Alzheon to Present New Evidence of Neurovascular Protection and Preserved Brain Microstructure from Oral Valiltramiprosate Trials in APOE4/4 Alzheimer’s Patients at AAIC 2026 Conference in London
Alzheon, Inc., a clinical-stage biopharmaceutical company developing investigational therapies and diagnostic assays for Alzheimer’s disease (AD) and other neurodegenerative disorders, announced that it will present nine posters on its lead investigational therapy, valiltramiprosate/ALZ-801, at the 2026 Alzheimer’s Association International Conference (AAIC) in London, United Kingdom.
The presentations will feature expanded analyses from the Phase 3 APOLLOE4 and Phase 2 biomarker trials and their long-term extensions, including new evidence of neurovascular protection; diffusion tensor imaging (DTI) and volumetric MRI (vMRI) correlations with clinical outcomes; sustained long-term efficacy and safety; and a quantitative systems pharmacology (QSP) analysis evaluating valiltramiprosate as a potential oral maintenance therapy for AD following plaque clearance with anti-amyloid antibody treatments.
Our AAIC presentations reflect more than a decade of disciplined execution in addressing one of the most urgent global unmet needs: Alzheimer’s disease in APOE4/4 patients, who carry the greatest genetic risk and are most vulnerable to complications from anti-amyloid antibodies,” said Martin Tolar, MD, PhD, Founder, President and CEO of Alzheon.
Across nine presentations covering Phase 3 efficacy and safety, imaging and plasma biomarkers, long-term safety, and a new analysis of valiltramiprosate as a potential oral maintenance therapy after anti-amyloid antibody treatment, we are positioning valiltramiprosate as a foundational oral therapy and strengthening Alzheon’s precision medicine platform. If approved, we are committed to making valiltramiprosate available to the patients who need it most, and to potentially extending its benefits to broader APOE4 populations in the years ahead.
Valiltramiprosate/ALZ-801 is an investigational oral agent in Phase 3 development as a potential first-in-class, disease-modifying treatment for Alzheimer’s disease. It acts upstream of anti-amyloid antibodies by inhibiting the formation of neurotoxic soluble amyloid oligomers. The Phase 3 program targets patients with MCI who are homozygous for the apolipoprotein ε4 allele (APOE4/4), a group that represents approximately 15% of all Alzheimer’s patients.
Because APOE4/4 AD patients have a high prevalence of cerebral amyloid angiopathy (CAA), they are particularly vulnerable to brain edema and microhemorrhage complications associated with anti-amyloid antibody therapies, known as amyloid-related imaging abnormalities (ARIA). The AAIC presentations will report neurovascular and ARIA outcomes from the placebo-controlled APOLLOE4 trial. These findings in APOE4/4 patients may also have implications for treating CAA patients and adults with Down syndrome, who similarly have a high prevalence of underlying CAA.
The presentations will also include diffusion tensor imaging and volumetric MRI analyses of hippocampal microstructure; plasma p-tau217 findings; biomarker correlations with imaging and clinical endpoints; sustained efficacy and safety data from long-term extension studies of up to four years; and a quantitative systems pharmacology (QSP) evaluation of valiltramiprosate as a potential oral maintenance therapy following donanemab or lecanemab treatment.
Although the Phase 3 trial did not meet its primary endpoint, we believe the new Phase 3 and long-term extension analyses being presented at AAIC reinforce valiltramiprosate’s differentiated profile as one of the few oral anti-amyloid agents with the potential to slow disease progression while preserving vascular integrity in APOE4/4 Alzheimer’s patients,” said Susan Abushakra, MD, Chief Medical Officer of Alzheon. “The expanded Phase 3 analyses showed consistent neurovascular protection, including lower ARIA rates in the overall population versus placebo, together with clinically meaningful benefits on cognition and brain volume in the pre-specified MCI subgroup from the Phase 3 and Phase 2 long-term extension studies.
New diffusion tensor imaging analyses further demonstrated preservation of hippocampal microstructure and correlations with clinical and brain volume outcomes. We believe these structural effects, together with a significant reduction in plasma p-tau217 in the overall population, support the disease-modifying potential of valiltramiprosate. Importantly, long-term extension findings in APOE4/4 and APOE3/4 carriers continue to show a favorable safety profile, with no symptomatic ARIA-E or ARIA-H observed through up to four years of treatment.
About Alzheon
Alzheon, Inc. is a clinical-stage biopharmaceutical company dedicated to advancing a diverse portfolio of product candidates and diagnostic assays for individuals affected by Alzheimer’s disease and other neurodegenerative disorders. The company is focused on innovating therapeutic solutions that directly target the underlying pathology of neurodegeneration. Its lead Alzheimer’s clinical candidate, valiltramiprosate/ALZ-801, is a first-in-class oral agent currently in Phase 3 clinical development as a potentially disease-modifying treatment for Alzheimer’s disease.
Valiltramiprosate is an orally administered small molecule shown in preclinical studies to completely inhibit the formation of neurotoxic soluble amyloid oligomers. Its well-differentiated follow-on candidate, ALZ-507, is a once daily oral therapy designed to inhibit the formation of amyloid oligomers while also correcting the high risk APOE4 gene. Leveraging clinical expertise and a robust technology platform, Alzheon pursues drug discovery and development using a precision medicine approach that incorporates individual genetic and biomarker profiles, aiming to advance therapies with meaningful benefits for patients.
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