Avenue Therapeutics, Inc. announced the publication of preclinical in vivo data in Drug Development Research highlighting BAER-101?s full suppression of seizure activity using the Genetic Absence Epilepsy Rats from Strasbourg (?GAERS?) model of absence epilepsy. Data published showcase BAER-101?s ability to selectively target GABAA a2 and a3 subtypes more than a1 and a5, potentially improving anti-convulsant and anxiolytic activity while minimizing the risk of tolerance and abuse associated with existing treatments in this drug class. The publication describes the extent of anti-seizure activity of BAER-101 in the GAERS model, a widely used and translationally relevant animal model.

The study demonstrated full suppression of seizure activity with a minimal effective dose (MED) of 0.3 mg/kg. The effect of BAER-101 was fast in onset and stable throughout the duration of testing. Results from the testing showed that the number of spike-wave discharges were dose-dependently reduced by BAER-101, and no adverse safety events were observed up to a dose 300x the MED.

BAER-101 is the first clinical candidate which is selective for only a2,3 and not for a1 or a5, a pharmacology consistent with anti-seizure activity that avoids the adverse side effects common to the GABAA positive allosteric modulators (PAM) class. Specifically, the a1-subtype of GABAAR is associated with dizziness and somnolence in both animal and human studies, and the a5-subtype of GABAAR is thought to play a key role in synaptic plasticity, cognition, and memory, suggesting that engagement of a5 risks anti-cognitive effects. The pharmacology of BAER-101 lacks activity at both the a1- and a5-subtypes of GABAAR, and these findings indicate that BAER-101?s on-target engagement with a selective subset of synaptic GABAARs is sufficient to suppress absence seizures while avoiding adverse side effects common to the GABAA PAM class.

Subject to obtaining the necessary financing, which could be provided through a strategic partnership, Avenue plans to initiate a Phase 2a clinical trial of BAER-101 to further study its anti-seizure properties in patients with common or rare epilepsies.