Drugs / valiltramiprosate
Trials 5
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 12 trials | ||||||
| Phase 1 | NCT04157712 | Sep 2015 → Jul 2016 | Alzheimer disease | Alzheon Inc. | Completed | No outcome recorded |
| Phase 1 | NCT04585347 | Sep → Nov 2015 | Alzheimer disease | Alzheon Inc. | Completed | No outcome recorded |
| Phase 21 trial · 1 met primary | ||||||
| Phase 2 | NCT04693520 | Sep 2020 → Jun 2023 | Alzheimer disease | Alzheon Inc. | Completed | Met primary |
| Phase 32 trials · 1 missed | ||||||
| Phase 3 | NCT06304883 | Apr 2024 → Mar 2026 | Alzheimer disease | Alzheon Inc. | Terminated | No outcome recorded Stop: Business |
| Phase 3 | NCT04770220 | May 2021 → May 2024 | Alzheimer disease | Alzheon Inc. | Completed | Missed primary |
News releases announcing trial results or a regulatory action · 38
| Date | Issuer | Release |
|---|---|---|
| 2026-09-08 | Alzheon Inc. | Results Alzheon Announces Peer-Reviewed Publication Showing Oral Valiltramiprosate/ALZ-801 Achieves Sustained Plasma Biomarker Reductions Linked to Better Cognitive, Functional, and Brain Atrophy Outcomes in APOE4 Carriers with Early Alzheimer’s Disease alzheon.com ↗
Phase 2 Findings Show Sustained Plasma Biomarker Reductions over Four Years, Supporting Valiltramiprosate’s Long-Term Disease-Modifying Potential in APOE4 Carriers |
| 2026-07-06 | Alzheon Inc. | Results 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.com ↗
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. |
| 2026-02-03 | Alzheon Inc. | Results Alzheon Reports Plasma Biomarker Results from Phase 3 and 2 Studies of Valiltramiprosate/ALZ-801, Validating Its First-in-Class Mechanism of Action and Underscoring Benefits in Cognition, Function, and Brain Volume Protection in Alzheimer’s Patients alzheon.com ↗ |
| 2025-12-02 | Alzheon Inc. | Results Alzheon Announces Peer-Reviewed Publication Providing Evidence for Hippocampal Volume as Surrogate Marker for Clinical Benefit and Neurodegeneration in Early Alzheimer’s Disease alzheon.com ↗
Multiple clinical studies, including those evaluating lecanemab, donanemab, and valiltramiprosate/ALZ-801, have shown that interventions slowing cognitive decline are also associated with reduced hippocampal atrophy. |
| 2025-11-19 | Alzheon Inc. | Results Alzheon to Present Multiple Clinical, Neuroimaging and Modeling Results for Oral Valiltramiprosate/ALZ-801 from Phase 2 and 3 Studies at CTAD Conference in San Diego, December 1–4, 2025 alzheon.com ↗
Valiltramiprosate Demonstrates Potential as the First Oral Disease-Modifying Agent to Slow Alzheimer’s Pathology, Indicated by Both Clinical and Volumetric MRI Data |
| 2025-10-14 | Alzheon Inc. | Results Alzheon Announces Peer-Reviewed Scientific Publication of Results from Pivotal APOLLOE4 Phase 3 Trial of Oral Valiltramiprosate/ALZ-801 in APOE4/4 Homozygous Individuals with Early Alzheimer’s Disease alzheon.com ↗
This publication provides an overview of effects of valiltramiprosate in the APOLLOE4 Phase 3 trial in patients with Early AD, comprised of Mild Cognitive Impairment (MCI) and Mild AD dementia, who carry two copies of the apolipoprotein E ε4 allele (APOE4/4 homozygotes). |
| 2025-07-15 | Alzheon Inc. | Results Alzheon to Present Insights into Oral Valiltramiprosate/ALZ-801 Mode of Action and Clinical Efficacy at Dedicated Symposium at Alzheimer’s Association International Conference in Toronto on July 30th, 2025 alzheon.com ↗
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). |
