Future Plans for ALZ-801 in Homo- and Heterozygous Populations: Susan Abushakra, MD


The chief medical officer at Alzheon provided perspective on the next steps for ALZ-801 following a successful phase 2 study of patients with APOE ε4/4 or APOE ε3/4 genotype. [WATCH TIME: 2 minutes]

WATCH TIME: 2 minutes

"In the future, it would be nice to enroll even non-carriers, but as long as we select them [patients] with having the right levels of amyloid and tau pathology, they should respond to ALZ-801 as well."

An oral antiamyloid agent, ALZ-801 (Alzheon) is an optimized prodrug of tramiprosate that has shown to inhibit amyloid-ß(Aß)42 aggregation into toxic oligomers. The therapy was assessed in a phase 2, open-label study (NCT04693520) of 84 patients with early Alzheimer disease (AD) who carried either the apolipoprotein (APOE) ε4/4 or ε3/4 genotype. Among the 75 participants who completed 52 weeks of treatment, ALZ-801-treated patients demonstrated a significant 41% reduction from baseline in plasma phosphorylated tau 181 (p-tau181)(P = .016). Additionally, the agent also significantly reduced plasma p-tau181/Aß42 ratio by 37% at the same time point (P = .032).

These findings were originally announced in September 2022 and further explained at the 2022 Clinical Trials on Alzheimer’s Disease (CTAD) conference, November 29 to December 2, in San Francisco, California. A potential disease-modifying therapy, ALZ-801 is currently being assessed in a phase 3 study (NCT04770220) called APOLLOE4 with the help of a $47 million grant from the National Institute on Aging. This 78-week trial evaluates the efficacy, safety, biomarker, and imaging effects of 265 mg twice daily oral dose of ALZ-801 in patients with early AD with APOE ε4/4 genotype, which constitutes approximately 15% of the AD population.

At CTAD 2022, NeurologyLive® caught up with Susan Abushakra, MD, chief medical officer of Alzheon and primary investigator of ALZ-801’s clinical program. Abushakra provided insight on the next steps for ALZ-801, including the ongoing phase 3 trial as well as other potential studies in carrier populations that include both homozygous and heterozygous patients. Additionally, she spoke on the pertinent end points and assessments needed to understand ALZ-801’s full potential.

1. Abushakra S, Hey J, Bennow K, et al. Significant effects of oral ALZ-801 on plasma biomarkers of Alzheimer’s disease: 12-month interim analysis of phase 2 biomarker study in APOE4 carriers with early AD. Presented at: 2022 CTAD Conference; November 29-December 2; San Francisco, CA. Abstract OC27
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