Research and videos
Valiltramiprosate (ALZ-801): the research and Kevin's videos
Is valiltramiprosate (ALZ-801) the drug APOE4/4 carriers are waiting for, and can we get access?
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This page has no results from Phoenix members yet. It shows what published research says, and what Kevin and the experts say in his videos.
See what Phoenix members have measuredIs valiltramiprosate (ALZ-801) the drug APOE4/4 carriers are waiting for, and can we get access?
It may help some APOE4/4 carriers in the earliest symptomatic stage: its phase 3 trial missed its main goal overall, the subgroup with mild cognitive impairment did better only nominally, and no brain swelling was seen. Those are secondary findings, mostly reported by the company, so another trial or a regulator's review is needed before it can be called effective. It is not a prevention drug for people without symptoms.
What the research says
Grade CAPOE4-specific evidence: yes
In APOE4/4 carriers with early Alzheimer's, valiltramiprosate missed its main goal but may help those at the MCI stage, with no ARIA; this needs confirming.
Key studies
Abushakra 2025, DrugsGrade C
Company-authored phase 3 report (325 APOE4/4): no significant clinical effect overall; hippocampal shrinkage 18% slower; MCI-subgroup gains nominal only; no increase in brain swelling or bleeding.
Read on PubMed (opens in a new tab)Hey 2026, DrugsGrade C
Company-authored biomarker analysis of the phase 3 trial: blood p-tau217 fell with the drug, most clearly in the MCI subgroup.
Read on PubMed (opens in a new tab)Abushakra 2024, Alzheimers Dement (N Y)
Design of APOLLOE4: 325 APOE4/4 homozygotes with early Alzheimer's, 78 weeks, placebo-controlled.
Read on PubMed (opens in a new tab)
1 more key study
Hey 2024, Drugs
Two-year single-arm phase 2 in APOE4 carriers: blood p-tau181 fell and hippocampal loss was slower than expected; no control group.
Read on PubMed (opens in a new tab)
What Kevin and experts say
5 passages for, 1 with a caution
Now showing: Dr. Kevin Tran · 0:15
Open on YouTube (opens in a new tab)For5 passages
No ARIA was seen with this oral drug in homozygotes.
“Zero brain swelling, zero bleeding, zero deaths.”
Dr. Kevin Tran, Founder of The Phoenix Community, Doctor of Pharmacy, APOE4/4 carrier
Homozygotes with MCI held cognition in this subgroup.
“52% benefit on [ADAS-Cog] and 102% benefit on [CDR-SB. ADAS-Cog] is maintaining above baseline for 52 weeks”
Dr. John Hey, Alzheon, developer of ALZ-801 (valiltramiprosate) (conference clip)
Newer research: the published phase 3 found no significant benefit on cognition or daily function overall; MCI-subgroup gains were only nominal.
Read the study (opens in a new tab)Daily function held steady for 78 weeks.
“The CDR sum of boxes remained at baseline for the entire 78 weeks.”
Dr. John Hey, Alzheon, developer of ALZ-801 (valiltramiprosate) (conference clip)
Newer research: the published phase 3 found no significant benefit on cognition or daily function overall; MCI-subgroup gains were only nominal.
Read the study (opens in a new tab)It slowed brain shrinkage across all regions.
“highly significant in all regions indicating a very substantial effect on [neurodegeneration]”
Dr. John Hey, Alzheon, developer of ALZ-801 (valiltramiprosate) (conference clip)
Newer research: the published phase 3 found 18% slower hippocampal shrinkage, but no significant clinical benefit in the full trial group.
Read the study (opens in a new tab)Homozygotes may gain the most from this drug.
“The impact of the AL 801 actually is the greatest in the [APOE4/4 homozygous] compared to the other groups.”
Dr. Hugo Geerts, Quantitative systems pharmacology modeler (conference clip)
With a caution1 passage
Promising, but the trial missed its main goal.
“this clinical trial actually did not meet its primary endpoint and these are only secondary findings.”
Dr. Kevin Tran, Founder of The Phoenix Community, Doctor of Pharmacy, APOE4/4 carrier
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