| 2025-04-10 | Alzheon Inc. | Results Topline Results from Pivotal APOLLOE4 Phase 3 Trial of Oral Valiltramiprosate/ALZ-801 in Patients with Early Alzheimer’s Disease Carrying Two Copies of APOE4 Gene alzheon.com ↗
Primary Endpoint of Slowing Cognitive Decline as Assessed by Alzheimer’s Disease Assessment Scale-Cognitive Subscale Was Not Met in APOLLOE4 Study |
| 2025-03-18 | Alzheon Inc. | Results Alzheon to Present Results from Pivotal APOLLOE4 Phase 3 Trial of Oral Valiltramiprosate/ALZ-801 at Dedicated Symposium at ADPD Conference in Vienna on April 1st, 2025 alzheon.com ↗
Results from the APOLLOE4 Phase 3 trial show promising clinical and biomarker effects for Alzheimer’s patients with the APOE4/4 genotype. |
| 2025-02-25 | Alzheon Inc. | Results Alzheon Announces Two New Scientific Papers Ahead of APOLLOE4 Phase 3 Trial Topline Symposium at ADPD Conference in Vienna on April 1st, Marking 15 Pathbreaking Peer-Reviewed Publications from Alzheon alzheon.com ↗
Publication in Clinical Pharmacokinetics Demonstrates Favorable Pharmacokinetic Properties of Valiltramiprosate, an Investigational Oral Disease-Modifying Therapy for Treatment of Early Alzheimer’s Disease |
| 2024-07-23 | Alzheon Inc. | Results Alzheon to Present Phase 2 Biomarker Trial Results and Baseline Characteristics from APOLLOE4 Phase 3 Trial of Oral ALZ-801/Valiltramiprosate in Patients with Early Alzheimer’s Disease at Alzheimer’s Association International Conference alzheon.com ↗
Robust and Sustained Plasma P-tau181 Reduction Observed in Phase 2 Biomarker Trial, Combined with Preservation of Brain Volume and Correlations with Cognitive Effects, Reinforces Potential of ALZ-801 to Slow Alzheimer’s Disease Progression |
| 2024-06-25 | Alzheon Inc. | Results Two Peer-Reviewed Scientific Publications Describe Fluid Biomarker, Brain Preservation and Clinical Benefits Following 2 Years of Treatment in Phase 2 Trial Evaluating Oral ALZ-801/Valiltramiprosate in APOE4 Carriers with Early Alzheimer’s Disease alzheon.com ↗
Early, Sustained and Statistically Significant Reduction in Plasma P-tau181 Reaching 31% at 24 Months Observed in Carriers of One or Two Copies of APOE4 Gene, who Represent Two Thirds of Alzheimer’s Patients |
| 2024-02-27 | Alzheon Inc. | Results Alzheon to Present Biomarker, Brain Preservation, and Clinical Results from Pivotal Program of Oral ALZ-801/Valiltramiprosate at International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders alzheon.com ↗ |
| 2023-10-31 | Alzheon Inc. | Results Alzheon CEO Dr. Martin Tolar to Discuss Positive Results from Phase 2 Biomarker Study and Upcoming Readout from Pivotal APOLLOE4 Phase 3 Study Evaluating Oral ALZ-801/Valiltramiprosate at Truist Securities BioPharma Symposium alzheon.com ↗
Robust and Statistically Significant P-tau181 Reduction in Plasma, Combined with Preservation of Brain Volume and Positive Correlations with Cognitive Effects, Supports Potential of ALZ-801 to Slow Alzheimer’s Disease Pathology |
| 2023-10-17 | Alzheon Inc. | Results Alzheon to Present Biomarker, Brain Preservation, and Clinical Results from Pivotal Program of Oral ALZ-801/Valiltramiprosate at 16th Annual Clinical Trials in Alzheimer’s Disease Conference alzheon.com ↗
Our efficacy data combined with a favorable safety profile with no events of vasogenic edema underscore the differentiated clinical profile of ALZ-801 tablet. |
| 2023-09-26 | Alzheon Inc. | Results Alzheon To Present Pivotal Program of Oral ALZ-801/Valiltramiprosate at Singularity Conference, Goldman Sachs Alzheimer’s Disease Day, and BIO Investor Forum alzheon.com ↗
