Objective
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.
Fields of science
Keywords
Programme(s)
- HORIZON.3.1 - The European Innovation Council (EIC) Main Programme
Funding Scheme
HORIZON-EIC-ACC-BF - HORIZON EIC Accelerator Blended FinanceCoordinator
30000 NIMES
France
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.