ALECTOR, INC. Alector, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to latozinemab, an investigational human monoclonal antibody designed to block sortilin to elevate progranulin (PGRN) levels for the potential treatment of frontotemporal dementia with a progranulin gene mutation (FTD-GRN). The FDA granted latozinemab Breakthrough Therapy Designation for FTD-GRN based upon data from the INVOKE-2 Phase 2 clinical trial of latozinemab in FTD-GRN participants. The FDA's Breakthrough Therapy Designation is granted to expedite the development and review of drugs in the United States that are intended to treat a serious condition, when preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint.
Latozinemab (AL001) is an investigational human monoclonal antibody designed to modulate progranulin (PGRN), a key regulator of immune activity in the brain with genetic links to multiple neurodegenerative disorders, including frontotemporal dementia (FTD), Alzheimer?s disease, and Parkinson?s disease. Latozinemab aims to increase the level of PGRN in humans by inhibiting sortilin, a degradation receptor for PGRN. Latozinemab has received Orphan Drug Designation for the treatment of FTD as well as both Breakthrough Therapy and Fast Track designations for the treatment of FTD due to a progranulin gene mutation (FTD-GRN) from the U.S. Food and Drug Administration.
Frontotemporal dementia (FTD) is a rare neurodegenerative disease, but it is one of the most common causes of early onset dementia. It affects an estimated 50,000 to 60,000 people in the United States and roughly 110,000 in the European Union, with potentially higher prevalence in Asia and Latin America. There are multiple heritable forms of FTD, and FTD patients with a progranulin gene mutation (FTD-GRN) represent 5% to 10% of all people with FTD.
Patients with FTD frequently develop symptoms such as behavioral changes, lapses in judgment, and diminished language skills when they are in their 40?s and 50?s with the disease running its course in 7-10 years.6 There are no U.S. Food and Drug Administration-approved treatment options available for any form of FTD.
