthrough product to counteract the pathological activation of Panx1 in patients with an ultra-resistant orphan epilepsy, with the potential to disrupt the market of paediatric epilepsy.