PannTheraPi Advances PTI5803 to Phase IIa Trial for Drug-Resistant Epilepsy
Key Insights
PannTheraPi (search) has submitted a pre-submission file to French regulatory agency ANSM (search) for its first Phase IIa clinical trial of PTI5803 (search) targeting Focal Cortical Dysplasia (search), a rare epileptic syndrome.
The company's lead drug candidate PTI5803 (search) selectively targets the pannexin 1 (search) channel, representing a novel therapeutic approach for drug-resistant epilepsy (search) with demonstrated safety and biomarker activity in Phase I studies.
The Phase IIa trial is planned to start in 2026 following completion of a €10 million funding round, with the EIC Accelerator (search) potentially contributing up to half the amount.
French biotechnology company PannTheraPi (search) has reached a significant regulatory milestone by submitting a pre-submission file to the ANSM (search) (French regulatory agency) for its first Phase IIa clinical trial of PTI5803 (search) in patients with Focal Cortical Dysplasia (search) (FCD), a rare epileptic syndrome with high medical need. The Phase IIa trial is planned to commence in 2026 following completion of the company's current funding round.
Novel Mechanism Targets Drug-Resistant Epilepsy
PTI5803 (search) represents a breakthrough approach to treating epilepsy (search) by selectively targeting the pannexin 1 (search) (Panx1) channel, a novel mechanism of action developed through research at the University of Paris Sciences et Lettres. The drug was developed by company founders Dr. Nathalie Rouach, INSERM research director at the Interdisciplinary Centre for Research in Biology at the Collège de France, and Dr. Gilles Huberfeld, a neurologist-epileptologist at the Adolphe de Rothschild Foundation Hospital.
Focal cortical dysplasia (search) is a malformation of cortical development and one of the leading causes of drug-resistant epilepsy (search), particularly affecting children. Despite current treatment options, the condition often has devastating impacts on quality of life and places significant burden on families and healthcare systems. Recent research has identified the Panx1 channel (search) as a potential therapeutic target for epileptic seizures, with increased expression linked to seizure activity.
Promising Preclinical and Phase I Results
PannTheraPi (search) has made significant progress in just 18 months, validating the production of a new pediatric formulation suitable for children aged one month and older. The company has demonstrated the safety of PTI5803 (search), its extended release profile, and the first signs of activity on the target via biomarkers in a Phase I study in healthy volunteers. These results have enabled the determination of optimal doses to be evaluated in patients.
The development plan has received validation from the European Medicines Agency (search) (EMA), which has approved the Paediatric Investigation Plan (PIP) required for authorization of a medicine for children. Scientific discussions with the US Food and Drug Administration (FDA) are ongoing in parallel.
Expanding Pipeline and Funding Strategy
Beyond PTI5803 (search), PannTheraPi (search) is developing a proprietary pipeline of novel, patent-protected small molecule drug candidates that act on the Panx1 channel (search). These differentiated molecules, identified using artificial intelligence tools and innovative proprietary models, show promising biological results and can potentially be developed for multiple neurological indications including multiple sclerosis (search), chronic pain (search), and neurological and psychiatric disorders.
The company is currently seeking to raise €10 million in funding to accelerate its move into Phase IIa clinical trials and progress its proprietary pipeline. The EIC Accelerator (search) programme may contribute up to half this amount through equity investment. Six pharmaceutical companies are actively monitoring PannTheraPi (search)'s progress under confidentiality agreements, demonstrating growing industry interest in the company's innovative approach.
Leadership Transition
As part of its strategic evolution, PannTheraPi (search) announced that Dr. Granier will step down as Chairman once the current fundraising is completed, with plans to establish a new management team aligned with the company's development objectives. Dr. Granier will remain available to support the company with his medical and scientific expertise as co-founder.