Company Summary: Avenue Therapeutics, Inc.

Overview

• Name: Avenue Therapeutics, Inc.
• Incorporation: Delaware, 2015
• Headquarters: 1111 Kane Concourse, Suite 301, Bay Harbor Islands, FL 33154
• Telephone: (781) 652-4500
• Website: www.avenuetx.com

Business Focus

Avenue Therapeutics is a specialty pharmaceutical company dedicated to the development and commercialization of therapies for neurologic diseases. The company primarily focuses on developing drug candidates for conditions such as spinal and bulbar muscular atrophy (SBMA), epilepsy, panic disorders, and acute pain management.

Core Product Candidates

1. AJ201

   • Treatment for SBMA (Kennedy's Disease), a rare inherited neuromuscular disorder affecting mainly men.
   • Designed to degrade mutant androgen receptor protein and activate cellular stress protection pathways (Nrf1, Nrf2).
   • Currently in a Phase 1b/2a clinical trial in the U.S., with initial dosing completed in July 2023.
   • Market: No FDA-approved treatments for SBMA; potential to treat other polyglutamine (polyQ) diseases such as Huntington’s and spinocerebellar ataxias.
   • Regulatory Designation: Orphan Drug designation in the U.S.

2. IV Tramadol

   • A parenteral version of tramadol, an opioid analgesic, for postoperative acute pain.
   • Under development due to unmet clinical need for safer IV opioid formulations.
   • Completed multiple Phase 3 efficacy trials and an open-label safety study.
   • Disputed license with Revogenex; final approval depends on further FDA review and ongoing clinical trials.
   • Regulatory Status: NDA submission in 2019, but received two Complete Response Letters from the FDA. Final agreement reached in January 2024 for a Phase 3 safety study to support further approval.

3. BAER-101 (formerly AZD7325)

   • A selective GABA-A α2/3 PAM (positive allosteric modulator).
   • Developed for epilepsy and panic disorders.
   • Acquired via the October 2022 acquisition of Baergic Bio, Inc.
   • Demonstrated efficacy in preclinical seizure models with minimal side effects.
   • In August 2023, preclinical data showed full suppression of seizure activity in a rat model.

Strategic Objectives

• Develop AJ201 for SBMA and possibly other polyQ neurodegenerative diseases.
• Obtain FDA approval for IV tramadol for managing postoperative pain.
• Develop BAER-101 for epilepsy and panic disorders.
• Expand and protect intellectual property rights related to these candidates.
• Maintain regulatory compliance and seek efficient approval pathways.
• Build and expand commercial capabilities upon approval.

Financial and Operational Data

• Employees: 2 full-time employees (as of December 31, 2024).
• Customer Base: No commercialized products yet; no revenue.
• Revenue: None to date.
• Market Capitalization (Equity Value): Approx. $3.91 million (last business day of the second fiscal quarter, based on market value of voting stock held by non-affiliates).
• Shares Outstanding: 3,149,702 (as of March 28, 2025).
• Revenue: No product sales; operating losses expected to continue.
• Financial Condition: As of December 31, 2024, cash and equivalents of $2.6 million, with an accumulated deficit of $102.6 million.

Business Model

• R&D focused with key assets in clinical development stages.
• Licensing in proprietary technology (AJ201 from AnnJi; BAER-101 from AstraZeneca).
• Collaborations with third-party manufacturing and clinical research organizations.
• No current product revenue; future revenue depends on successful approval, commercialization, and market penetration of product candidates.

Note: The company is in the development stage with no approved products and is primarily relying on licensing, ongoing clinical trials, and regulatory approvals to reach commercialization.