Phase 1 trial of investigational therapy ALX-001 doses first patient

A Phase 1 clinical trial for ALX-001, a potential treatment for neurodegenerative diseases, has dosed its first Parkinson’s disease patient. This trial, taking place at Duke Clinical Research Institute and funded by The Michael J. Fox Foundation, is evaluating the safety of ALX-001 compared to a placebo. Participants, aged 21–80, will receive 50 mg or 100 mg doses twice daily for 28 days. The study will also monitor changes in dopamine transporter protein levels, which serve as an early marker of response. Parkinson's disease is marked by the loss of dopamine-producing neurons, which leads to motor dysfunction. Researchers will also assess the therapy’s pharmacokinetics—how it moves through the body. Allyx Therapeutics is also testing ALX-001 in Alzheimer’s disease with a parallel study. ALX-001, previously known as BMS-984923, is an oral small molecule that penetrates the brain, blocks the mGluR5 receptor, and helps restore synapses without affecting other glutamate pathways important for cognition. It is being developed as a disease-modifying treatment for Parkinson’s and Alzheimer’s. Earlier trials with healthy volunteers showed the therapy was safe and well-tolerated, with doses of 50 mg and 100 mg selected for ongoing studies. Supported by more than $20 million in grants, Allyx Therapeutics plans to advance to Phase 2 trials for Parkinson’s patients.