On 16 March, Alector, a biotechnology company developing immuno-neurology therapies to treat neurodegenerative diseases, presented results from its Phase II clinical trial of AL001 at the AD/PD™ 2022 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders taking place virtually and in person in Barcelona, Spain. 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. INFRONT-2 is an open-label study investigating the safety and tolerability of AL001 in people at risk or with frontotemporal dementia. The study included three cohorts of participants with FTD: asymptomatic FTD-GRN mutation carriers, symptomatic FTD-GRN participants, and symptomatic FTD-C9orf72 participants. Data presented focused on the FTD-C9orf72 cohort and included 12-month data for up to 10 participants with at least one post-baseline clinical outcomes assessment, who received 60 mg/kg of AL001 every four weeks. As of the data cut, six FTD-C9orf72 participants had completed 12 months of treatment and all biomarker and clinical outcomes assessments. Preliminary results demonstrated that AL001 is generally well tolerated. The treatment with AL001 demonstrated target engagement and resulted in increases in progranulin levels in participants. Clinical outcome assessments found participants treated with AL001 demonstrated a trend toward a delay in disease progression. In addition, Alector is currently enrolling participants in the US, Europe and Asia for its INFRONT-3 Phase III pivotal clinical study of AL001. 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.
