A new study is set to evaluate the safety and tolerability of a treatment in patients with mutations in their progranulin-coding gene (GRN), which causes frontotemporal dementia (FTD) – 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 the treatment VES001 in patients with GRN mutations who are currently asymptomatic.
It will be performed at one clinical centre in the Netherlands and another in the United Kingdom. The first patient has now been enrolled and enrolment and dosing is expected to be completed by mid-2025.
Mads Fuglsang Kjølby, co-Founder and interim Chief Medical Officer at Vesper Bio, which is carrying out the trial, 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.”
Paul Little, CEO at Vesper Bio, said: “It is an incredible achievement by the Vesper team that we have been able to progress VES001 so quickly into this next clinical trial phase.
“We are committed to bringing this important new oral treatment option to families living with FTD, where there is no approved therapy available today. We are all striving for a future free from FTD for patients and their families.”
The SORT-IN-2 study will be conducted at Erasmus University Medical Centre, Rotterdam, the Netherlands, under principal investigator Professor Harro Seelaar; and the Leonard Wolfson Experimental Neurology Centre clinical research facility, National Hospital for Neurology and Neurosurgery, University College London, UK, under principal investigator Professor Jonathan Rohrer.
