Avenue Therapeutics, Inc.

Company focus

Avenue Therapeutics is a specialty pharmaceutical company developing therapies for neurologic and related diseases. Its current development programs focus on ATX-04 and IV tramadol. The company previously owned BAER-101 (a GABA-A PAM) through Baergic and sold that asset to Axsome Therapeutics in 2025.

Product candidates

• ATX-04

  • Agent: clenbuterol, a selective β2-adrenergic agonist.
  • Indication: Initial focus on Pompe disease as an adjunct to enzyme replacement therapy (ERT).
  • Development status: Pursuing a late-stage development path. In February 2026 Avenue secured an exclusive worldwide license from Duke University for patents and know-how related to ATX-04 for lysosomal storage diseases and plans to discuss pivotal trial design with the FDA.

• IV tramadol

  • Agent: intravenous tramadol (U.S. trademarked program).
  • Indication: Phase 3 program for postoperative acute pain.
  • Development status: The program has experienced multiple FDA communications, including CRLs and advisory committee activity. A final Phase 3 safety protocol was agreed in January 2024. Initiation of a safety study depends on obtaining financing or a partnership.

• BAER-101 (AXS-17)

  • Status: Sold to Axsome Therapeutics on November 5, 2025.
  • Rights and next steps: Axsome acquired worldwide rights and intends to evaluate AXS-17 for epilepsy. Under the Baergic Agreement, Avenue retains contingent rights to milestone and royalty payments and is expected to receive roughly 74% of future Baergic payments allocated to former Baergic stockholders.

Key licensing and transaction activity

• ATX-04 (Duke University): Exclusive worldwide license for clenbuterol patents and know-how covering lysosomal storage diseases; the agreement includes an upfront payment, development and regulatory milestones, and a tiered low single-digit royalty on net sales.
• AnnJi / AJ201: License terminated in 2025 under a License Termination and Program Transfer Agreement (April 2025). Avenue repurchased AnnJi’s shares and may receive contingent payments; it agreed to a 48‑month non‑compete with respect to AJ201 in specified territories.
• Baergic / BAER-101: Fortress-related stock contribution in 2022 facilitated initial acquisition of Baergic; Baergic was sold to Axsome in 2025 with Avenue retaining contingent rights to future consideration (approximately 74% of payments earmarked for former Baergic stockholders).
• Revogenex / IV tramadol: Fortress-originated license to IV tramadol rights (exclusive in most territories, with certain exceptions); the program has an extended history of FDA interactions and associated development milestones.

Corporate structure and control

Avenue is a majority-controlled subsidiary of Fortress Biotech, Inc. Fortress holds a controlling voting interest through Class A Preferred Stock and maintains governance influence via related agreements, management services, and rights to certain equity-related grants and royalties. Shared personnel and director relationships with Fortress affiliates create potential conflicts of interest.

Intellectual property

• IV tramadol: A broad U.S. patent family and continuations from the Revogenex licenses, with several patents expiring between 2032 and 2037 subject to potential extensions.
• ATX-04: Includes the Duke “478” family and related provisional filings, with expected expiry around 2031 absent term extensions.
• Regulatory pathways: The company may pursue the 505(b)(2) pathway for some candidates, with possible exclusivity outcomes (3‑year or 5‑year periods), pediatric exclusivity, and orphan designations where applicable.

Manufacturing and operations

Avenue does not own manufacturing facilities and relies on contract development and manufacturing organizations (CDMOs).

• IV tramadol is produced by Polpharma with a subcontractor under cGMP. Avenue has minimum purchase commitments, per‑dose fees, royalties, and a milestone payment tied to FDA approval.
• ATX-04 supply arrangements are being established with a CDMO, and the company intends to qualify backup manufacturers as needed.

Market, reimbursement, and commercialization

Avenue has no approved products and no product revenues to date. Future revenue will depend on regulatory approvals, commercialization strategies, and outcomes of collaborations or licensing arrangements. Approved products would face standard payer coverage, reimbursement, pricing, and formulary challenges and competition from established therapies and generics.

Financial and operating snapshot (as of 12/31/2025)

• Employees: 1 full‑time employee
• Cash and cash equivalents: $2.9 million
• Accumulated deficit: Approximately $105.5 million
• Revenue: None reported from product sales
• Going concern: The company reports substantial doubt about its ability to continue as a going concern and is pursuing additional funding or strategic transactions
• Public market status: Delisted from Nasdaq; trading on the OTC Pink Open Market under the ticker ATXI (per filings in 2025)
• Financing needs: Ongoing need for additional capital via equity/debt financings, collaborations, asset dispositions, or other strategic transactions

Customers and commercial footprint

Avenue has no marketed products and does not report customers or current revenue from product sales.

Governance and risk posture

The company depends heavily on third‑party relationships (licensing partners, CDMOs, CROs) and on Fortress for governance and potential funding support. The business carries standard biotech risks, including development and regulatory uncertainty and no guaranteed path to profitability or timely approvals.

Summary

Avenue Therapeutics is a Fortress‑controlled specialty pharma company advancing ATX‑04 (clenbuterol) for Pompe disease adjunctive use and IV tramadol for postoperative pain. The company has divested BAER‑101, retains contingent rights to future payments, operates with minimal internal resources, relies on external partners for development and manufacturing, and requires additional funding to execute its development plans.