Alzheon to present insights into oral Valiltramiprosate/ALZ-801 mode of action and clinical efficacy
- Valiltramiprosate evaluated in APOLLOE4 trial, first interventional Phase 3 Alzheimer’s trial focused on high-risk APOE4/4 homozygotes
- Anti-amyloid oligomer action underpins valiltramiprosate’s potential benefit in Alzheimer’s disease at MCI stage of disease
- Valiltramiprosate designed to inhibit amyloid aggregation by distinct upstream mode of action to slow progression of Alzheimer’s disease
- Precision medicine approach supported by valiltramiprosate’s favorable safety in high-risk APOE4 carriers with major unmet medical need
- Valiltramiprosate has potential to become the first oral agent to slow Alzheimer’s pathology in patients
Alzheon, Inc., will present new data on valiltramiprosate at the Alzheimer’s Association International Conference, highlighting the differentiated mode of action that acts upstream in the amyloid pathway, blocking formation of neurotoxic amyloid oligomers that drive disease progression. Valiltramiprosate showed positive clinical and volumetric MRI effects in patients with Mild Cognitive Impairment (MCI) who carry one or two copies of the ε4 allele of the apolipoprotein E gene (APOE3/4 heterozygotes and APOE4/4 homozygotes, respectively).
“Valiltramiprosate represents a promising advancement in targeting the early highly toxic forms of beta amyloid that drive Alzheimer’s disease onset and progression,” said Sam Gandy, M.D., Ph.D., Professor of Neurology and Psychiatry at Icahn School of Medicine at Mount Sinai. “By focusing on oligomer formation upstream, this oral therapy has the potential to change the treatment paradigm for patients at risk for Alzheimer’s and those with early symptomatic disease, particularly the high-risk APOE4 carriers.”
Valiltramiprosate is an investigational oral AD treatment in Phase 3 clinical development with an upstream mechanism of action from anti-amyloid antibodies that prevents formation of neurotoxic amyloid oligomers. A prodrug of tramiprosate with optimised pharmacokinetics and brain penetration, valiltramiprosate was designed to prevent amyloid aggregation at the initial stage of the amyloid cascade. Soluble amyloid oligomers play a central role in the pathogenesis and progression of Alzheimer’s disease.
Results from the APOLLOE4 Phase 3 trial evaluating valiltramiprosate showed promising clinical and volumetric MRI effects in Alzheimer’s patients with the APOE4/4 genotype at the earliest symptomatic stage of disease.


