ENX-101

Seizures are typically provoked by uncontrolled excitatory neurotransmission.¹ GABA, the primary inhibitory neurotransmitter in the brain, acts as a counterbalance to excitatory neurotransmission.² Nonselective GABA_A receptor modulators like benzodiazepines are highly effective in controlling seizures, but long-term use is generally limited by a rapid loss of efficacy and significant side effects, including sedation, cognitive impairment, and motor impairment. These undesirable effects largely result from activation of α1 subunit–containing GABA_A receptors.³

ENX-101 enhances neurotransmission through GABA_A receptors
containing α2, α3, or α5 subunits while blocking α1.⁴

This profile is expected to facilitate sustained efficacy with chronic dosing and a favorable safety and tolerability profile that minimizes many of the risks and side effects associated with α1-mediated GABAergic transmission. The bioavailability and receptor occupancy of ENX-101 were engineered to deliver a best-in-class pharmacologic profile. It may also be used synergistically with other antiseizure medications.⁴

In clinical studies, ENX-101 was well-tolerated with once-daily, oral dosing without a need for dose titration and demonstrated evidence of persistent target engagement predictive of antiseizure efficacy. The Phase 2 study (ENACT) is designed to assess ENX-101 for the treatment of focal seizures.⁴

For more information on the ENACT trial, please click here: https://clinicaltrials.gov/ct2/show/NCT05481905