The chief medical officer of Alzheon discussed the impact a therapy like ALZ-801 can have on the overall Alzheimer community considering its effects on carriers of APOE e4 alleles. [WATCH TIME: 3 minutes]
WATCH TIME: 3 minutes
"All the outcomes we saw fit together well with our basic mechanism of action, which is the following: you’re able to inhibit the formulation of the soluble amyloid oligomers, which are the most toxic species. By that, we’re able to reduce p-tau early, significantly, and robustly. Because it’s not enough to just have any p-tau reduction, even if its significant at 10% it may not be meaningful. We have multiples of that, 41% is a large effect that starts early and is consistent over a year."
Of the several therapies being presented at the 2022 Clinical Trials on Alzheimer Disease (CTAD) conference, held November 29 to December 2, in San Francisco, California, Alzheon’s ALZ-801 may represent a unique approach, as it has shown early success of patients at high-risk for Alzheimer disease (AD). ALZ-801 or valiltramiprosate, is a prodrug of homotaurine, a modified amino acid, and has been evaluated across a few studies, including a notable phase 2 open-label biomarker study (NCT04693520) and an ongoing phase 3 study, dubbed APOLLOE4 that began in May 2021.
Led by Susan Abushakra, MD, the phase 2 study included 84 patients with early AD with either apolipoprotein (APOE) e4/4 or APOE e3/4 genotypes and a prior positive amyloid-PET or cerebrospinal fluid biomarkers fulfilling amyloid positive and tau positive criteria. The findings presented at CTAD 2022 showed significant reduction of plasma phosphorylated-tau (p-tau)181 at 13 and 26 weeks, that reached to –41% after 52 weeks of treatment. Additionally, bilateral hippocampal volume atrophy at 1 year was reduced by 25% compared with matched subjects from the Alzheimer’s Disease Neuroimaging Initiative.
APOE e4 carriers, considered the most at risk for AD, are considered a high area of research focus; however, few therapies have specifically been tested in these subsets alone. In an interview with NeurologyLive®, Abushakra, the chief medical officer of Alzheon, provided perspective on the significance of ALZ-801’s impact on genetically at-risk individuals, and the need to treat this population as early as possible.