Danish biotechnology company Vesper Bio announced that it reached a participation milestone in its phase Ib/IIa clinical trial evaluating a treatment for FTD-GRN. Six participants have been enrolled in the study at two sites in Europe.

VES001 Supports Progranulin Production by Reducing Degradation of Proteins

In FTD caused by inherited mutations of the GRN gene, production of the neuroprotective protein progranulin is suppressed. A different protein, sortilin, binds to progranulin and targets it for degradation, further reducing production. Lowered progranulin levels are associated with neuronal dysfunction and abnormal protein accumulations that result in FTD.

Vesper Bio’s current trial is evaluating the drug VES001, which is designed to support progranulin production by selectively inhibiting sortilin. A phase 1 study of the drug in people without FTD-GRN, which concluded last year, produced encouraging data: VES001 normalized progranulin levels when dosed once or twice daily, and was shown to be safe to administer and well tolerated by participants.

The current phase Ia/IIb trial will involve participants with FTD-GRN, and will evaluate the efficacy of VES001 as a potential disease-modifying treatment. While the trial has reached its enrollment milestone, it is still recruiting at both trial sites in Europe: Erasmus University Medical Center in Rotterdam, the Netherlands, and the National Hospital for Neurology and Neurosurgery in London.

“There is a pressing need for a safe, effective and easy-to-administer treatment for people with frontotemporal dementia, a seriously debilitating condition for which there is currently no treatment available,” said Vesper Bio CEO Paul Little, PhD. “The rapid advancement of our VES001 clinical development program brings us one step closer towards meeting that significant need.”

Are you interested in participating in clinical trials like this? Join the FTD Disorders Registry to not only keep up to date on clinical trial opportunities, but to share your lived experiences to guide researchers. If you have any questions about clinical trial participation, reach out to AFTD’s HelpLine at 1-866-507-7222 or info@theaftd.org.