Summary
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.
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| Web resources: | https://cordis.europa.eu/project/id/190121670 |
| Start date: | 01-10-2023 |
| End date: | 30-09-2025 |
| Total budget - Public funding: | 4 078 906,00 Euro - 2 499 500,00 Euro |
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Original description
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.Status
SIGNEDCall topic
HORIZON-EIC-2023-ACCELERATOROPEN-01Update Date
12-03-2024
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