Drug intelligence / Profile preview

ENX-101

Development stage
Phase 2
Lead developer
Engrail Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

ENX-101 is a subtype-selective positive allosteric modulator (PAM) of the gamma-aminobutyric acid type A (GABA A) receptor, specifically targeting receptors containing the α2, α3, and α5 subunits while lacking activity at the α1 subunit. This selectivity is designed to maximize antiseizure efficacy while minimizing side effects such as sedation and tolerance that are commonly associated with non-selective GABA A modulators like benzodiazepines. Preclinical studies have demonstrated that ENX-101 is highly effective in rodent models of epilepsy and does not cause motor impairment. In clinical trials, it has shown a favorable safety and pharmacokinetic profile in healthy volunteers, with mild and transient adverse events. ENX-101 is being developed primarily as an adjunctive treatment for focal epilepsy in patients who do not achieve adequate seizure control with existing medications[1][2][3][4][5][6][8].

02

Targets

α5-GABAAR (Gamma-aminobutyric acid type A receptor alpha5beta2gamma2 subtype (GABAA receptor alpha5beta2gamma2 subtype))GABRA2 (Gamma-aminobutyric acid receptor subunit alpha 2)

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