ALZ-801, developed by Alzheon, Inc., has shown encouraging results in clinical trials as an experimental oral medication for Alzheimer’s patients with the APOE4 gene. The phase 3 trial, conducted by researchers at Alzheon, involved 325 participants with early Alzheimer’s or mild cognitive impairment, all of whom carried the APOE4 gene. The study, which lasted 18 months, aimed to evaluate the drug’s effectiveness in slowing memory loss and brain atrophy.
The trial revealed that ALZ-801 reduced brain shrinkage—particularly in the hippocampus, a region critical for memory and cognitive function—by about 18% compared to the placebo group. The most significant improvements were observed in patients with the earliest stages of Alzheimer’s who had mild cognitive impairment, where the drug showed a 50% reduction in memory decline and nearly halted daily cognitive decline. However, these benefits were not statistically significant across the entire study population.
The researchers reported that the drug’s side effects included nausea, vomiting, and appetite suppression. Notably, ALZ-801 works by preventing the formation of amyloid plaques in the brain, which differentiates it from currently approved IV treatments like lecanemab and donanemab. These latter treatments are monoclonal antibodies that target existing plaques and have been associated with side effects such as brain swelling and bleeding—effects that were not observed with ALZ-801.
Dr. Christopher Weber, a senior director at the Alzheimer’s Association, commented on the significance of these findings.