▎Edited by the WuXi AppTec content team

Alector Therapeutics and GSK announced today that the U.S. FDA hasGranting Breakthrough Therapy Designation to Investigational Antibody Therapy Latozinemab. This potential "first-in-class" humanized monoclonal antibody aims to inhibit a receptor protein named sortilin, thereby increasing the levels of progranulin (PGRN). It is currently being used to treat conditions associated with the encoding of PGRN.GRNPatients with Frontotemporal Dementia (FTD) due to gene mutation.

This breakthrough therapy designation is based on the results of a Phase 2 clinical trial named INFRONT-2. The trial data showed,Latozinemab restores PGRN levels in patient plasma and cerebrospinal fluid to those of age-matched healthy volunteers.

▲LatOzinemab Restores PGRN Levels in Patient Plasma and Cerebrospinal Fluid (Image Source: Alector's Official Website)

At the same time, preliminary evaluation data shows,Compared to historical controls, latozinemab may slow disease progression in patients.

▲Latozinemab may slow down the progression in carriersGRNDisease Progression in Frontotemporal Dementia Patients with Gene Mutations (Image Source: Alector's Official Website)

Latozinemab (AL001) is a monoclonal antibody targeting PGRN. PGRN is a key regulator of immune responses in the brain and is associated with various neurodegenerative diseases, including FTD, Alzheimer's disease, and Parkinson's disease. The mechanism of action of Latozinemab involves inhibiting sortilin, the receptor that mediates PGRN degradation, with the aim of increasing PGRN levels in patients. It has received Orphan Drug Designation, Fast Track Designation, and Breakthrough Therapy Designation from the FDA. GSK has partnered withAlector Achieved in 2021R&D Collaboration, and jointly develop this antibody.

FTD represents a group of rare neurodegenerative diseases and is one of the leading causes of dementia in young adults. FTD includes various genetic forms, with 5% to 10% of FTD cases associated withGRNIt is related to gene mutations. Symptoms typically appear between the ages of 40 and 50, manifesting as behavioral changes, impaired judgment, and declining language abilities. Currently, the U.S. FDA has not approved any treatment for FTD.

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