Business Description

Overview Avenue Therapeutics, Inc. (“Avenue” or the “Company”) is a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases. Our current product candidates are ATX-04, a selective β2-adrenergic agonist for Pompe disease, intravenous tramadol (“IV tramadol”), a schedule IV opioid for the treatment of post-operative acute pain and previously, through November 5, 2025, BAER-101 for the treatment of epilepsy and panic disorders. In February 2026, we announced the entry into an exclusive license agreement with Duke University for patents and know-how relating to clenbuterol (referred to as ATX-04), a selective β2-adrenergic agonist, for the treatment of lysosomal storage diseases with a focus on Pompe disease. In November 2022, we completed a Stock Contribution Agreement, dated May 11, 2022 (the “Contribution Agreement”) with Fortress Biotech, Inc (“Fortress”) to acquire the shares in Baergic Bio, Inc. (“Baergic”), which was developing BAER-101, a novel α2/3–subtype-selective gamma-aminobutyric acid ("GABA") A positive allosteric modulator (“PAM”). As a result, Baergic was a majority-controlled and owned subsidiary company of Avenue. On November 5, 2025, we announced we had entered into an agreement for Baergic to be acquired by Axsome Therapeutics, Inc. (“Axsome”) including the global rights to BAER-101 (also known as AZD7325), a novel oral GABAA α2,3 subtype-selective receptor positive allosteric modulator (PAM). BAER-101 was originally licensed by Baergic from AstraZeneca AB and will be referred to as AXS-17 by Axsome going forward. As described in further detail below, we and the other former stockholders of Baergic will be eligible to receive from Axsome certain additional payments and royalties based on development, regulatory, and commercial milestones and net sales. Axsome intends to evaluate AXS-17 as a potential treatment for epilepsy. We have been developing IV tramadol since inception of the Company and prior to our initial public offering in 2017. As used throughout this filing, the words “we”, “us” and “our” may refer to Avenue individually or, until November 5, 2025, together with our former subsidiary, Baergic, each as dictated by context. We are a majority-controlled subsidiary of Fortress. Recent Developments ATX-04 License On February 18, 2026, Avenue entered into a license agreement with Duke University (“Duke”), pursuant to which Avenue obtained an exclusive worldwide license (the "ATX-04 License") from Duke to certain patents and know-how pertaining to clenbuterol for the treatment of lysosomal storage diseases. Under the ATX-04 License, Avenue made an upfront payment and reimbursed certain patent expenses to Duke and has an obligation to make development, regulatory, and commercial milestone payments upon the achievement of certain milestones. In addition, Avenue is obligated to pay a tiered low single-digit royalty on future net sales of ATX-04. Avenue intends to advance ATX-04 through a late-stage clinical development program leveraging existing human safety and efficacy data, with an initial focus on treating Pompe disease as an adjunct to enzyme replacement therapy (“ERT”). AJ201 Termination On February 28, 2023, Avenue entered into a license agreement with AnnJi Pharmaceutical Co. Ltd. ("AnnJi"), pursuant to which Avenue obtained an exclusive license (the "AnnJi License Agreement") from AnnJi to the intellectual property rights pertaining to the molecule known as JM17, which activates Nrf1 and Nrf2, enhances androgen receptor degradation and underlies AJ201, a clinical product candidate currently in a Phase 1b/2a clinical trial in the U.S. for the treatment of spinal and bulbar muscular atrophy (“SBMA”, also known as Kennedy's Disease).…