The pharmacokinetic data revealed that the asset is effectively distributed to plasma and the central nervous system after oral administration. Credit: Atthapon Raksthaput/Shutterstock.

Vesper Bio has reported data from a Phase I study of VES001, a potential disease-modifying treatment for frontotemporal dementia (FTD), specifically targeting patients with progranulin gene mutations (GRN).

Last December, the company dosed the first participant in the trial.

The trial, which involved 78 healthy subjects, was designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamic target engagement of VES001.

It comprised two parts, with separate dose levels evaluated for their impact.

The study results confirmed that all endpoints were met, with findings from the seven-day multiple ascending dose (MAD) stage reinforcing the data from the findings in the single ascending dose (SAD) stage reported in May this year.

Throughout the trial, VES001 demonstrated a high safety and tolerability profile without any serious or treatment-emergent adverse events.

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The pharmacokinetic data revealed that VES001 is effectively distributed to plasma and the central nervous system after oral administration.

Moreover, the treatment showed significant target engagement, with progranulin levels increasing in volunteers who received daily doses.

The data also suggested that VES001 could substantially normalise progranulin concentration in patients after seven days of dosing.

Based on these promising findings, Vesper Bio has submitted a clinical trial application (CTA) to initiate a Phase IIa proof-of-concept study with patient dosing expected to commence in the fourth quarter of 2024.

VES001 is a brain-penetrant, small-molecule sortilin inhibitor developed to modify the course of FTD by inhibiting the sortilin receptor, which competes with progranulin receptors and leads to neuronal damage when progranulin binds to it.

By preventing this binding, VES001 aims to maintain and normalise progranulin levels, offering a convenient and patient-friendly treatment option through daily oral dosing.

Vesper Bio CEO Paul Little said: “At Vesper, we are motivated by our patients and their relatives, who inspire our mission to develop innovative therapies that can help fight this awful disease.

“These promising clinical data coupled with VES001’s patient-friendly profile bring us one step closer to transforming patient outcomes in frontotemporal dementia.”