Neurostrategix

A Verified Framework for Preclinical Antiseizure Drug Discovery

2025

In preclinical epilepsy therapy screening, it is difficult to know which animal models can be most predictive of clinical success. This study establishes a framework to demonstrate that the models with the highest clinical concordance were: maximal electroshock test and 6 Hz test, followed closely by the corneal kindled mouse.

A Novel Positive Allosteric Modulator of the GABA-A Receptor - In Vivo Studies in Mice and Rats

2025

ENX‑101 targets specific GABA receptor types (α2, α3, and α5 subunits) linked to seizure control, avoiding the α1 subtype responsible for strong sedation. This study reports the in vitro and in vivo studies to profile ENX-101, including activity in amygdala-kindled rats and the mouse 6 Hz test.

Targeting Brain Cholesterol Metabolism - A First-In-Class Treatment for Dravet Syndrome

2023

Soticlestat is a first-in-class therapy under clinical development for pediatric epilepsy, with a particular focus on Lennox Gastaut and Dravet Syndromes. This preclinical study evaluated the anticonvulsant and disease-modifying potential in a mouse model of infection-induced acquired seizures. Notably, soticlestat demonstrated robust suppression of seizure presentation in vivo.

In Vitro to In Vivo Screening Across Model Systems Uncovers a New GABA Receptor Modulator for Seizures

2020

New approach methods (NAMs) are becoming useful to reduce animal testing. NAMs can uncover new therapeutic options at molecular targets of relevance to epilepsy. We thus assessed the feasibility of a multiorganismal compound library screening pipeline to identify a small molecule GABA-A receptor modulator.

Nucleic acid-based disease modification therapy for a rare epilepsy condition

2026

Dentatorubral-pallidoluysian atrophy (DRPLA) is a fatal neurodegenerative disease caused by a CAG repeat expansion in the atrophin-1 (ATN1) gene and is thought to arise from toxic gain-of-function mechanisms. This study evaluated the disease modifying impact of a novel antisense oligonucleotide (ASO) in a mouse DRPLA model.

Therapies

A Verified Framework for Preclinical Antiseizure Drug Discovery

A Novel Positive Allosteric Modulator of the GABA-A Receptor - In Vivo Studies in Mice and Rats

Targeting Brain Cholesterol Metabolism - A First-In-Class Treatment for Dravet Syndrome

In Vitro to In Vivo Screening Across Model Systems Uncovers a New GABA Receptor Modulator for Seizures

Nucleic acid-based disease modification therapy for a rare epilepsy condition