Danish clinical stage biotech Vesper Bio ApS announced on Wednesday that it has successfully completed a Phase I study for its lead candidate, VES001, a potential disease-modifying treatment for frontotemporal dementia (FTD).

The study demonstrated excellent safety, tolerability and target engagement.

VES001 is a first-in-class oral, brain-penetrant, small molecule sortilin inhibitor designed to increase progranulin levels in FTD patients with mutations in the progranulin gene (FTD(GRN)). The Phase I data support the advancement of VES001 into a Phase IIa proof-of-concept study.

Study results showed high levels of safety and tolerability, with no serious adverse events reported. VES001 demonstrated strong target engagement, with increased levels of progranulin observed in both plasma and the central nervous system.

Vesper Bio has filed a clinical trial application for a Phase IIa study, with dosing expected to begin in the fourth quarter of 2024.