Alzheon Reports Valiltramiprosate Shows Promise as an Oral Potentially Disease-Modifying Agent to Slow Alzheimer’s Disease Based on Clinical, MRI, and Biomarker Data in APOE4/4 MCI Patients
Two Oral Presentations and Six Posters Highlight Consistent Effects of Oral Valiltramiprosate on Brain Volume, Microstructural Integrity, Neurodegeneration, Systems Modeling, and Long-Term Clinical Stability
Valiltramiprosate Designed to Inhibit Amyloid Aggregation via Distinct Upstream Mode of Action, Targeting Soluble Amyloid Oligomers – A Key Driver of Alzheimer’s Disease Pathology
Precision Medicine Approach Supported by Favorable Safety Results in High-Risk APOE4/4 Patients in Phase 3 & Phase 2 Trials
Alzheon, Inc., a clinical-stage biopharmaceutical company developing a broad portfolio of investigational therapies and diagnostic assays for patients with Alzheimer’s disease (AD) and other neurodegenerative disorders, today announced it will present new efficacy, imaging, safety, biomarker, and quantitative systems pharmacology findings for its lead investigational therapy, valiltramiprosate/ALZ-801 during the 2026 International Conference on Alzheimer’s and Parkinson’s Disease (AD/PD) in Copenhagen, Denmark.
“Valiltramiprosate stands out as the only late-stage oral Alzheimer’s treatment with potential for a major near-term impact,” said Susan Abushakra, Chief Medical Officer of Alzheon. “Our results at AD/PD align with previous clinical, imaging, safety, and biomarker data. Targeting toxic amyloid oligomers continues to show promise for preserving cognition, daily function, and brain volume. As we prepare for regulatory discussions, our priority is approval of valiltramiprosate for Alzheimer’s patients with the APOE4 genotype and expanding to additional high-risk groups.”
Valiltramiprosate is an investigational oral therapeutic agent in Phase 3 development that works upstream of anti-amyloid antibodies by preventing the formation of neurotoxic soluble amyloid oligomers. As a valine-conjugated prodrug of tramiprosate with improved pharmacokinetics and brain penetration, valiltramiprosate targets early amyloid aggregation, a core driver of Alzheimer’s pathology and disease progression. Valiltramiprosate aims to preserve brain structure and cognitive function in individuals with early-stage Alzheimer’s disease, with particular emphasis on APOE4/4 homozygotes, which are the highest-risk genetic subgroup, characterized by aggressive neurodegeneration and limited treatment options.
New results to be presented at AD/PD include safety and ARIA analyses from the APOLLOE4 Phase 3 placebo-controlled trial, quantitative systems pharmacology (QSP) analyses of amyloid aggregation dynamics, diffusion MRI findings showing preserved microstructural integrity, and clinical-imaging correlations in the pre-specified Mild Cognitive Impairment (MCI) subgroup of the Phase 3 study. These results are further supported by responder analyses showing reversal of hippocampal atrophy, and long-term MMSE outcomes from a Phase 2 study which had disease stability over four years of treatment in APOE4 MCI carriers.
Details of Presentations at AD/PD Conference
Alzheon will deliver two oral presentations and showcase six posters covering clinical, MRI, microstructural, biomarker, and QSP analyses.
Symposium: Advances in AD Treatment
Saturday, March 21, 2026 – 2:10pm- 4:10pm
Podium Presentation: Clinical Efficacy and Imaging Results of Oral Valiltramiprosate in APOE4/4 Homozygotes with Early AD: Phase 3 Topline Results from 78-Week APOLLOE4 Trial
- Presenter: Dr. Aidan Power, Chief Development Officer, Alzheon, Inc.
- Lecture Time: 2:10pm – 2:25pm
Podium Presentation: Clinical Stabilization in MCI APOE4 Carriers Over 3 Years Correlates with Improved Hippocampal Volume in Valiltramiprosate Phase 2 LTE Study
- Presenter: Dr. John Hey, Chief Scientific Officer, Alzheon, Inc.
- Lecture Time: 2:25pm – 2:40pm
Tuesday, March 17 from 7:30am – Thursday, March 19th 11:00am
Poster: Biomarker‑Positive APOE4 Carriers with MCI Show Disease Stability Over 4 Years of Valiltramiprosate/ALZ‑801 Treatment: MMSE Responder Analysis from the ALZ‑801 Phase 2 Long‑Term Extension Study
- Presenter: Patrick Kesslak, Senior Research Fellow, Alzheon, Inc.
Thursday, March 19th from 1:50pm – Saturday, March 21st 5:00pm
Poster: Positive Effects of Oral Valiltramiprosate on Grey and White Matter Microstructural Integrity by DTI in APOE4/4 Homozygotes with Early AD: APOLLOE4 78‑Week Phase 3 Results
- Presenter: Earvin Liang, VP of Clinical Operations, Alzheon, Inc.
Poster:Safety and ARIA Analyses of the Oral Anti‑Amyloid Oligomer Agent Valiltramiprosate in APOE4/4 Homozygotes with Early AD: Results of the 78‑Week Phase 3 APOLLOE4 Trial
- Presenter: Dr. David Watson, CEO, Alzheimer’s Research & Treatment Center
Poster: Quantitative Systems Pharmacology Analyses of Beta‑Amyloid Aggregation Dynamics: Additive/Synergistic Effects of Valiltramiprosate Used in Combination with Leqembi and Kisunla
- Presenter: Jean Schaefer, VP of CMC & Project Management, Alzheon, Inc.
Poster: Valiltramiprosate Reverses Neurodegeneration and Improves Clinical Outcomes in Subgroup of Early Symptomatic AD in Homozygotes: Results from the 78‑Week Phase 3 APOLLOE4 Trial
- Presenter: Dr. John Hey, Chief Scientific Officer, Alzheon, Inc.
Poster: Valiltramiprosate Effects in the Pre‑Specified MCI Group Show Significant Correlations Between Clinical and Brain Volume Benefits: Phase 3 APOLLOE4 Results in Early AD APOE4/4 Homozygotes
- Presenter: Dr. Aidan Power, Chief Development Officer, Alzheon, Inc.
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