Avenue Therapeutics, Inc. (Nasdaq: ATXI), a specialized pharmaceutical company dedicated to advancing therapies for neurologic diseases, has successfully enrolled all participants in the Phase 1b/2a clinical trial of AJ201, its lead drug candidate for the treatment of spinal and bulbar muscular atrophy (SBMA), also known as Kennedy's Disease. The company anticipates releasing topline data from the trial in the second quarter of 2024.

Dr. Alexandra MacLean, CEO of Avenue, expressed satisfaction with the rapid enrollment, highlighting the substantial unmet need for effective treatments in SBMA. The 12-week, multicenter, randomized, double-blind Phase 1b/2a trial included 25 patients, randomly assigned to receive either AJ201 (600 mg/day) or a placebo. The primary objective is to assess the safety and tolerability of AJ201 in individuals with clinically and genetically defined SBMA.

Given the gradual progression of SBMA, making it challenging to demonstrate clinical efficacy over a short period, Avenue has included various biomarkers as secondary endpoints. These biomarkers aim to provide insights into target engagement and the potential for clinical success. They include pharmacodynamic data measuring changes in mutant androgen receptor protein levels in skeletal muscle and alterations in fat and muscle composition observed on MRI scans.

SBMA is a rare, X-linked genetic neuromuscular disease predominantly affecting males. It is characterized by the expansion of trinucleotide CAG repeats in the androgen receptor, resulting in the production of a mutant polyglutamine AR protein leading to muscular atrophy in the limbs and bulbar region.

AJ201, the investigational drug, is designed to address SBMA through multiple mechanisms, including the degradation of abnormal androgen receptor protein and the stimulation of Nrf1 and Nrf2 pathways, which play a role in protecting cells from oxidative stress. The drug has demonstrated an excellent safety and pharmacokinetic profile in a Phase 1 study involving 72 healthy volunteers. Currently being evaluated in a Phase 1/2a multicenter trial across the U.S., AJ201 has received Orphan Drug Designation from the FDA for multiple polyglutamine diseases, including SBMA, Huntington’s disease, and spinocerebellar ataxia. Avenue holds the exclusive license for AJ201 in various regions.

Polyglutamine diseases, characterized by expanded CAG repeats, encompass a group of neurodegenerative disorders. Mutant protein aggregation is a key pathological feature, and AJ201 aims to modulate various cellular pathways to address SBMA and potentially other polyglutamine diseases.

 

Source: globenewswire.com

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