globenewswire news

Phase 2a study to evaluate PannTheraPi’s first-in-class Pannexin 1-targeting therapy in patients with drug-resistant FCD, a rare and severe form of epilepsy

Nîmes, France, 8 September 2026 — PannTheraPi, a clinical-stage biopharmaceutical company developing innovative treatments for neurological diseases by selectively targeting the pannexin 1 (Panx1) channel, today announces approval by the French National Agency for the Safety of Medicines and Health Products (ANSM) of its Phase 2a clinical trial evaluating PTI5803 in patients with drug-resistant epilepsy associated with focal cortical dysplasia (FCD).

PTI5803 is an oral small molecule targeting the Panx1 channel and is being developed as a potential first-in-class treatment for FCD, a rare and severe form of epilepsy. PannTheraPi previously demonstrated positive Phase 1 results for PTI5803 in healthy volunteers, including 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 evaluate PTI5803 across three dose levels. The primary objective is to assess safety and tolerability, with preliminary efficacy, including changes in seizure frequency, assessed as a secondary objective. Patients completing the main study will be eligible to continue treatment in a long-term open-label extension phase.

“ANSM approval marks an important milestone in the development of PTI5803 and brings us closer to evaluating its potential in patients with FCD, who have very limited treatment options, particularly when surgery is not possible or has failed. Having worked to understand the role of pannexin 1 in disease and translate that research into a potential therapy, it is particularly rewarding to see PTI5803 now advance into Phase 2a. We look forward to initiating patient recruitment and evaluating PTI5803 as a potential treatment targeting the pannexin 1 channel,” said Dr Gilles Huberfeld, neurologist-epileptologist at the Rothschild Foundation Hospital (Paris), and Dr Nathalie Rouach, neurobiologist at the Collège de France, co-founders of PannTheraPi.

Dr Marion Quirins, neurologist-epileptologist at Hôpital Fondation Adolphe de Rothschild in Paris, will serve as Principal Investigator of the study. The trial will also involve epilepsy centres at Marseille-Timone University Hospital (Prof. Fabrice Bartolomei), Hospices Civils de Lyon (Prof. Sylvain Rheims) and Fondation La Teppe in Tain-l’Hermitage (Dr Nicolas Mezouar).

“This approval represents an important milestone in PannTheraPi’s development as we advance PTI5803 into Phase 2a. It also supports the continued development of our broader pipeline of innovative treatments for rare neurological diseases, grounded in disease pathophysiology and designed to optimise efficacy while preserving brain function. We believe the therapeutic potential of targeting Panx1 may extend beyond FCD,” said Dr Luc-André Granier, President and co-founder of PannTheraPi.

xxx

About PannTheraPi

PannTheraPi is a French clinical-stage biopharmaceutical company developing innovative treatments targeting the pannexin 1 (Panx1) channel for rare neurological diseases. Its lead candidate, PTI5803, is an extended-release formulation of probenecid advancing into Phase 2a development in focal cortical dysplasia (FCD), with potential application in other orphan epilepsies. The company is also advancing a programme of new chemical entities (NCEs) specifically targeting Panx1 in other neurological conditions.

For more information, visit www.panntherapi.com and follow us on LinkedIn.

Contact
PannTheraPi
Sophie BINAY, CSO & General Manager
communication@panntherapi.com

MEDiSTRAVA
Sylvie Berrebi
Sylvie.berrebi@medistrava.com