Vesper Bio reports positive topline results for dementia candidate
Charlie Blackie-Kelly | November 7, 2025 | News story | Research and Development | Frontotemporal degeneration, Vesper Bio, clinical trials, dementia
Vesper Bio, a clinical-stage biotech developing novel oral therapies for neurodegenerative and neuropsychiatric disorders, has reported positive topline data from its phase 1b/2a clinical trial evaluating its lead candidate, VES001, in patients with frontotemporal degeneration (FTD).
FTD – also known as frontotemporal lobar degeneration (FTLD) – encompasses a group of brain disorders characterised by progressive degeneration of the frontal and temporal lobes. It is the leading cause of dementia in people under 60, affecting behaviour, judgement, communication and the ability to perform daily activities.
The open-label, single-arm phase 1b/2a SORT-IN-2 study assessed VES001, a selective sortilin inhibitor, in asymptomatic patients with FTD caused by mutations in the progranulin (GRN) gene. Participants received daily oral doses of VES001, initially at a lower dose followed by a higher dose escalation.
The trial demonstrated that VES001 was safe and well tolerated, and achieved a statistically significant increase in mean progranulin levels. According to Vesper Bio, the findings support advancement of VES001 into phase 2b and phase 3 studies to further evaluate its clinical efficacy and biomarker outcomes.
Mads Kjolby, Co-Founder and Chief Medical Officer at Vesper Bio, said: “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 this topline phase 1b/2a data, we believe VES001 has the potential to normalise progranulin levels not only in asymptomatic individuals with GRN mutations, but in symptomatic people too, without affecting other sortilin functions crucial for neuronal health. We therefore think VES001 has great potential to slow or even arrest FTD-GRN disease progression.”
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