PannTheraPi Advances PTI5803 into Phase 2a for Rare Epilepsy, Secures European Patent and Appoints New CSO

15 April 2026 | Wednesday | News

Regulatory filing via CTIS initiates mid-stage trial in focal cortical dysplasia, while expanded IP protection and the appointment of Sophie Binay strengthen the company’s clinical and strategic trajectory.

  • Regulatory submission filed to initiate a Phase 2a trial of its drug candidate, PTI5803 in patients with focal cortical dysplasia (FCD), a rare and severe form of epilepsy
  • PannTheraPi secures European patent covering the use of its drug candidate in epilepsy
  • Sophie Binay appointed as General Manager and Chief Scientific Officer

PannTheraPi, a biotechnology company developing innovative neurological treatments targeting the pannexin 1 (Panx1) channel, announces significant progress in its clinical development alongside a key leadership appointment to strengthen its scientific and operational capabilities.

The company has submitted a regulatory application via the European Clinical Trials Information System (CTIS) to initiate a Phase 2a clinical trial of PTI5803, an oral small molecule drug candidate for the treatment of focal cortical dysplasia (FCD) in both adults and children. The Phase 2a study aims to evaluate the safety, tolerability, pharmacological profile and initial signals of efficacy of PTI5803 in patients with FCD, a rare and severe drug-resistant epilepsy with limited treatment option.

The submission of this application marks an important milestone in the clinical development of PTI5803 for patients with focal cortical dysplasia,” said Dr. Gilles Huberfeld, neurologist-epileptologist and co-founder of PannTheraPi.This step brings us closer to delivering a much-needed therapeutic solution for patients who currently have limited options.”

In parallel, PannTheraPi secured a European patent covering the use of its drug candidate in epilepsy. This follows previously granted patents in the United States, Canada, and Japan, securing protection through to 2037.

To support this new phase of development, PannTheraPi announces the appointment of Sophie Binay as General Manager and Chief Scientific Officer.

Sophie will lead the acceleration of PannTheraPi’s clinical strategy and operational development. She brings 20 years of experience advancing innovative R&D programmes, from proof of concept through to clinical development, across startups and mid-sized life sciences companies.

I am delighted to join PannTheraPi at such a pivotal time. PTI5803 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, newly appointed General Manager and Chief Scientific Officer.

With the arrival of Sophie Binay and the upcoming entry into Phase 2a of PTI5803, our flagship program, PannTheraPi is entering a new phase of growth and structuring around international ambitions,” added Dr. Luc-André Granier, Chairman and CEO of PannTheraPi.

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