Avenue Therapeutics, Inc. announced that BAER-101, the Company?s potentially best-in-class selective GABA-A a2,3 positive allosteric modulator (?PAM?), significantly suppressed seizures in a translational animal model of absence epilepsy. BAER-101 underwent preclinical in vivo evaluation in SynapCell's Genetic Absence Epilepsy Rat from Strasbourg (?GAERS?) model of absence epilepsy. The GAERS model mimics behavioral, electrophysiological and pharmacological features of human absence seizures.

The GAERS model is a proven, early, informative indicator of efficacy in anti-seizure drug development, with high predictability of response in humans. In the model, BAER-101 demonstrated full suppression of seizure activity with a minimal effective dose of 0.3 mg/kg, PO. The effect was fast in onset and stable throughout the duration of testing.

BAER-101 has previously been shown to be safe with minimal side effects in clinical studies of hundreds of patients in a program targeting a different indication by AstraZeneca. The combination of safety, tolerability and efficacy in an established translationally relevant epilepsy model support BAER-101?s continued development. The selective potentiation of alpha 2,3-containing GABA-A receptors with minimal impact on a1 and a5-containing GABA-A receptors differentiates BAER-101 from competitors, and predicts a lower potential for sedation, motor incoordination, cognitive impairment and/or tolerance.

Further study of the anti-seizure properties of BAER-101 is being planned in patients with focal seizures and other seizure disorders for which current treatments are not fully efficacious. Avenue is developing BAER-101 via its subsidiary Baergic Bio for epilepsy disorders. BAER-101 is a PAM of a2,3 subunit-containing GABA-A receptors with minimal activity at a1 or a5-containing receptors, which are believed to mediate many of the issues impacting the medical use of benzodiazepines such as those noted with diazepam use (tolerance, dependence, abuse, sedation and impaired cognition).

As a result, BAER-101 may have the potential to treat epilepsy, anxiety and other disorders in which benzodiazepines are currently used, while minimizing the benzodiazepine associated adverse effects. BAER-101 was licensed in from AstraZeneca with a large safety database in over 700 patients and an efficacy signal in a ?subset of patients with anxiety.