An Efficacy and Safety Study of ALZ-801 in APOE4/4 Early AD Subjects

Summary

This study is being conducted to evaluate the safety and efficacy of ALZ-801 in Early Alzheimer's disease (AD) subjects with the APOE4/4 genotype. This is a double-blind, randomized trial with one dose of ALZ-801 compared to placebo.

Timeline

Start

2021-05-19

Primary completion

2024-05-29

Completion

2024-07-29

Outcome

Missed primary endpoint

registry analysis (other test); Placebo vs ALZ-801; p = 0.6058; Least Squares Mean Difference -0.51; MMRM; NCT04770220 ↗

paper overall efficacy population did not show significant effects on ADAS-Cog13 or other clinical outcomes compared with placebo (ADAS-Cog13: 11% slowing; p = 0.607, N = 320) PMID 41015981 ↗

release “Primary Endpoint of Slowing Cognitive Decline as Assessed by Alzheimer’s Disease Assessment Scale-Cognitive Subscale Was Not Met in APOLLOE4 Study” alzheon.com ↗

release “Although the Phase 3 trial did not meet its primary endpoint, we believe the new Phase 3 and long-term extension analyses” alzheon.com ↗

Publications

• Abushakra S, Power A, Watson D, Porsteinsson A, Sabbagh M, MacSweeney E, Cohen S, Boada Rovira M, Doraiswamy PM, Liang E, Flint S, Kesslak JP, McLaine R, Albayrak A, Schaefer J, Yu J, Tolar L, Dickson S, Hey JA, Tolar M. Clinical Efficacy, Safety and Imaging Effects of Oral Valiltramiprosate in APOEepsilon4/epsilon4 Homozygotes with Early Alzheimer's Disease: Results of the Phase III, Randomized, Double-Blind, Placebo-Controlled, 78-Week APOLLOE4 Trial. Drugs. 2025 Nov;85(11):1455-1472. doi: 10.1007/s40265-025-02250-5. Epub 2025 Sep 28.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	valiltramiprosate	Small molecule	265 mg	Oral

Indications