FDA Grants Latozinemab Breakthrough Therapy Designation for Frontotemporal Dementia Due to a Progranulin Gene Mutation (FTD-GRN)

Core Insights - Latozinemab has received Breakthrough Therapy Designation from the FDA for the treatment of frontotemporal dementia with a progranulin gene mutation (FTD-GRN), marking it as the first investigational medicine to achieve this status for FTD-GRN [1][2] - The drug is currently being studied in the pivotal INFRONT-3 Phase 3 clinical trial, which reached its target enrollment in October 2023 [2] - Alector, in collaboration with GSK, aims to address the unmet medical need for FTD-GRN, a serious condition with no FDA-approved treatment options available [2][4] Company Overview - Alector is a clinical-stage biotechnology company focused on immuno-neurology, targeting immune dysfunction as a root cause of neurodegenerative diseases [6] - The company has developed a portfolio of programs aimed at repairing genetic mutations affecting the brain's immune system [6] - Alector is headquartered in South San Francisco, California [7] Collaboration with GSK - Alector entered into a collaboration and license agreement with GSK in July 2021, receiving $700 million in upfront payments [5] - The agreement includes potential additional payments of up to $1.5 billion based on clinical development, regulatory, and commercial milestones [5] - Profits and losses from the commercialization of latozinemab and AL101 will be shared equally in the U.S., while Alector will receive double-digit tiered royalties outside the U.S. [5] Disease Context - Frontotemporal dementia (FTD) is a rare neurodegenerative disease and a leading cause of early onset dementia, affecting an estimated 50,000 to 60,000 people in the U.S. and approximately 110,000 in the European Union [4] - FTD patients with a progranulin gene mutation (FTD-GRN) represent 5% to 10% of all FTD cases, with symptoms typically manifesting in individuals in their 40s and 50s [4]