Alector: First frontotemporal dementia patient dosed in Phase 1b study

Alector announced dosing of the first frontotemporal dementia patient in the Phase 1b portion of the Infront clinical study of AL001 after completion of the healthy volunteer single ascending dose escalation portion of the study. “AL001 was generally well tolerated, with no drug-related serious adverse events in healthy volunteers, achieving the study’s primary objective. Moreover, AL001 successfully demonstrated proof of mechanism in healthy volunteers by showing a dose dependent increase in progranulin levels, a disease specific biomarker, in plasma and in cerebrospinal fluid,” said Robert Paul, M.D., Ph.D., chief medical officer. In a subset of FTD patients, mutations in a single copy of the progranulin gene lead to a 50% or greater decrease in the level of progranulin, which in turn leads to development of FTD with greater than 90% penetrance. This subset of patients is known as FTD-GRN. Alector aims to deploy AL001 to increase the level of progranulin in FTD-GRN patients, by inhibiting a progranulin degradation mechanism. “We are proceeding with the Phase 1b study which will assess the safety of multiple doses of AL001 as a primary objective. AL001’s therapeutic approach is conceptually similar to enzyme replacement therapy and as such AL001 is designed to increase the level of progranulin in these FTD-GRN patients. In addition to the safety, we will also measure the levels of progranulin in plasma and in cerebrospinal fluid to demonstrate proof of mechanism in FTD-GRN patients by increasing the levels of this critical factor,” said Omer Siddiqui, vice president of development operations. “These data will inform future clinical studies, including rational dose selection in potential registrational studies.”

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