Early-Phase Trial Launches of VES001 for Gene-Related Frontotemporal Dementia
Vesper Bio anticipates completing enrollment and dosing by mid-2025 in the Phase 1B/2A trial for VES001.
Vesper Bio announced the initiation of a Phase 1B/2A proof-of-concept trial of VES001 in patients with frontotemporal dementia (FTD) caused by mutations in the GRN gene (GRN), the company said in a news release.1
The SORT-IN-2 study (NCT06705192) will evaluate the safety, tolerability, and potential efficacy of VES001 in asymptomatic individuals.1
FTD is a rapidly progressive and invariably fatal neurodegenerative disorder in which patients harbor deficient levels of progranulin, a protein essential for neuronal health. VES001, administered orally, is designed to normalize these progranulin levels by targeting sortilin, a neuronal surface receptor that leads to progranulin degradation. By inhibiting this pathway, VES001 has the potential to maintain adequate progranulin concentrations in plasma and cerebrospinal fluid (CSF).1
SORT-IN-2 is an open-label, dual-center study enrolling asymptomatic patients with GRN mutations. Investigators will monitor changes in progranulin levels in both CSF and plasma, as well as general safety and tolerability measures. The trial will take place at Erasmus University Medical Centre in Rotterdam, the Netherlands and at the Leonard Wolfson Experimental Neurology Centre at the National Hospital for Neurology and Neurosurgery at University College in London.1
“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,” said Mads Fuglsang Kjølby, Co-Founder and interim Chief Medical Officer at Vesper Bio, said in a news release. “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."1
Vesper Bio previously completed a Phase 1A study of VES001 in healthy volunteers. Results showed an acceptable safety profile with no reported serious or treatment-emergent adverse events. Orally administered VES001 was detected in both plasma and central nervous system compartments, indicating broad distribution and strong target engagement, as evidenced by increased progranulin levels.1
“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,” Kjølby added.1
Vesper Bio anticipates completing enrollment and dosing by mid-2025, the company said.1
Reference
1. Vesper Bio Initiates Phase Ib/IIa Proof-of-Concept Study of VES001 in Asymptomatic Patients With Gene Mutations That Cause Frontotemporal Dementia (FTD). [News Release].Published January 7, 2025. Accessed January 23, 2025. https://www.vesperbio.com/_files/ugd/61ce8f_91afc73135404715a573a4bff693a706.pdf
