Avenue Therapeutics announces first patient dosed in Phase 1b/2a trial of AJ201

Avenue Therapeutics 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. Enrollment in the trial is expected to be complete by the end of 2023 or early 2024 with potential topline data in 2024. “We are excited to announce that the first patient has been dosed in our Phase 1b/2a clinical trial evaluating AJ201 for the treatment of SBMA, a progressive and devastating neurodegenerative disease that currently has no approved treatments available,” said Alexandra MacLean, M.D., Chief Executive Officer of Avenue. “We are encouraged by the Phase 1 clinical data of AJ201 that demonstrate the drug’s excellent safety profile in healthy volunteers. Additionally, compelling preclinical data in a mouse model showed efficacy signals, including improvement in motor function, robust degradation of mutant androgen receptors, a disease-signaling protein, and activation of the Nrf1 and Nrf2 pathways. In this Phase 1b/2a clinical trial, we 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. We look forward to advancing this much needed drug, as we continue to deliver on our mission of bringing impactful therapies to people suffering from neurologic diseases.”