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,  announced that all patients have been enrolled 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. AJ201 is currently the lead drug candidate in the clinic for SBMA, and topline data are anticipated in the second quarter of 2024.

“We are pleased with the speed of enrollment in our Phase 1b/2a study evaluating AJ201 for the treatment of SBMA, reflecting the large unmet need in this debilitating neurodegenerative disease,” said Alexandra MacLean, M.D., Chief Executive Officer of Avenue. “We are intently focused on advancing this much needed drug for patients and their caregivers, and we look forward to reporting topline data in the second quarter of 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. The primary endpoint of the study is to assess safety and tolerability of AJ201 in subjects with clinically and genetically defined SBMA. Although the disease is slow in progression, limiting the potential to demonstrate clinical efficacy over 12 weeks, Avenue has included as secondary endpoints various biomarkers addressing proof of target engagement, which could reflect the likelihood of clinical success. These biomarkers include the pharmacodynamic data measuring change from baseline in mutant androgen receptor protein levels in skeletal muscle and changes in the fat and muscle composition as seen on MRI scans.