Preclinical and clinical investigation of VES001, an oral small molecule sortilin receptor inhibitor for the treatment of frontotemporal dementia: a first-in-human randomized placebo-controlled phase 1 trial
Louise Klem, Daniel Dumas, Imogen J Swift, Sofie L Frandsen, Ditte Nedergaard Mikkelsen, Laerke Fensbaek, Yalçin Yavuz, Okke Merton, Jesminne Castricum, Simone Prichardt, Graham M Wynne, Anders Riisager, Nina Schultz, Paul B Little, Ove Pedersen, Eva Thijssen, Anders Nykjaer, Philip Kremer, Mads Kjo · 2026
Read Preclinical and clinical investigation of VES001, an oral small molecule sortilin receptor inhibitor for the treatment of frontotemporal dementia: a first-in-human randomized placebo-controlled phase 1 trial by Louise Klem, Daniel Dumas, Imogen J Swift, Sofie L Frandsen, Ditte Nedergaard Mikkelsen, Laerke Fensbaek, Yalçin Yavuz, Okke Merton, Jesminne Castricum, Simone Prichardt, Graham M Wynne, Anders Riisager, Nina Schultz, Paul B Little, Ove Pedersen, Eva Thijssen, Anders Nykjaer, Philip Kremer, Mads Kjo (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-13669324.txt.
Description
Louise Klem and a large team test a first-in-human dementia drug that raises progranulin. Their phase one trial from twenty twenty-six covers the oral drug VES001. Low progranulin from a gene fault causes frontotemporal dementia. Blocking the sortilin receptor lifts progranulin without removing the receptor.
Animal work first showed rises in blood, spinal fluid, and brain fluid. Seventy-nine healthy adults then joined the randomised human trial. Single and week-long dosing schedules were both tested. Safety goals were met with no serious drug-linked harms. Headache was among the common minor complaints reported. Patient trials can now test whether raising progranulin helps. Early human data justify that next step.
Catalog record
| CREATOR | Louise Klem, Daniel Dumas, Imogen J Swift, Sofie L Frandsen, Ditte Nedergaard Mikkelsen, Laerke Fensbaek, Yalçin Yavuz, Okke Merton, Jesminne Castricum, Simone Prichardt, Graham M Wynne, Anders Riisager, Nina Schultz, Paul B Little, Ove Pedersen, Eva Thijssen, Anders Nykjaer, Philip Kremer, Mads Kjo |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 77 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |