In a first-in-human trial in 79 healthy adults, the oral drug VES001 was well tolerated and raised levels of progranulin, a protein that is too low in one genetic form of frontotemporal dementia, in blood and spinal fluid.
In the INFRONT-3 phase 3 trial, published in October 2026 in Alzheimer's & Dementia, latozinemab raised progranulin levels in people with frontotemporal dementia from GRN mutations, but didn't slow the disease over 96 weeks.
A meta-analysis of 21 studies estimated the global prevalence of frontotemporal dementia, a leading cause of dementia before age 65, at 30 cases per 100,000 people, with extreme differences between studies.