POC study initiated for frontotemporal dementia treatment
Vesper Bio has initiated a Phase Ib/IIa Proof of Concept (POC) study of its lead candidate VES001 for frontotemporal dementia.
The SORT-IN-2 study will evaluate VES001 in patients with mutations in their progranulin-coding gene (GRN), which are causal for FTD(GRN). FTD(GRN) is an early onset degenerative brain disease which is invariably fatal.
SORT-IN-2 is an open-label, dual centre study assessing the clinical efficacy of VES001 in patients with GRN mutations who are currently asymptomatic. It will be performed at Erasmus University Medical Centre, Rotterdam, the Netherlands, under Principal Investigator (PI) Professor Harro Seelaar; and the Leonard Wolfson Experimental Neurology Centre clinical research facility, National Hospital for Neurology and Neurosurgery, University College London, UK, under PI Professor Jonathan Rohrer.
The first patient has now been enrolled and Vesper expects to have completed enrolment and dosing by mid-2025.
Mads Fuglsang Kjølby, Co-Founder and interim Chief Medical Officer at Vesper Bio, said: “The aim of this study is to further demonstrate the safety and tolerability of VES001 and confirm whether VES001 has a positive effect on progranulin levels in the cerebrospinal fluid and blood plasma of these patients. Progranulin is vital for maintaining neuronal health, however, progranulin levels in asymptomatic people with GRN mutations are typically half that of people without such mutations. Based on the data from our successful First-in-Human trial, we believe VES001 will normalise progranulin levels, and thus has great potential to slow or even arrest FTD(GRN) disease progression.”