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)

Topline results of the Phase 1b/2a clinical trial anticipated mid-year 2024

MIAMI, May 16, 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 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.

“We are very excited to announce the last patient visit in the Phase 1b/2a clinical trial of AJ201 in SBMA, the final milestone before the anticipated topline data are reported in the middle of this year,” said Alexandra MacLean, M.D., Chief Executive Officer of Avenue. “I want to sincerely thank the trial investigators, the patients, our partner AnnJi Pharmaceutical Co. Ltd., and our internal team for their dedication to completing this study on schedule and for their continued confidence in AJ201. Backed by the drug’s promising preclinical efficacy profile and excellent clinical safety data in healthy volunteers, we look forward to further assessing the safety and tolerability of AJ201 in patients with SBMA, as well as AJ201’s effect on potential pharmacokinetic and pharmacodynamic biomarkers of SBMA, including degradation of mutant AR proteins in muscles, MRI changes and neuroinflammation. We remain committed to advancing AJ201 for SBMA patients who currently have no effective, approved treatments available, as we work to achieve our mission of delivering impactful therapies to patients suffering from neurologic diseases.”

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).