Drug intelligence / Profile preview

PRX-2904

Development stage
Preclinical
Lead developer
Praxis Precision Medicines
Modality
Small Molecules
Administration
Oral
01

Overview

PRX-2904 is an investigational **small molecule** inhibitor of the sodium-activated potassium channel **KNa1.1**, encoded by **KCNT1**. It was developed by **Praxis Precision Medicines** as a selective blocker of KCNT1 channel activity for the treatment of **KCNT1-related epilepsies**, including developmental and epileptic encephalopathies driven by gain-of-function KCNT1 mutations. Preclinical reports describe oral activity, selectivity for KNa1.1, and reversal or suppression of abnormal electrophysiologic and seizure phenotypes in KCNT1 mutant mouse models, supporting its use as a targeted therapy for epilepsy associated with KCNT1 hyperactivity. Publicly available evidence indicates the program has remained in **preclinical development**; no brand name or marketed formulation has been identified.

02

Targets

KCNT1 (Potassium channel subfamily T member 1)

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