PannTheraPi receives French ANSM nod to advance PTI5803 into Phase 2a trial
核心洞察
France's ANSM (搜索) has approved PannTheraPi (搜索)'s phase 2a trial of PTI5803 (搜索) in patients with drug-resistant epilepsy (搜索) associated with focal cortical dysplasia (搜索).
PTI5803 (搜索) is an oral small molecule pannexin 1 (搜索) channel inhibitor, formulated as extended-release probenecid, being developed as a potential first-in-class therapy.
The multicentre study will enrol adults and adolescents across three dose levels, with safety and tolerability as the primary objective and seizure frequency as a secondary endpoint.
PannTheraPi (搜索) has received approval from the French National Agency for the Safety of Medicines and Health Products (ANSM (搜索)) to advance PTI5803 (搜索) into a phase 2a trial in patients with drug-resistant epilepsy (搜索) associated with focal cortical dysplasia (搜索) (FCD). PTI5803 is an oral small molecule targeting the pannexin 1 (搜索) (Panx1) channel and is an extended-release formulation of probenecid, being developed as a potential first-in-class treatment for FCD, a rare and severe form of epilepsy. The company previously reported positive phase 1 results in healthy volunteers covering safety, pharmacokinetic and pharmacodynamic findings.
The multicentre phase 2a study will initially enrol adult and adolescent patients with drug-resistant seizures associated with FCD and will evaluate PTI5803 (搜索) at three dose levels. Safety and tolerability is the primary objective, with preliminary efficacy, including changes in seizure frequency, assessed as a secondary objective. Patients who complete the main study will be eligible to continue treatment in a long-term open-label extension phase.
The trial will involve epilepsy centres at Marseille-Timone University Hospital, Hospices Civils de Lyon and Fondation La Teppe in Tain-l'Hermitage. PannTheraPi (搜索) said the therapeutic potential of targeting Panx1 may extend beyond FCD, and that PTI5803 (搜索) has potential application in other orphan epilepsies.