Avenue Therapeutics, Inc. announced the completion of the last patient?s final visit in the Company?s Phase 1b/2a clinical trial of AJ201 for the treatment of spinal and bulbar muscular atrophy (?SBMA?), also known as Kennedy's Disease. Topline data are expected to be reported mid-year 2024. The 12-week, multicenter, randomized, double-blind Phase 1b/2a clinical trial of AJ201 enrolled 25 patients, randomly assigned to AJ201 (600 mg/day) or placebo (3:1).

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 pharmacokinetic and pharmacodynamic data measuring change from baseline in mutant AR protein levels in skeletal muscle and changes from baseline in expression of Nrf2-activated genes in skeletal muscle. Exploratory objectives of the study include changes in the fat and muscle composition as seen on MRI scans.

These endpoints 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). In April 2024, Avenue hosted a virtual key opinion leader (?KOL?) event highlighting expert perspectives on SBMA.

The event featured Christopher Grunseich, M.D., Lasker Clinical Research Scholar and Investigator and Head of the Inherited Neuromuscular Diseases Unit at the National Institute of Neurological Disorders and Stroke, and Tahseen Mozaffar, M.D., Professor of Neurology, Pathology and Laboratory Medicine, Director of the Division of Neuromuscular Diseases and Director of the ALS and Neuromuscular Center at the University of California, Irvine. The two featured speakers discussed the characteristics and treatment landscape of SBMA, as well as the trial design and potential of AJ201 in SBMA.