PannTheraPi prepares plans to conduct its first Phase IIa clinical trial with PTI5803 in patients with resistant epilepsy and accelerate the development of its proprietary pipeline
PannTheraPi prepares plans to conduct its first Phase IIa clinical trial with PTI5803 in patients with resistant epilepsy and accelerate the development of its proprietary pipeline
- Pre-submission file completed with French regulatory agency (ANSM) to discuss clinical trial protocol
- Phase IIa clinical trial planned to start in 2026 once fund raising is closed
- Strategic evolution of PannTheraPi’s management to drive upcoming milestones
Nimes, France – 26th August, 2025 - PannTheraPi, a company developing innovative treatments in neurology by selectively targeting the pannexin 1 (Panx1) channel, announces a new milestone in its development with the pre-submission to the ANSM (the public body that regulates drugs in France) of the protocol for its first Phase IIa clinical trial of PTI5803 in patients with Focal Cortical Dysplasia (FCD), a rare epileptic syndrome with high medical need.
This trial will focus on PannTheraPi’s patent-protected lead oral small molecule drug candidate, PTI5803, which targets the pannexin 1 channel. This innovative mode of action has been shown to be involved in certain forms of epilepsy through research carried out at the University of Paris Sciences et Lettres (PSL) by two of the company’s founders, Dr Nathalie ROUACH, INSERM research director at the Interdisciplinary Centre for Research in Biology at the Collège de France (CNRS UMR 7241 – INSERM U1050) and Dr Gilles HUBERFELD, currently a neurologist-epileptologist at the Adolphe de Rothschild Foundation Hospital and team leader at the Paris Institute of Psychiatry and Neurosciences.
Focal cortical dysplasia (FCD) is a malformation of cortical development and one of the leading causes of drug-resistant epilepsy, particularly in children. Despite current treatment options, it often has devastating impacts on quality of life and places a significant burden on families and healthcare systems. Recent research1 has identified the Panx1 channel as a potential therapeutic target for epileptic seizures, with increased expression linked to seizure activity. By targeting and blocking Panx1 channels, this novel therapeutic approach has shown promise in preclinical models1, potentially offering a transformative new strategy to control seizures. This represents a critical step forward in addressing a major unmet need in epilepsy care.
Based on this work and, with the support of several partners including the EIC Accelerator programme, BPI and independent investors, PannTheraPi has made significant progress in just 18 months, validating the production of a new paediatric formulation (suitable for children aged one month and older) and demonstrating the safety of PTI5803, 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 made it possible to determine the optimal doses to be evaluated in patients. With input from international scientists and clinicians, the development plan has been submitted to the European Medicines Agency (EMA), which has validated the Paediatric Investigation Plan (PIP) required for authorisation of a medicine for children. Scientific discussions with the US Food and Drug Administration (FDA) are ongoing in parallel.
At the same time, PannTheraPi is developing a proprietary pipeline of novel, patent-protected small molecule drug candidates that act on the Panx1 channel. These differentiated molecules, with completely new structures, were identified using artificial intelligence tools and innovative proprietary models, with very promising biological results. The Patent Cooperation Treaty (PCT) report confirms the novelty and strength of the patent covering this class of molecules. These new molecules can be developed in several neurological indications where the target has demonstrated its potential involvement such as multiple sclerosis, chronic pain, and neurological and psychiatric disorders.
Under the chairmanship of Dr GRANIER and the management of Dr Elsa BRILLAUD, and thanks to the commitment of the entire team, PannTheraPi has achieved its initial objectives. The company is now looking to raise funds to accelerate its move into a Phase IIa clinical trial with PTI5803 in epilepsy and progress its proprietary pipeline, as well as expanding operational capability and personnel in line with PannTheraPi’s growth plans. The current funding round targets €10 million, with the possibility of the EIC Accelerator contributing up to half this amount in the form of an equity investment.
Aware of his many other commitments and the significant demands on his time, Dr. GRANIER has informed the Supervisory Board of his intention to step down as Chairman of PannTheraPi once the current fundraising has been completed, at which time the Company will set up a new management team, in line with its development objectives. As co-founder of PannTheraPi and deeply committed to its success, Dr GRANIER will remain available to the future Supervisory Board to support the company with his medical and scientific expertise.
About PannTheraPi
PannTheraPi is a French biotechnology company dedicated to the development of innovative treatments in neurology, with unique expertise in modulators of the pannexin 1 channel. Its objective is to meet unmet medical needs in severe neurological and psychiatric disorders.
PannTheraPi's work and rigorous project development are attracting growing interest and recognition from its peers: six pharmaceutical companies are actively monitoring its progress under confidentiality agreements. They also benefit from international scientific recognition, with PannTheraPi regularly invited to present its advances at various conferences and dedicated events, including the upcoming European Consortium for Epilepsy Trials in parallel with the 36th Congress of the International League Against Epilepsy (ILAE) (Lisbon, Portugal; 30 August - 3 September) and the EIC TechTour – Brain Health (Porto, Portugal; 11-12 September).
For more information, please go to www.panntherapi.com or visit our LinkedIn page
Contacts
PannTheraPi
Sarah Delbaere
CFO
communication@panntherapi.com
1 Dossi et al 2018 : Pannexin-1 channels contribute to seizure generation in human epileptic brain tissue and in a mouse model of epilepsy PMID: 29848662 DOI: 10.1126/scitranslmed.aar379
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