Statistically Significant P-tau181 Reduction in Plasma, Combined with Preservation of Brain Volume and Positive Correlations with Cognitive Benefits, Reinforces Potential of ALZ-801 to Halt Alzheimer’s Disease Progression |
| 2023-09-13 | Alzheon Inc. | Results Alzheon Reports Industry-Leading Biomarker, Brain Preservation and Clinical Benefits Following 24 Months of Treatment in Phase 2 Trial of Oral ALZ-801 (Valiltramiprosate) in Patients with Early Alzheimer’s Disease alzheon.com ↗
We are pleased with the growing body of evidence that supports ALZ-801’s potential as the first oral disease modifying therapy for Alzheimer’s disease. |
| 2023-07-11 | Alzheon Inc. | Results Alzheon to Present Baseline Imaging Characteristics from Ongoing APOLLOE4 Phase 3 Trial of Oral Tablet ALZ-801 (Valiltramiprosate) and Positive Biomarker/Clinical Correlations from Phase 2 Biomarker Study at Alzheimer’s Association International Conference in Amsterdam alzheon.com ↗
Reduction of Hippocampal Atrophy and Lateral Ventricle Enlargement Correlates with Clinical Benefit of ALZ-801 in APOE4 Carriers with Early AD |
| 2023-03-22 | Alzheon Inc. | Results Alzheon to Present Biomarker, Brain Preservation and Clinical Effects of Oral Tablet ALZ-801 (Valiltramiprosate) at Upcoming AD/PD 2023 Conference in Gothenburg, Sweden alzheon.com ↗
Robust Disease-Modifying Effects Observed Through 52 Weeks of Treatment with ALZ-801 Tablet in APOE4 Carriers with Early Alzheimer’s Disease |
| 2022-11-22 | Alzheon Inc. | Results Alzheon to Present Industry-Leading Biomarker, Brain Preservation, and Clinical Effects with Oral ALZ-801 (Valiltramiprosate) at 15th Annual Clinical Trials in Alzheimer’s Disease Conference alzheon.com ↗
Significant Improvement from Baseline on Memory Tests at 3 and 6 Months, and Disease Stabilization Above Baseline at 1-Year Timepoint, to be Further Confirmed in Ongoing APOLLOE4 Phase 3 Trial |
| 2022-09-20 | Alzheon Inc. | Results Alzheon Reports Industry-Leading Biomarker, Brain Preservation and Clinical Effects Following 12 Months of Treatment in Phase 2 Trial of Oral ALZ-801 (Valiltramiprosate) in Patients with Early Alzheimer’s Disease alzheon.com ↗ |
| 2022-07-26 | Alzheon Inc. | Results Alzheon to Present Industry-Leading Phase 2 Biomarker and Clinical Data with ALZ-801 (Valiltramiprosate) Oral Tablet at 2022 Alzheimer’s Association International Conference alzheon.com ↗
New Results Position ALZ-801 to Potentially Become the First Oral Agent that Can Slow or Even Stop and Prevent Alzheimer’s Pathology in Patients and Healthy Individuals at Risk for the Disease |
| 2022-06-07 | Alzheon Inc. | Results Alzheon to Present ALZ-801 (Valiltramiprosate) Oral Tablet Program Update as Potential Treatment for Alzheimer’s Disease in Adults with Down Syndrome at 4th International Trisomy 21 Research Conference alzheon.com ↗
Significant Effects of ALZ-801 on Beta Amyloid and Tau Biomarkers in Patients with Alzheimer’s Support its Clinical Development in Genetically Determined Disorders of Brain Amyloidosis |
| 2022-03-08 | Alzheon Inc. | Results Alzheon to Present ALZ-801 (Valiltramiprosate) Phase 3 Program Update and Industry-Leading Disease Modifying Effects from Phase 2 Trial in Patients with Early Alzheimer’s Disease at AD/PD and NDDS Scientific Conferences alzheon.com ↗
New Results Position ALZ-801 Tablet to Potentially Become the First Oral Agent that Can Slow or Even Stop and Prevent Alzheimer’s Pathology in Patients and Healthy Individuals at Risk for the Disease |
