ATXI Avenue Therapeutics Inc

Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today 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 α2 and α3 subtypes more than α1 and α5, 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 α2,3 and not for α1 or α5, a pharmacology consistent with anti-seizure activity that avoids the adverse side effects common to the GABAA positive allosteric modulators (PAM) class. Specifically, the α1-subtype of GABAAR is associated with dizziness and somnolence in both animal and human studies, and the α5-subtype of GABAAR is thought to play a key role in synaptic plasticity, cognition, and memory, suggesting that engagement of α5 risks anti-cognitive effects. The pharmacology of BAER-101 lacks activity at both the α1- and α5-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.

“Epilepsy remains one of the most prevalent neurological diseases worldwide, with a population of approximately 65 million patients, but there remains a great unmet need for a safe and effective treatment option that suppresses seizure activity without drug resistance or harmful side effects such as sedation, cognitive impairment, ataxia and addiction,” said Alexandra MacLean, M.D., Chief Executive Officer of Avenue. “The preclinical data published in Drug Development Research demonstrate BAER-101’s ability to fully suppress seizures in the GAERS model, a translational animal model for anti-seizure drug development with a documented high predictability of response in humans. Additionally, BAER-101 demonstrated full efficacy with a minimal effective dose of 0.3 mg/kg, indicating that BAER-101 is the most potent compound yet reported in this model, as well as the first to show that a GABA PAM that is selective for the α2 and α3-subtype GABAARs is active in this model. Building on BAER-101’s proven safety profile in over 700 patients and healthy human volunteers, these first-in-class preclinical findings support BAER-101’s continued development in a Phase 2a trial.”

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.

About Avenue TherapeuticsAvenue Therapeutics, Inc. (Nasdaq: ATXI) is a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases. It is currently developing three assets including AJ201, a first-in-class asset for spinal and bulbar muscular atrophy, BAER-101, an oral small molecule selective GABAA α2, α3 receptor positive allosteric modulator for CNS diseases, and IV tramadol, which is in Phase 3 clinical development for the management of acute postoperative pain in adults in a medically supervised healthcare setting. Avenue is headquartered in Miami, FL and was founded by Fortress Biotech, Inc. (Nasdaq: FBIO). For more information, visit www.avenuetx.com.

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Contact: Jaclyn JaffeAvenue Therapeutics, Inc. (781) 652-4500ir@avenuetx.com