PannTheraPi Gets French Approval to Start Phase 2a Trial of PTI5803 in Drug-Resistant Epilepsy

French clinical-stage biopharmaceutical company PannTheraPi has received approval from the French National Agency for the Safety of Medicines and Health Products (ANSM) to conduct a Phase 2a clinical trial of PTI5803.

The trial will study the treatment in patients with drug-resistant epilepsy associated with focal cortical dysplasia (FCD).

FCD is a rare condition in which part of the brain develops abnormally. This abnormal area can cause repeated seizures. For some patients, available medicines do not control the seizures, while surgery may not be possible or may not work.

PannTheraPi is developing PTI5803 as a potential new treatment for these patients.

What Is PTI5803?

PTI5803 is an oral small-molecule drug designed to target the pannexin 1, or Panx1, channel.

Panx1 is a type of channel found in cells and is involved in several biological processes. PannTheraPi is studying whether selectively targeting this channel can help control disease processes involved in neurological disorders.

The company is developing PTI5803 as a potential first-in-class treatment for FCD.

In simple terms, the company is testing whether changing the activity of the Panx1 channel can help reduce seizures in patients whose epilepsy is difficult to treat.

PTI5803 Has Already Been Tested in Healthy Volunteers

Before moving into patients with FCD, PTI5803 was evaluated in a Phase 1 clinical study involving healthy volunteers.

PannTheraPi previously reported positive Phase 1 results.

The study provided information about the safety and tolerability of PTI5803. Researchers also looked at pharmacokinetics and pharmacodynamics.

Pharmacokinetics refers to what the body does to a drug, including how the drug is absorbed, distributed, and eliminated.

Pharmacodynamics looks at what the drug does in the body and whether it produces the expected biological effect.

These early findings supported the company's decision to move PTI5803 into the next stage of clinical development.

Phase 2a Trial Will Study Patients With Drug-Resistant Epilepsy

The newly approved Phase 2a study will be a multicentre clinical trial in France.

The study will initially enrol adults and adolescents who have drug-resistant seizures associated with FCD.

Patients will receive PTI5803 at one of three dose levels.

The main purpose of this stage is to understand whether the treatment is safe and well tolerated in people with FCD.

Researchers will also collect early information about whether the treatment may help reduce seizures.

Safety Will Be the Main Focus

The primary objective of the Phase 2a study is to evaluate the safety and tolerability of PTI5803.

This means researchers will closely monitor patients for side effects and other safety issues while they receive the treatment.

This is an important step because a drug that shows promise in laboratory studies or healthy volunteers still needs to be carefully evaluated in patients with the disease it is intended to treat.

The study will also provide information that can help determine how PTI5803 should be developed in later clinical trials.

Researchers Will Also Look at Seizure Frequency

Although safety is the main focus, the trial will also examine preliminary signs of effectiveness.

One of the secondary measures will be changes in seizure frequency.

Researchers will compare seizure activity during treatment to understand whether PTI5803 may have an effect on the number of seizures experienced by patients.

Because this is a Phase 2a study, these results will mainly provide early evidence rather than definitive proof of effectiveness.

Larger and later-stage clinical studies would normally be needed to confirm whether the treatment works.

Patients Can Continue Into a Long-Term Extension Study

Patients who complete the main Phase 2a study will have the option to continue receiving treatment through a long-term open-label extension phase.

In an open-label study, both the researchers and patients know which treatment is being given.

This extension can provide additional information about the longer-term safety and tolerability of PTI5803.

It may also allow researchers to observe how patients respond to continued treatment over a longer period.

French Epilepsy Centres Will Take Part

The Phase 2a study will involve several specialist epilepsy centres in France.

Dr Marion Quirins, neurologist-epileptologist at Hôpital Fondation Adolphe de Rothschild in Paris, will serve as the Principal Investigator.

Other participating centres include Marseille-Timone University Hospital, where the study will involve Prof. Fabrice Bartolomei; Hospices Civils de Lyon, with Prof. Sylvain Rheims; and Fondation La Teppe in Tain-l’Hermitage, with Dr Nicolas Mezouar.

The involvement of specialist epilepsy centres is intended to support the recruitment and clinical evaluation of patients with FCD.

PannTheraPi Focuses on the Panx1 Channel

PannTheraPi was founded by scientists working in neurology and neurobiology.

Dr Gilles Huberfeld, a neurologist-epileptologist at the Rothschild Foundation Hospital in Paris, and Dr Nathalie Rouach, a neurobiologist at the Collège de France, are co-founders of the company.

The company is focused on developing treatments that selectively target the Panx1 channel.

Its research approach is based on understanding the biological mechanisms involved in neurological diseases and then developing medicines designed to act on those mechanisms.

Company Says the Approval Brings PTI5803 Closer to Patients

Dr Gilles Huberfeld and Dr Nathalie Rouach said the ANSM approval allows PannTheraPi to move PTI5803 into Phase 2a testing in patients with FCD.

They highlighted the limited treatment options available for people with FCD, particularly when surgery cannot be performed or has failed.

The researchers said they are looking forward to starting patient recruitment and studying PTI5803 as a treatment targeting the Panx1 channel.

PannTheraPi Sees Potential Beyond FCD

Dr Luc-André Granier, President and co-founder of PannTheraPi, said the Phase 2a approval supports the company's broader development plans for treatments targeting rare neurological diseases.

He also said the company believes the therapeutic potential of targeting Panx1 could extend beyond FCD.

This means PannTheraPi is not viewing PTI5803 only as a potential treatment for one form of epilepsy. The company is also exploring whether its Panx1-focused approach could eventually be useful in other neurological conditions.

However, those potential applications will need to be evaluated through further research and clinical studies.

PTI5803 Is Based on an Extended-Release Formulation

PannTheraPi describes PTI5803 as an extended-release formulation of probenecid.

The formulation is being developed for use in FCD and could potentially be studied in other rare forms of epilepsy.

The company is using its understanding of the Panx1 channel to investigate how this approach could be applied to neurological diseases while aiming to preserve normal brain function.

What Comes Next for PTI5803?

The immediate next step is patient recruitment for the Phase 2a clinical trial.

The study will provide additional information about the safety and tolerability of PTI5803 in people with FCD. Researchers will also examine early signs of potential efficacy by looking at seizure frequency.

Patients who complete the main study can continue into the long-term open-label extension phase, giving the company additional information about longer-term treatment.

The Phase 2a results will help PannTheraPi determine the next stage of development for PTI5803 and assess its potential as a treatment for drug-resistant epilepsy associated with focal cortical dysplasia.

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