BAER-101 is a potentially best-in-class targeted therapy in development for the treatment of epilepsy and acute anxiety.
BAER-101 is a positive allosteric modulator (PAM) of α2,3 subunit‐containing GABAA receptors with minimal activity at α1 or α5‐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, impaired cognition). As a result, BAER-101 may have 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 from Astra Zeneca with a large safety database in over 700 patients and an efficacy signal in a subset of patients with anxiety.
BAER-101 underwent preclinical in vivo evaluation in SynapCell’s 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. Effects of compounds in this model have been shown to predict efficacy and safety in patients with epilepsy. In the model, BAER-101 demonstrated full suppression of seizure activity, and the effect was fast in onset and stable throughout the duration of testing.
The combination of documented safety and tolerability in hundreds of patients and the efficacy in an established translationally relevant epilepsy model support BAER-101’s continued development. Further study of the antiseizure properties of BAER-101 in a Phase 2a clinical trial is being planned in patients with focal epilepsy and other seizure disorders for which current treatments are not fully efficacious.