Alzheon’s Precision Medicine Approach to Resurrect an Alzheimer’s Drug Fails Pivotal Study

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Summary

An experimental Alzheimers disease therapy designed by Alzheon to reduce depositions of amyloid plaque in the brain failed to beat a placebo in a Phase 3 study, the latest blow to a drug whose history is marked by clinical trial shortfalls.The twice-daily pill, valiltramiprosate, did not meet the main clinical trial goal of slowing cognitive decline measured at 78 weeks, Alzheon announced Thursday. Alzheons analysis of the previous trial data also found signs of improvement in patients who carry the ApoE4 gene, a variant that increases the risk of developing Alzheimers. Alzheons Phase 3 test enrolled 325 patients who carry two copies of ApoE4.The new Alzheimers medications approved by the FDA in the past two years, Leqembi from Eisai and Kisunla from Eli Lilly, are antibodies, large molecule drugs administered by infusion. While Leqembi and Kisunla may be used to treat such patients under their FDA approvals, the labels of these products carry black box warnings that flag this higher complication risk. Last June, privately held Alzheon raised $100 million in Series E financing to support completion of the pivotal study and prepare for potential commercialization of its Alzheimers drug.

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