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vormatrigine
- Development stage
- Phase 3
- Lead developer
- Praxis Precision Medicines
- Modality
- Classical Binding Small Molecules → Small Molecules
- Administration
- Oral
01
Overview
Vormatrigine is an oral small molecule antiepileptic drug developed by Praxis Precision Medicines. It acts as a next-generation, functionally selective voltage-gated sodium channel inhibitor that precisely targets the hyperexcitable state of sodium channels in the brain. Vormatrigine is being investigated as a once-daily treatment for adult focal onset seizures and generalized epilepsy. Preclinical and clinical data indicate superior selectivity for disease-state NaV channel hyperexcitability compared to standard therapies and demonstrate unprecedented potency in translational seizure models. Vormatrigine has shown consistent safety and tolerability profiles in Phase 1 studies with no significant food effect on absorption[1][3][8].
- Other names
- vormatrigine3-(ethoxydifluoromethyl)-6-(5-fluoro-6-(2,2,2-trifluoroethoxy)pyridin-3-yl)-[1,2,4]triazolo[4,3-a]pyridine
02
Targets
NaV (Voltage-gated sodium channels)
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