#Denmark #Copenhagen #Vesper Bio #FTD #VES001

Vesper Bio Initiates Pioneering Study for Frontotemporal Dementia Treatment

In a significant advancement for the treatment of frontotemporal dementia (FTD), Vesper Bio has launched a Phase Ib/IIa proof-of-concept study for its leading drug candidate, VES001. This clinical trial targets asymptomatic patients with specific gene mutations known to cause this form of dementia, namely the GRN mutations. Following approvals from the Netherlands and the United Kingdom, the SORT-IN-2 trial aims to explore the therapeutic potential of VES001, which is considered a first-in-class, orally administered treatment that penetrates the brain.

FTD is a relentless neurodegenerative disease that severely impacts cognitive functions and behavior, often leading to fatal outcomes. FTD(GRN), the form of FTD that the study focuses on, results from mutations in the progranulin (GRN) gene, leading to significantly reduced levels of progranulin, which is a protein crucial for neuronal health. It is estimated that patients with FTD(GRN) experience a drastic drop in progranulin levels, potentially being halved compared to those without the mutation. VES001 is engineered to normalize these levels, offering hope to the estimated 17,400 individuals currently affected in key markets.

The SORT-IN-2 study will be conducted in two participating centers: the Erasmus University Medical Centre in Rotterdam, Netherlands, and the Leonard Wolfson Experimental Neurology Centre at the National Hospital for Neurology and Neurosurgery, University College London, UK. The principal investigators overseeing this landmark study are Professor Harro Seelaar and Professor Jonathan Rohrer, respectively. Early recruitment efforts have begun, with Vesper aiming to complete dosing of all study participants by mid-2025.

According to Mads Fuglsang Kjølby, co-founder and interim Chief Medical Officer at Vesper Bio, the primary goal of the study is to evaluate both the safety and the tolerability of VES001. Moreover, it seeks to verify whether the treatment can positively affect the levels of progranulin in the cerebrospinal fluid and blood plasma of patients harboring GRN mutations. The research approach is promising, especially given previous favorable outcomes from the initial human trials of VES001, which showed high safety and tolerability. There were no reported significant adverse events, reinforcing the potential of the treatment to significantly enhance progranulin levels in the body.

Paul Little, the Chief Executive Officer at Vesper Bio, expressed pride in the company's rapid progression into this next phase of clinical trials. He emphasized the company's commitment to delivering this oral treatment option to families grappling with FTD, a disease currently without FDA-approved therapies. Little underscored the urgent need for a solution, voicing shared hopes for a future free of FTD challenges for affected individuals and their families.

The unique mechanism of VES001 involves its capacity to interact with sortilin, a receptor on neuronal surfaces that competes with other receptors for binding progranulin. The process which ensues results in the protection of cells by stabilizing and sustaining adequate progranulin levels. Achieving normalization of progranulin levels is pivotal, especially as FTD patients see a decline in these levels due to the receptor’s activity.

Frontotemporal dementia encompasses a group of brain disorders impacting the frontal and temporal lobes, influencing personality, relationships, and functional abilities. It stands as the predominant dementia cause in individuals below 60 years and is frequently misidentified as Alzheimer's disease. The hereditary nature of FTD(GRN) adds a crucial layer, drawing attention to the genetic underpinnings of this ailment. With a focus on innovative treatment options and the latest scientific advancements, Vesper Bio is at the forefront of battling this debilitating disease, promising brighter prospects for current and future patients.

In conclusion, as Vesper Bio embarks on this significant clinical trial, the hope for modifying the course of frontotemporal dementia draws closer to realization, paving the way for improved quality of life and potential long-term remission for individuals afflicted by this harrowing condition.