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AD/PD 2024 | The potential of ALZ-801 as an oral small molecule treatment for Alzheimer’s disease

John Hey, PhD, Alzheon, Inc., Framingham, MA introduces ALZ-801 as an orally-available small molecule under investigation for the treatment of Alzheimer’s disease (AD), giving an update on the progress of the Phase II and III studies to date. ALZ-801 is a pro-drug of tramiprosate, designed to modify the progression of AD. It changes the conformation of amyloid beta (Aβ) 40 and 42 monomers, inhibiting the formation of toxic oligomers and thus, preventing aggregation. ALZ-801 acts upstream of other agents in the Aβ pathway, preventing the formation of harmful aggregates and plaques rather than removing them. This unique mechanism makes it an appealing option for AD patients seeking a convenient oral medicine with disease-modifying potential. The development pipeline has taken a precision medicine approach, initially focusing on APOE4 homozygous individuals. Future studies may extend to APOE4 heterozygous and non-carrier populations. This interview took place at the AD/PD™ 2024 congress in Lisbon, Portugal.

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