Alzheon has dosed the first patient in its APOLLOE4 Phase III study evaluating the efficacy and safety of oral anti-amyloid drug ALZ-801 in early Alzheimer’s disease (AD) patients.
With a novel anti-amyloid oligomer mechanism of action, ALZ-801 is an optimised prodrug of tramiprosate that has shown promising results in analyses of Phase III clinical data.
The APOLLOE4 randomised, double-blind, placebo-controlled trial is supported by a $47m grant from the National Institute on Aging.
For the trial, 300 APOE4/4 homozygotes with early AD will be enrolled at approximately 85 sites in the US, Canada, and Europe.
The trial’s primary objective is to measure the impact of the drug on cognition using the Alzheimer’s Disease Assessment Scale – cognitive subscale (ADAS-cog).
Assessments of function, ability to perform daily activities, and neuropsychiatric symptoms form the secondary endpoints of the trial.
Additionally, the study will evaluate ALZ-801’s effects on fluid and imaging biomarkers shown to be sensitive early markers of the disease progression and neuroinflammation.
Alzheon founder, president and CEO Martin Tolar said: “We have designed this confirmatory Phase III trial in an unprecedented way by applying a precision medicine approach, focusing initially on high-risk patients with the APOE4/4 genotype shown to respond to anti-amyloid agents, and incorporating state-of-the-art fluid and imaging biomarkers.
“By fully blocking the formation of neurotoxic amyloid oligomers in brain, but without interaction with amyloid plaques, the APOLLOE4 trial may also provide definitive proof of the central role of amyloid oligomers in the initiation and progression of AD.”
In the APOLLOE4 trial, AD patients with the APOE4/4 genotype will be enrolled into either placebo or active treatment arms, each comprising 150 patients.
Patients will be given 265mg of ALZ-801 or matching placebo tablets two times a day for 78 weeks.
For all patients, frequent cognitive tests, functional assessments, and volumetric magnetic resonance imaging will be performed. They will also have plasma biomarker assessments.
In 2017, the US Food and Drug Administration granted Fast Track designation to ALZ-801.