| 2022-02-08 | Alzheon Inc. | Results Alzheon Reports Industry-Leading Disease Modifying Effects from Phase 2 Biomarker Trial of Oral Tablet ALZ-801 (Valiltramiprosate) in Patients with Early Alzheimer’s Disease alzheon.com ↗
Alzheon, Inc., a clinical-stage biopharmaceutical company developing a broad portfolio of product candidates and diagnostic assays for patients suffering from Alzheimer’s disease (AD) and other neurodegenerative disorders, today reported a clinically relevant and statistically significant plasma biomarker reduction and memory improvement in Alzheimer’s patients following 6 months of treatment with oral tablet ALZ-801 (valiltramiprosate) in its Phase 2 biomarker trial. |
| 2021-11-01 | Alzheon Inc. | Results Alzheon to Present New Data from Ongoing Phase 2 Biomarker Trial of ALZ-801 Oral Tablet at Clinical Trials in Alzheimer’s Disease (CTAD) Conference alzheon.com ↗
Baseline Data in Early Alzheimer’s Disease Shows Significant Differences in CSF Biomarker Profiles Between APOE4/4 Homozygotes and APOE3/4 Heterozygotes Validating Patient Selection for APOLLOE4 Phase 3 Study with ALZ-801 Tablet |
| 2020-08-17 | Alzheon Inc. | Results Peer-Reviewed Scientific Publication Profiles First Wave of Disease-Modifying Alzheimer’s Treatments with Potential for Near Term Approval: Aducanumab, Gantenerumab, BAN2401 and ALZ-801 alzheon.com ↗
The research team at Alzheon has built upon this work to demonstrate that our lead oral drug candidate, ALZ-801, works by inhibiting the oligomerization of beta amyloid protein that drives the downstream pathology and cognitive decline in Alzheimer’s disease. |
| 2019-07-14 | Alzheon Inc. | Results Alzheon Presents Data on the Discovery of 3-SPA, an Endogenous Substance in the Human Brain, at the Alzheimer’s Association International Conference alzheon.com ↗
The substance was identified as 3-sulfopropanoic acid (3-SPA), the primary metabolite of tramiprosate and of its prodrug ALZ-801 in humans. |
| 2018-08-06 | Alzheon Inc. | Results Alzheon Scientists Discover Endogenous Substance in Human Brain that Inhibits Formation of Toxic Beta Amyloid Oligomers, Key Driver of Alzheimer’s Disease alzheon.com ↗
The new analyses found that 3-SPA inhibits the formation of toxic soluble Aβ oligomers, comparable to the recently described effects of tramiprosate. |
| 2018-07-23 | Alzheon Inc. | Results Alzheon Presents New Findings in APOE4/4 Homozygous Patients with Mild Alzheimer’s Disease, Further Supporting Precision Medicine Approach and Planned Confirmatory Clinical Trial with ALZ-801 alzheon.com ↗
Large clinical benefit on both cognition and function have been observed in APOE4/4 AD patients with Mild disease in the tramiprosate North American study. |
| 2017-10-24 | Alzheon Inc. | Regulatory FDA Grants Fast Track Designation to Alzheon’s ALZ-801 Development Program for the Treatment of Alzheimer’s Disease alzheon.com ↗
Alzheon, Inc., a clinical stage biopharmaceutical company focused on developing new medicines for patients suffering from Alzheimer’s disease (AD) and other neurological and psychiatric disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead clinical investigational drug, ALZ-801, for the treatment of Alzheimer’s disease. |
| 2017-10-24 | Alzheon Inc. | Results Alzheon Publishes Clinical Results that Demonstrate Safety Profile and Efficacy Potential of ALZ-801 as an Orally-Available Precision Medicine to Treat Alzheimer’s Disease alzheon.com ↗
