Vesper Bio announces data from Phase I frontotemporal dementia treatment trial

Vesper Bio has shared results from a Phase I study of VES001, a potential disease-modifying treatment for frontotemporal dementia (FTD) targeting patients with progranulin gene mutations (GRN). The trial began in December with the first participant and included 78 healthy subjects to evaluate VES001's safety, tolerability, pharmacokinetics, and pharmacodynamics.

The study was divided into two parts, assessing different dose levels. Both the single ascending dose (SAD) and seven-day multiple ascending dose (MAD) stages met their endpoints. VES001 exhibited a strong safety profile with no serious adverse events and showed effective distribution to both plasma and the central nervous system. It also demonstrated significant target engagement, increasing progranulin levels in participants.

Following these positive results, Vesper Bio has applied for a clinical trial application (CTA) to start a Phase IIa proof-of-concept study, with patient dosing planned for Q4 2024. VES001 is designed as a brain-penetrant, small-molecule sortilin inhibitor to help manage FTD by maintaining progranulin levels through daily oral dosing.