ENX-101 is an orally active partial positive allosteric modulator (PAM) of the (GABAA) receptor. It selectively targets the α2β2γ2L (EC50= 0.76 nM), α2β3γ2L (EC50= 0.61 nM), α3 (EC50= 1.97 nM), and α5 (EC50= 0.85 nM) subunits of the GABA receptor. ENX-101 demonstrates anticonvulsant activity in various animal models.

ENX-101 (0.01 nM-10 μM) effectively enhances currents generated by GABA in Chinese hamster ovary (CHO) cells with receptors containing α2, α3, or α5 subunits, while its potentiation effect on receptors with α1 subunits is minimal.

Administering ENX-101 (30-300 mg/mL, intraperitoneally, 0.25 hours before electrical stimulation) in the hybrid adult male CF-1 mouse model (20-35 g) effectively protects most animals stimulated with 32 mA current, though fewer at 44 mA. In an epileptic rat model, ENX-101 (1-100 mg/kg, orally, 2 hours before stimulation) markedly reduces behavioral seizures in a dose-dependent manner and shortens the mean post-discharge duration in the amygdala and cortex. For male Strasbourg Wistar genetic absence epilepsy rats (GAERS), ENX-101 (0.075-100 mg/kg, orally, single dose) significantly decreases total spike-wave discharges (SWD) during the post-treatment observation period (10-130 minutes). Furthermore, a single oral dose of ENX-101 (10-100 mg/kg) in a rat model of epilepsy does not cause any motor impairments at anticonvulsant doses.