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Vesper Bio ApS announces that the first volunteer has been dosed with its lead compound VES001, a patient friendly, first-in-class, brain penetrant, oral treatment, that could bring a meaningful disease-modifying benefit to patients suffering from Frontotemporal Dementia harbouring loss of function mutations in the progranulin gene. VES001 aims to rebalance circulating progranulin in life changing diseases.
Vesper has successfully initiated a randomised, double-blind, single and multiple ascending dose Phase I study in healthy volunteers. The study will assess safety, tolerability and pharmacokinetics and target engagement biomarkers across a range of single and multiple doses of this new treatment in healthy volunteers before continuing to proof of concept in asymptomatic GRN mutation carriers. As a small molecule, VES001 is ideal for convenient oral daily dosing and is designed to cross the blood brain barrier. There are currently no approved disease-modifying treatments for FTD(GRN) with current standard of care treatments only able to address symptoms of the disease, such as anxiety, depression and agitation.
Paul Little, PhD, Chief Executive Officer of Vesper Bio, said: “We have taken a significant step forward in bringing VES001 closer to treating patients suffering from FTD(GRN). 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 pre-clinical trials with our small molecule, VES001, have shown the effectiveness of targeting the uptake of progranulin in this way. I am grateful to the Vesper team, scientific advisors and patient groups who have been involved in bringing Vesper Bio to this important moment, as we transition to a clinical stage company.
“FTD is a disease that hits very hard and affects the lives of patients and their families in profound ways, making normal life all but impossible. We are striving to bring a product forward with the aim of halting this terrible disease.”
Anders Nykjaer, MD, PhD Chief Scientific Officer of Vesper Bio, commented: “The ultimate goal for every scientist in biomedical research is to make an observation that may enable the development of a treatment against a devastating or fatal disorder. I have been privileged to experience such a journey. Starting many years ago, when we identified the Vps10p-domain receptor sortilin, and later demonstrating that it may represent an attractive target to normalize progranulin levels that are commonly too low in FTD(GRN), we have today dosed the first human subjects with the first orally available sortilin antagonist. This has truly been one of the greatest experiences of my professional life.”