PannTheraPi Advances Phase 2a Trial for Rare Epilepsy Drug PTI5803 Targeting Pannexin 1 Channel
Key Insights
PannTheraPi (search) has submitted a regulatory application to initiate a Phase 2a clinical trial of PTI5803 (search), an oral small molecule targeting the pannexin 1 (search) channel for focal cortical dysplasia (search) treatment.
The Phase 2a study will evaluate safety, tolerability, pharmacological profile and initial efficacy signals of PTI5803 (search) in both adult and pediatric patients with this rare, drug-resistant epilepsy (search).
The company secured European patent protection for PTI5803 (search) in epilepsy (search), adding to existing patents in the US, Canada, and Japan with protection extending through 2037.
French biotechnology company PannTheraPi (search) has submitted a regulatory application via the European Clinical Trials Information System (CTIS) to initiate a Phase 2a clinical trial of PTI5803 (search), an oral small molecule drug candidate targeting the pannexin 1 (search) (Panx1 (search)) channel for the treatment of focal cortical dysplasia (search) (FCD). The rare and severe form of epilepsy (search) represents a significant unmet medical need with limited treatment options for patients experiencing drug-resistant seizures.
Clinical Trial Design and Objectives
The Phase 2a study will evaluate the safety, tolerability, pharmacological profile and initial signals of efficacy of PTI5803 (search) in patients with FCD, including both adults and children. FCD is characterized as a rare and severe drug-resistant epilepsy (search) with limited treatment options currently available to patients.
"The submission of this application marks an important milestone in the clinical development of PTI5803 (search) for patients with focal cortical dysplasia (search)," said Dr. Gilles Huberfeld, neurologist-epileptologist and co-founder of PannTheraPi (search). "This step brings us closer to delivering a much-needed therapeutic solution for patients who currently have limited options."
Intellectual Property Expansion
Concurrent with the regulatory submission, PannTheraPi (search) secured a European patent covering the use of its drug candidate in epilepsy (search). This patent protection follows previously granted patents in the United States, Canada, and Japan, providing comprehensive intellectual property coverage through 2037.
Leadership Strengthening
To support the company's transition into this new phase of clinical development, PannTheraPi (search) appointed Sophie Binay as General Manager and Chief Scientific Officer. Binay brings 20 years of experience advancing innovative R&D programmes from proof of concept through clinical development across startups and mid-sized life sciences companies.
"I am delighted to join PannTheraPi (search) at such a pivotal time. PTI5803 (search) has strong potential, and I look forward to working with the team to accelerate its development and strengthen the company's broader pipeline," said Sophie Binay.
Sophie will lead the acceleration of PannTheraPi (search)'s clinical strategy and operational development as the company advances its flagship program.
Strategic Positioning
"With the arrival of Sophie Binay and the upcoming entry into Phase 2a of PTI5803 (search), our flagship program, PannTheraPi (search) is entering a new phase of growth and structuring around international ambitions," added Dr. Luc-André Granier, Chairman and CEO of PannTheraPi.
PannTheraPi (search) specializes in developing innovative neurological treatments with unique expertise in the pannexin 1 (search) channel, aiming to address unmet medical needs in severe neurological and psychiatric disorders. The company's focus on the Panx1 (search) channel represents a novel therapeutic approach for treating drug-resistant epilepsy (search) conditions.