Pharmaceuticals
enterpriseTarget: Specialty drug developer
If you are a specialty drug developer dealing with the 70% drug resistance rate in orphan epilepsy — this project developed PTI5803 that inhibits seizures in over 80% of preclinical cases.
PANNEXIN · Project
First-in-class Drug Treatment for Ultra-Resistant Pediatric Orphan Epilepsy
healthTestedTRL 5
Imagine the brain has tiny gates that should stay closed, but in some children, these gates stay open and cause electrical storms called seizures. This project created a specialized key to lock those gates shut. It stops the seizures without messing with the child's ability to learn or think.
By the numbers
70%
Drug resistance rate in some orphan epilepsy syndromes
80%
Preclinical seizure inhibition rate
6 months
Minimum patient age for the formulation
1%
General population affected by epilepsy
The business problem
What needed solving
Up to 70% of patients with certain pediatric orphan epilepsies do not respond to current anti-seizure drugs. This creates a massive unmet medical need for a treatment that stops seizures without impairing cognitive development.
The solution
What was built
A first-in-class drug candidate, PTI5803, which is a reformulated version of probenecid designed as a long-release pediatric formulation.
Audience
Who needs this
Pediatric neurology clinicsOrphan drug pharmaceutical companiesNeurological research institutesClinical research organizations (CROs)
Business applications
Who can put this to work
Pediatric Healthcare
mid-sizeTarget: Children's hospital pharmacy
If you are a children's hospital pharmacy dealing with patients as young as 6 months who fail standard care — this project developed a long-release pediatric formulation to improve bioavailability.
Neurology Research
SMETarget: Biotech firm focusing on chronic neurological diseases
If you are a biotech firm dealing with the genesis of crises in multiple sclerosis or pain — this project developed a method to block the Panx1 channel which is involved in these conditions.
Frequently asked
Quick answers
What is the cost or pricing of the treatment?
Based on available project data, the specific cost or price per dose for PTI5803 is not provided.
Is the production ready for industrial scale?
The project has developed a long-release pediatric formulation, but based on available project data, the industrial manufacturing scale is not yet detailed.
What is the IP or licensing status of the drug candidate?
The drug candidate PTI5803 is described as a first-in-class product based on innovation from ERC Consolidators; however, specific patent numbers are not listed in the data.
What is the timeline for clinical availability?
The project period runs from 2023-10-01 to 2026-03-31, with the current goal being the first study in healthy volunteers.
How does the drug integrate into existing pediatric care?
It is designed as a long-release formulation adapted for children starting from 6 months of age to improve bioavailability.
Consortium
Who built it
The project is led by a single SME, Panntherapi, based in France. With a 100% industry ratio and a single partner, the project is streamlined for commercial translation, moving directly from academic discovery (via Paris Sciences et Lettres University) to clinical development.
How to reach the team
Contact Panntherapi in France
Next steps
Talk to the team behind this work.
Contact us to explore licensing opportunities for Panx1 channel blockers.
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