New Research on ALZ‑801 (valiltramiprosate) in APOE‑4 Homozygotes
What it means for APOE4 homozygotes
A major study called APOLLOE4 focused on people with early Alzheimer’s or mild cognitive impairment who have two APOE‑ε4 genes (APOE‑4 homozygotes). These individuals are known to decline faster and are at higher risk overall.
Here’s what researchers found:
Brain scans showed real benefits — in the group with earlier disease (MCI), patients taking ALZ‑801 had less shrinkage in the hippocampus and cortex, and better preservation of whole brain volume, compared to those on placebo
Cognitive and daily function measures improved — they saw benefits on memory and thinking tests (ADAS‑Cog), daily living (DAD), and a trend toward improvement on CDR‑SB, even though the full trial didn’t hit its main endpoint
The effects were consistent with how ALZ‑801 is supposed to work: it prevents harmful amyloid clumps (oligomers) from forming in the brain Importantly, ALZ‑801 is taken orally (pill form) and was well tolerated — there were no serious side effects like the brain swelling (ARIA) sometimes seen with antibody treatments
Why it matters
Hippocampal shrinkage is tightly linked to memory loss—and APOE‑4 homozygotes often lose this brain region fastest. So, a drug that slows or protects against that loss could be a real game-changer.
🔬 Bottom line: For people at highest genetic risk (APOE‑4 homozygotes) in the early stages of Alzheimer’s, ALZ‑801 showed promising brain imaging and early cognitive benefits—with a safe and convenient oral formulation. That sets it apart as one of the most interesting new therapies now being tested.