Avenue Therapeutics Announces Last Patient Last Visit in Phase 1b/2a Clinical Trial of AJ201 for the Treatment of Spinal and Bulbar Muscular Atrophy (Kennedy's Disease)

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].