Alzheon, Inc. announced statistically significant and clinically relevant reduction in plasma biomarkers of neurodegeneration, preservation of brain volume, and positive cognitive effects in Early AD patients who are carriers of apolipoprotein e4 allele (APOE4) following 24 months of treatment with investigational agent ALZ-801 in the Phase 2 biomarker trial. Oral ALZ-801 (valiltramiprosate) is an investigational oral disease-modifying therapy in Phase 3 development for the treatment of Early AD. In mechanism of action studies, ALZ-801 fully blocked the formation of neurotoxic soluble beta amyloid (Ab) oligomers at the Phase 3 clinical dose.

Oral ALZ-801 has shown potential for robust clinical efficacy in the highest-risk and most fragile late-onset Alzheimer's population - patients with two copies of the apolipoprotein e4 mutation (APOE4/4 homozygotes), and favorable safety with no increased risk of vasogenic brain edema. At baseline, study subjects were 52% female, with mean age 69 years, MMSE 26 (range 22-30), and 70% having mild cognitive impairment (MCI). In November 2022, Alzheon reported positive study results from a pre-specified analysis following 52 weeks of treatment with ALZ-801.

An ongoing long-term extension of the trial evaluates ALZ-801 for an additional 52 weeks of treatment for a total of 156 weeks.