Vesper Bio announces positive Phase Ib/IIa topline results for lead candidate VES001 for frontotemporal degeneration
- Phase Ib/IIa study found VES001, the first oral therapy being tested in FTD, led to >95% mean increase in progranulin levels in CSF, compared to baseline
- Topline results indicate VES001 normalises levels of progranulin in people who have a shortage of this vital protein for genetic reasons, potentially stopping them developing symptomatic frontotemporal dementia (FTD)
- VES001 had a favourable safety and tolerability profile during the three-month daily dosing regimen, with few adverse events (AEs) reported
- Results support progressing VES001 into Phase IIb/III trials in people with FTD-GRN, the variant of FTD caused by genetically low progranulin levels
Copenhagen, Denmark, 31 October 2025 – Vesper Bio ApS (“Vesper” or “the Company”), a clinical stage biotech developing novel oral therapies for neurodegenerative and neuropsychiatric diseases, today announces positive topline results from its Phase Ib/IIa SORT-IN-2 clinical study evaluating VES001 in asymptomatic carriers with frontotemporal degeneration (FTD) caused by mutations in the progranulin gene (GRN).
