PannTheraPi Receives ANSM Approval to Advance First-in-Class Panx1-Targeting Therapy PTI5803 into Phase 2a in Focal Cortical Dysplasia
核心洞察
PannTheraPi (搜索) received French ANSM (搜索) approval to launch a Phase 2a trial of PTI5803 (搜索) in patients with drug-resistant epilepsy associated with focal cortical dysplasia (搜索) (FCD).
PTI5803 (搜索) is an oral, first-in-class small molecule targeting the pannexin 1 (搜索) (Panx1) channel, with positive Phase 1 safety, pharmacokinetic, and pharmacodynamic results in healthy volunteers.
The multicentre study will enroll adult and adolescent patients across three dose levels, assessing safety and tolerability as the primary objective and seizure frequency as a secondary endpoint.
PannTheraPi (搜索), a clinical-stage biopharmaceutical company developing treatments for neurological diseases by selectively targeting the pannexin 1 (搜索) (Panx1) channel, announced on 8 September 2026 that the French National Agency for the Safety of Medicines and Health Products (ANSM (搜索)) has approved its Phase 2a clinical trial evaluating PTI5803 (搜索) in patients with drug-resistant epilepsy associated with focal cortical dysplasia (搜索) (FCD).
PTI5803 (搜索) 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. The company previously demonstrated positive Phase 1 results for PTI5803 in healthy volunteers, including safety, pharmacokinetic and pharmacodynamic findings.
Trial Design and Objectives
The multicentre Phase 2a study will initially enrol adult and adolescent patients with drug-resistant seizures associated with FCD and evaluate PTI5803 (搜索) 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.
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, Hospices Civils de Lyon, and Fondation La Teppe in Tain-l'Hermitage.
Addressing an Unmet Need
"ANSM (搜索) approval marks an important milestone in the development of PTI5803 (搜索) 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," 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 (搜索). "Having worked to understand the role of pannexin 1 (搜索) in disease and translate that research into a potential therapy, it is particularly rewarding to see PTI5803 now advance into Phase 2a."
Broader Pipeline Implications
"This approval represents an important milestone in PannTheraPi (搜索)'s development as we advance PTI5803 (搜索) 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.
PTI5803 (搜索) is an extended-release formulation of probenecid, advancing into Phase 2a development in focal cortical dysplasia (搜索), 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.