On 12 November, Alector, a biotechnology company developing immuno-neurology therapies to treat neurodegenerative diseases, presented results from its Phase II clinical trial of AL001 at the 14th Clinical Trials on Alzheimer's Disease (CTAD). AL001 is a monoclonal antibody developed for the treatment of people at risk for or with symptomatic frontotemporal dementia (FTD) due to a progranulin gene mutation (FTD-GRN). AL001 is designed to elevate the levels of progranulin, which is a key regulator of immune activity in the brain.
The INFRONT-2 Phase II clinical trial was designed to assess the safety and tolerability of chronic dosing of AL001 in people at risk or with frontotemporal dementia. Overall, AL001 was well tolerated in the Phase II study. Data presented focused on the symptomatic FTD-GRN Cohort and included 12-month data for up to twelve participants who received 60 mg/kg of AL001 every four weeks. Findings showed that AL001 restored progranulin to normal levels in both plasma and cerebrospinal fluid. Multiple biomarkers of lysosomal dysfunction and complement activation, known to be elevated in this cohort, decreased toward normal levels following treatment with AL001.
In addition, Alector is currently enrolling participants in its INFRONT-3 Phase 3 pivotal clinical study of AL001. The study aims to recruit up to 180 people with at-risk and symptomatic carriers of the progranulin mutation causative of FTD in the US, Europe and Asia. Participants will receive either AL001 or placebo intravenously every four weeks for the duration of the 96-week study and will be given the option to continue receiving treatment in an optional open-label extension study after the 96-week treatment period.