Benzinga | Jul 24, 2020 08:33AM EDT

Alector Reports Dosed First Patient In Pivotal Phase 3 INFRONT-3 Trial Evaluating AL001 In Patients With Frontotemporal Dementia

Alector, Inc. (NASDAQ:ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, today announced dosing of the first patient in its pivotal Phase 3 clinical trial, named INFRONT-3, evaluating AL001 in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). AL001 is the company's wholly owned, investigational human monoclonal antibody designed to modulate progranulin, a key regulator of immune activity in the brain.

Frontotemporal Dementia (FTD) is a rapidly progressing and severe form of dementia found most frequently in people less than 65 years old at the time of diagnosis. It affects 50,000 to 60,000 people in the United States and roughly 110,000 in the European Union. There are multiple heritable forms of FTD, and FTD-GRN patients represent 5% to 10% of all people with FTD. There are currently no approved treatments options available for FTD.

"The initiation of our global pivotal Phase 3 trial of AL001 is a significant step towards achieving our mission of helping patients suffering from FTD and other neurodegenerative diseases," said Robert Paul, M.D., Ph.D., chief medical officer of Alector. "Our Phase 3 study has been designed to measure slowing of disease progression in both symptomatic and pre-symptomatic FTD-GRN patients. We believe the INFRONT-3 trial will successfully demonstrate AL001's ability to significantly alter the course of FTD and potentially transform the lives of people affected by this condition. We are incredibly grateful to the patients, healthcare professionals and clinical sites that participated during earlier phases of study, allowing us to rapidly progress to Phase 3 evaluation in less than two years."

The randomized, double-blind, placebo-controlled trial will enroll up to 180 FTD-GRN mutation carriers across approximately 50 sites in the United States, Europe and Australia. Symptomatic and pre-symptomatic participants will be randomized to receive AL001 or placebo intravenously every four weeks. Participants will also be given the option to continue receiving treatment in an open-label extension study.

The primary endpoint of the pivotal Phase 3 trial is to measure the effect of AL001 on clinical decline by utilizing the CDR(r) plus NACC FTLD-SB assessment, which evaluates clinical impairments in behavior, language, memory, judgment, and functional activities in trial participants. In addition, the trial will assess secondary clinical endpoints, multiple biomarkers and safety.