Alzheon, Inc., today announced the publication of clinical results demonstrating that ALZ-801 achieved levels of therapeutic efficacy with favorable safety and tolerability profile for use as an orally-administered medicine for the treatment of Alzheimer’s disease (AD). |
| 2017-06-22 | Alzheon Inc. | Results Alzheon Publishes New Analyses Showing Clinical Benefit of Tramiprosate in APOE4/4 Homozygous Patients at the Mild Stage of Alzheimer’s Disease; Clinical Effects Suggest Disease Modification Potential and Align with Preclinical Effects on Beta Amyloid Oligomer Formation alzheon.com ↗
The new analyses group patients according to APOE4 genotype and disease severity, and show tramiprosate’s largest efficacy signals in APOE4/4 homozygous patients with Mild Alzheimer’s disease. |
| 2017-04-25 | Alzheon Inc. | Results Alzheon Scientists Discover Novel Therapeutic Mechanism of Inhibition of Formation of Toxic Beta Amyloid Oligomers, Key Driver of Alzheimer’s Disease Pathogenesis alzheon.com ↗
Alzheon, Inc. today announced publication of a newly elucidated molecular mechanism of action for tramiprosate, the active agent in the company’s lead clinical drug candidate, ALZ-801. |
| 2016-07-25 | Alzheon Inc. | Results Alzheon Presents Positive Results of ALZ-801 Clinical Studies that Lead to Advancement to Pivotal Phase 3 Study in Alzheimer’s Patients with APOE4/4 Homozygous Genotype alzheon.com ↗
The Phase 1b study results demonstrated favorable gastrointestinal tolerability, consistent and sustained plasma levels, and allowed selection of an ALZ-801 clinical dose that is bioequivalent to the tramiprosate dose that showed promising efficacy in prior Phase 3 trials. |
| 2015-03-20 | Alzheon Inc. | Results Alzheon Announces Phase 1 Results of ALZ-801, a First-in-Class Inhibitor of Amyloid Formation and Neurotoxicity for Alzheimer’s Disease alzheon.com ↗
The Phase 1 results and Phase 3 post-hoc clinical analyses were presented at a poster session today at the 12th International Conference on Alzheimer’s and Parkinson’s Diseases in Nice, France. |
| 2015-01-05 | Alzheon Inc. | Regulatory Alzheon Announces Patent Allowance in Europe for ALZ-801 in Development for Alzheimer’s Disease alzheon.com ↗ |
| 2014-04-15 | Alzheon Inc. | Regulatory Alzheon Announces Notice of Allowance for U.S. Patent for its Lead Drug Candidate ALZ-801 in Clinical Development for Alzheimer’s Disease alzheon.com ↗ |
All press releases naming this drug 54 releases
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 8 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | valiltramiprosate | “INTRODUCTION: ALZ-801/valiltramiprosate is an oral, small-molecule inhibitor of β-amyloid (Aβ) oligomer formation in late-stage development as a potential disease-modifying...” PMID 39907966 ↗ Feb 2025 |
| Known as | ALZ-801 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04157712 ↗ |
| Known as | BLU-8499 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650301 ↗ |
| Known as | NRM-8499 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650301 ↗ |
| Action | Inhibit | “small-molecule inhibitor of β-amyloid (Aβ) oligomer formation” PMID 39907966 ↗ Feb 2025 |
| Modality | Small molecule | “ALZ-801 is an orally available, valine-conjugated prodrug of tramiprosate. Tramiprosate, the active agent, is a small-molecule β-amyloid (Aβ) anti-oligomer and aggregation inhibitor” PMID 29063518 ↗ Mar 2018 |
| Route | Oral | “ALZ-801 is an orally available, valine-conjugated prodrug of tramiprosate.” PMID 29063518 ↗ Mar 2018 |
| Target | APP | “Amyloid-beta A4 protein stabiliser” CHEMBL4650301 ↗ |