Avenue Therapeutics, Inc. announced that the first patient has been dosed in the Phase 1b/2a clinical trial of AJ201 for the treatment of spinal and bulbar muscular atrophy ("SBMA"), also known as Kennedy's Disease. A recent study used genetic analysis to estimate disease prevalence of 1:6,887 males.(1) AJ201 is currently the lead drug candidate in the clinic for SBMA and enrollment in the trial is expected to be complete by the end of 2023 or early 2024 with potential topline data in 2024. Additionally, compelling preclinical data in a mouse model showed efficacy signals, including improvement in motor function, robust degradation of mutant androgen receptors ("AR"), a disease-signaling protein, and activation of the Nrf1 and Nrf2 pathways.

In this Phase 1b/2a clinical trial, the company aim to demonstrate how AJ201's novel, multi-fold mechanism of action reduces accumulation of mutant AR aggregates to potentially decrease neuroinflammation, protect cells from oxidative stress, and ultimately, improve clinical outcomes for SBMA patients. The 12-week, multicenter, randomized, double-blind Phase 1b/2a clinical trial of AJ201 is expected to enroll approximately 24 patients, randomly assigned to AJ201 (600 mg/day) or placebo. The primary endpoint of the study is to assess safety and tolerability of AJ201 in subjects with clinically and genetically defined SBMA.

Secondary endpoints include pharmacodynamic data measuring change from baseline in mutant AR protein levels in skeletal muscle and changes in the fat and muscle composition as seen on MRI scans, which are believed to be biomarkers indicating likelihood for longer term clinical improvement. Further details about this study can be found at ClinicalTrials.gov (Identifier: NCT05517603).