The Phase I trial will consist of one arm with 48 healthy participants receiving one single ascending dose, and the second arm receiving multiple ascending doses consisting of 30 subjects. Credit: Shutterstock / Atthapon Raksthaput.

Danish biotech firm Vesper Bio has dosed the first participant in the Phase I trial evaluating oral treatment VES001 in frontotemporal dementia (FTD).

The randomised, double-blind trial is examining the safety and tolerability of Vesper Bio’s compound VES001, described by the company as a first-in-class brain penetrant.

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The Phase I trial will consist of one arm with 48 healthy participants receiving one single ascending dose, and the second arm receiving multiple ascending doses consisting of 30 subjects.

Vesper Bio plans to follow this up with a Phase Ib/c proof of concept study with asymptomatic GRN mutation carriers.

VES001 works by blocking the uptake of progranulin into cells via the sortilin receptor, the drug candidate returns the amount of progranulin in the patient’s blood and nervous system to normal levels with the aim of protecting the brain from further damage.

Paul Little, CEO of Vesper Bio, said: “This is a devasting disease for both patients and their families and we hope to show that this is a potentially game-changing treatment. The role of progranulin is becoming better understood, with clinical validation using antibody approaches, and preclinical trials with our small molecule, VES001, have shown the effectiveness of targeting the uptake of progranulin in this way.”

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GlobalData’s Pharmaceutical Intelligence Center predicts that the FTD treatment market will be worth an estimated $39m by the end of 2024, with the figure expected to rise significantly to an estimated value of $1.5bn by the end of 2029. VES001 is competing with eight other treatments currently in Phase I trials, with nine in Phase II and only one in Phase III.

GlobalData is the parent company of Clinical Trials Arena.

So far eight other treatments have made it to market. However, Vesper Bio argues that currently there are no approved disease-modifying treatments for FTD.

In October 2023, UK firm AviadoBio commenced its Phase I/II study investigating AVB-101 gene therapy for patients with FTD targeting the same progranulin mutations.