Core Viewpoint - Avenue Therapeutics has completed the last patient's final visit in the Phase 1b/2a clinical trial of AJ201 for spinal and bulbar muscular atrophy (SBMA), with topline data expected mid-year 2024 [1][2]. Company Overview - Avenue Therapeutics, Inc. is a specialty pharmaceutical company focused on developing therapies for neurologic diseases, currently working on three assets including AJ201, BAER101, and IV tramadol [8]. - The company is headquartered in Miami, FL, and was founded by Fortress Biotech, Inc. [8]. Clinical Trial Details - The Phase 1b/2a clinical trial of AJ201 enrolled 25 patients, with a random assignment of 600 mg/day of AJ201 or placebo in a 3:1 ratio [2]. - The primary endpoint is to assess the safety and tolerability of AJ201 in subjects with clinically and genetically defined SBMA, while secondary endpoints include pharmacokinetic and pharmacodynamic data [2]. - The trial aims to evaluate changes in mutant AR protein levels and expression of Nrf2-activated genes in skeletal muscle, with exploratory objectives including MRI scans to assess fat and muscle composition [2]. Drug Information - AJ201 is a first-in-class asset designed to treat SBMA by degrading the abnormal androgen receptor protein and stimulating protective cellular pathways [5]. - It has shown an excellent safety and pharmacokinetic profile in a Phase 1 study involving 72 healthy volunteers [5]. - AJ201 has received Orphan Drug Designation from the FDA for multiple polyQ diseases, including SBMA [6]. Disease Background - SBMA is a rare, X-linked genetic neuromuscular disease primarily affecting men, caused by a mutation in the androgen receptor leading to muscular atrophy [4]. - The estimated prevalence of SBMA is 1 in 6,887 males, and currently, there are no FDA or EMA approved treatments available [4]. Expert Insights - Avenue hosted a virtual key opinion leader event featuring experts discussing the characteristics and treatment landscape of SBMA, as well as the trial design and potential of AJ201 [3].

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number 001-38114 AVENUE THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 47-4113275 (State or othe ...

Exhibit 99.1 Avenue Therapeutics Reports First Quarter 2024 Financial Results and Recent Corporate Highlights - Raised a total of $9.4 million in gross proceeds from warrant exercise transactions since January 2024 – Miami, FL – May 15, 2024 – 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 reported financial results and recent corporate highl ...

– Raised a total of $9.4 million in gross proceeds from warrant exercise transactions since January 2024 – MIAMI, May 15, 2024 (GLOBE NEWSWIRE) -- 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 reported financial results and recent corporate highlights for the first quarter ended March 31, 2024. "The first quarter was incredibly productive for ...

MIAMI, April 29, 2024 (GLOBE NEWSWIRE) -- 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 entry into definitive agreements for the immediate exercise of certain outstanding warrants to purchase an aggregate of 689,680 shares of the Company's common stock. The exercised warrants are comprised of warrants to purchase shares of commo ...

MIAMI, April 24, 2024 (GLOBE NEWSWIRE) -- 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 that it will effect a 1-for-75 reverse split of its issued and outstanding common stock. Avenue expects its common stock to begin trading on a split-adjusted basis on the Nasdaq Capital Market as of the commencement of trading on April 26, 2024 w ...

Table of Contents (Title of Class) Trading Symbol(s) (Name of exchange on which registered) Common Stock, par value $0.0001 per share ATXI Nasdaq Capital Market UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Fiscal Year Ended December 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Transition Period from to . Commissi ...

Exhibit 99.1 Avenue Therapeutics Reports Full Year 2023 Financial Results and Recent Corporate Highlights - Enrollment completed in Phase 1b/2a clinical trial of AJ201 for spinal and bulbar muscular atrophy; topline data expected in second quarter of 2024 - - Presented positive BAER-101 preclinical data at American Epilepsy Society (AES) and American Society for Experimental Neurotherapeutics (ASENT) Annual Meetings and in publication Drug Development Research - - Final agreement reached with FDA on study d ...

- Enrollment completed in Phase 1b/2a clinical trial of AJ201 for spinal and bulbar muscular atrophy; topline data expected in second quarter of 2024 - - Presented positive BAER-101 preclinical data at American Epilepsy Society (AES) and American Society for Experimental Neurotherapeutics (ASENT) Annual Meetings and in publication Drug Development Research - - Final agreement reached with FDA on study design and analysis approach for Phase 3 safety study of IV tramadol – MIAMI, March 18, 2024 (GLOBE NEWSWIR ...

MIAMI, March 15, 2024 (GLOBE NEWSWIRE) -- 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 that by decision dated March 11, 2024, the Nasdaq Hearings Panel granted the Company's request for an extension to evidence compliance with all applicable criteria for continued listing on The Nasdaq Capital Market, including the $1.00 bid price ...