Comunicato Stampa: Alzheon to Present Clinical, Imaging, and Fluid Biomarker Results from Trials with Oral Valiltramiprosate/ALZ-801 at 2026 AD/PD Conference in Copenhagen, Denmark

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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 19^th 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 19^th from 1:50pm – Saturday, March 21^st 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.

About ALZ-801

Valiltramiprosate/ALZ-801 is an investigational oral agent currently in Phase 3 development as a potential first-in-class, disease-modifying treatment for Alzheimer’s disease.^3-7,9,12 Valiltramiprosate is designed to inhibit the formation of neurotoxic soluble beta amyloid oligomers that contribute to cognitive decline in individuals with AD.^4-8,10,15 Preclinical mechanism-of-action studies have demonstrated that ALZ-801 can completely block the formation of these neurotoxic oligomers at the dosage used in Phase 3 clinical trials.^3,9,12,14 Valiltramiprosate employs an enveloping molecular mechanism of action intended to prevent the aggregation of soluble amyloid oligomers in the human brain,¹⁴ which are associated with the onset and progression of cognitive impairment in AD patients.^3,4,7,9,10 In recognition of its therapeutic promise, valiltramiprosate received Fast Track designation from the U.S. Food and Drug Administration in 2017 for the treatment of Alzheimer’s disease.

Clinical trial data indicate that valiltramiprosate exhibits strong clinical efficacy at the MCI stage, and a favorable safety profile, with no observed increase in the risk of brain vasogenic edema.^5-1-10,13,15 The initial Phase 3 program for valiltramiprosate targets Early AD patients who are homozygous for the apolipoprotein ε4 allele (APOE4/4), with plans to expand future research to include AD treatment and prevention in individuals carrying one copy of the APOE4 gene.^3–10

Valiltramiprosate APOLLOE4 Phase 3 Trial

An Efficacy and Safety Study of Valiltramiprosate in APOE4/4 Early Alzheimer's Disease Subjects (NCT04770220): This trial was designed to evaluate the efficacy, safety, biomarker and imaging effects of 265 mg twice daily oral dose of valiltramiprosate in Early AD subjects with two copies of the apolipoprotein ε4 allele (APOE4/4 homozygotes), who constitute approximately 15% of Alzheimer's patients. This double-blind, randomized trial compared oral valiltramiprosate to placebo treatment over 78 weeks. The APOLLOE4 trial was supported by a grant from the National Institute on Aging to Alzheon, with Susan Abushakra as the principal investigator.

Valiltramiprosate APOLLOE4 Long Term Extension Trial (Phase 3 LTE)

An ongoing long-term extension of the trial, APOLLOE4-LTE, evaluates valiltramiprosate in subjects who complete the core APOLLOE4 study for an additional 104 weeks of treatment for a total of 182 weeks or 3.5 years over the core and LTE study. This LTE study ended in January 2026 (NCT06304883).

Valiltramiprosate Phase 2 Biomarker Trial

Biomarker Effects of Valiltramiprosate in APOE4 Carriers with Early Alzheimer's Disease (NCT04693520): This trial was designed to evaluate the effects of 265 mg twice daily oral dose of valiltramiprosate on biomarkers of AD pathology in subjects with Early AD, who have either the APOE4/4 or APOE3/4 genotype and constitute 65-70% of Alzheimer's patients. The primary outcome was the change from baseline in plasma p-tau₁₈₁. The trial also included evaluation of clinical efficacy, safety, tolerability, and pharmacokinetic profile of valiltramiprosate over 104 weeks of treatment. A completed long-term extension of the trial evaluated the same dose of valiltramiprosate for an additional 104 weeks of treatment for a total of 208 weeks.^3,7,8

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. 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.

Alzheon Scientific Publications

¹Abushakra S, et al: Hippocampal Atrophy on Magnetic Resonance Imaging as a Surrogate Marker for Clinical Benefit and Neurodegeneration in Early Symptomatic Alzheimer’s Disease: Synthesis of Evidence from Observational and Interventional Trials, CNS Drugs 2025; 40, 199-214.

²Abushakra S, et al: Clinical Efficacy, Safety and Imaging Effects of Oral Valiltramiprosate in APOEε4/ε4 Homozygotes with Early Alzheimer’s Disease: Results of the Phase III, Randomized, Double-Blind, Placebo-Controlled, 78-Week APOLLOE4 Trial, Drugs 2025; 85, 1455-1472.

³Pearson D, et al: Polymorph Analysis of ALZ-801 (Valiltramiprosate), a Valine-Conjugated Oral Prodrug of Tramiprosate in Late-Stage Clinical Development for Alzheimer’s Disease, Journal of Chemical Crystallography 2025; 55, 206-215.

⁴Hey JA, et al: Clinical Pharmacokinetics of Oral ALZ-801/Valiltramiprosate in a Two-Year Phase 2 Trial of APOE4 Carriers with Early Alzheimer’s Disease, Clinical Pharmacokinetics 2025; 64, 407-424.

⁵Aye S, et al: Point of View: Challenges in Implementation of New Immunotherapies for Alzheimer's Disease, The Journal of Prevention of Alzheimer’s Disease 2025;12(1):100022.

⁶Abushakra S, et al: APOLLOE4 Phase 3 Study of Oral ALZ-801/Valiltramiprosate in APOE ε4/ε4 Homozygotes with Early Alzheimer’s Disease: Trial Design and Baseline Characteristics, Alzheimer’s & Dementia 2024; 10(3): e12498.

⁷Tolar M, et al: The Single Toxin Origin of Alzheimer’s Disease and Other Neurodegenerative Disorders Enables Targeted Approach to Treatment and Prevention, International Journal of Molecular Sciences 2024; 25(5), 2727.

⁸Hey JA, et al: Analysis of Cerebrospinal Fluid, Plasma β Amyloid Biomarkers, and Cognition from a 2-Year Phase 2 Trial Evaluating Oral ALZ-801/Valiltramiprosate in APOE4 Carriers with Early Alzheimer’s Disease Using Quantitative Systems Pharmacology Model, Drugs 2024; 84(7), 825-839.

⁹Hey JA, et al: Effects of Oral ALZ-801/Valiltramiprosate on Plasma Biomarkers, Brain Hippocampal Volume, and Cognition: Results of 2-Year Single Arm, Open Label, Phase 2 Trial in APOE4 Carriers with Early Alzheimer’s Disease, Drugs 2024; 84(7), 811-823.

¹⁰Tolar M, et al: Neurotoxic Soluble Amyloid Oligomers Drive Alzheimer’s Pathogenesis and Represent a Clinically Validated Target for Slowing Disease Progression, International Journal of Molecular Sciences 2021; 22(12), 6355.

¹¹Abushakra S, et al: APOE ε4/ε4 Homozygotes with Early Alzheimer’s Disease Show Accelerated Hippocampal Atrophy and Cortical Thinning that Correlates with Cognitive Decline, Alzheimer’s & Dementia 2020; 6(1): e12117.