Avenue Therapeutics, Inc.

What they do

Avenue is a specialty pharmaceutical company focused on therapies for neurologic diseases. Its current product candidates are ATX-04, a selective beta-2 adrenergic agonist licensed from Duke University in February 2026 for lysosomal storage diseases with an initial focus on Pompe disease as an adjunct to enzyme replacement therapy, and IV tramadol, a Schedule IV opioid for post-operative acute pain that the company has been developing since before its 2017 IPO. It previously held BAER-101 for epilepsy and panic disorders through subsidiary Baergic Bio until that business was acquired by Axsome Therapeutics on November 5, 2025, and it terminated its AnnJi license for AJ201 in 2025. The company is a majority-controlled subsidiary of Fortress Biotech and has no drug products for sale.

Revenue drivers

• Product sales — None. The company states it currently has no drug products for sale; quarterly revenue was $0.00 in the quarters ended 2025-09-30 and 2026-06-30.
• AXS-17 (formerly BAER-101) milestone and royalty rights — After the Baergic disposition to Axsome, Avenue and former Baergic stockholders are eligible for additional payments and royalties tied to development, regulatory and commercial milestones and net sales, with no control over the program.
• AJ201 milestone and royalty rights — Following AnnJi's March 2025 termination notice for the AnnJi License Agreement, potential economics from AJ201 consist solely of contingent milestone and royalty payments dependent on AnnJi's efforts.
• ATX-04 (clenbuterol) — Licensed from Duke in February 2026; Avenue owes development, regulatory and commercial milestones plus a tiered low single-digit royalty on future net sales, so it is a cost and future revenue opportunity rather than a current revenue source.

Recent performance

Revenue was $0.00 in 2024 and $1.4 million in 2025, with the 2025 amount not recurring in recent quarters ($0.00 for the quarters ended 2025-09-30 and 2026-06-30). Net income improved from a $11.7 million loss in 2024 to a $2.9 million loss in 2025, and operating cash use fell from $9.0 million in 2024 to $1.8 million in 2025. As of June 30, 2026, total assets were $2.0 million, total liabilities $1.4 million, stockholders' equity $569,000, and cash and cash equivalents $1.9 million, down from $2.855 million at December 31, 2025. The balance sheet shows $882,000 of accounts payable and accrued expenses owed to a related party at June 30, 2026. Diluted EPS improved from -$15.79 in 2024 to -$0.93 in 2025.

Strategy

The company's stated direction is to identify and develop new product candidates after losing AJ201 and BAER-101, its two prior primary candidates. In February 2026 it exclusively licensed clenbuterol (ATX-04) from Duke University for lysosomal storage diseases and intends to advance it through a late-stage clinical development program leveraging existing human safety and efficacy data, initially for Pompe disease as an adjunct to enzyme replacement therapy. It continues to hold IV tramadol for post-operative acute pain. Its economics from AJ201 and AXS-17 now depend entirely on the efforts and decisions of AnnJi and Axsome. The 10-K risk factors state the company has substantial doubt about its ability to continue as a going concern and will need substantial additional funding.

Risks

• Pipeline concentration and loss of prior candidates — The company states its remaining portfolio is limited to two product candidates after terminating the AnnJi license for AJ201 and disposing of Baergic and rights to BAER-101.
• Going-concern and funding need — The 10-K discloses substantial doubt about the ability to continue as a going concern, and $1.9 million of cash at June 30, 2026 against continuing development spending.
• Dependence on third parties for milestone and royalty value — Any future economics from AJ201 and AXS-17 depend on AnnJi and Axsome achieving development, regulatory and commercial milestones, and those counterparties may delay, reprioritize or discontinue the programs.
• Listing and liquidity constraints — The company received delisting notices on 2025-03-17 and 2024-11-29, and its common stock trades on OTC Markets rather than a national exchange.

Outlook

Management expects to advance ATX-04 through late-stage clinical development for Pompe disease as an adjunct to enzyme replacement therapy, and to continue development or partnering of IV tramadol. It states that future value from AJ201 and AXS-17 is limited to contingent milestones and royalties controlled by AnnJi and Axsome. The 10-K states the company will need substantial additional funding, without which it could be forced to delay, reduce or eliminate development programs. No revenue guidance or product revenue is disclosed for the current period.