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( MENAFN - PR Newswire) COPENHAGEN, Denmark, May 30, 2024 /PRNewswire/ -- Vesper Bio ApS ("Vesper" or "the Company"), a clinical stage biotech and world leader in sortilin receptor biology, today announces completion of the single ascending dose stage of its first-in-human trial of VES001 in healthy volunteers. VES001 is the first oral, brain penetrant, small molecule sortilin inhibitor designed as a potentially disease-modifying treatment for the neuro-cognitive disorder fronto-temporal dementia (FTD(GRN)). Data returned from this study (NCT06226064) demonstrate the safety and tolerability of VES001 across the full range of doses tested and show it has excellent pharmacokinetics and distribution to relevant parts of the brain.

The data predict a once or twice daily efficacious dose. Importantly, volunteers who received VES001 experienced significant and robust increases in levels of progranulin, demonstrating target engagement. Progranulin is a protein known to play a key role in promoting neuron survival, growth and function.



People with FTD(GRN) have inherited gene mutations associated with low progranulin levels. Mads Kjølby, Chief Medical Officer of Vesper Bio, said : "These excellent early-stage results are highly encouraging, and spur us on to develop VES001 as a highly effective new treatment for FTD(GRN). Vesper Bio will continue to seek ways to bring this exciting discovery to benefit the lives of patients and their families.

" He added: "We look forward to building.

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