Vesper Bio completes single ascending dose trial of VES001 for fronto-temporal dementia

COPENHAGEN, Denmark, May 30, 2024 – Vesper Bio ApS, a clinical-stage biotech company and leader in sortilin receptor biology, has successfully completed the single ascending dose stage of its first-in-human trial for VES001, an innovative oral therapy targeting frontotemporal dementia (FTD(GRN)).

VES001 represents the first brain-penetrant, small molecule sortilin inhibitor designed to modify disease progression in neuro-cognitive disorders like FTD(GRN). The recent study (NCT06226064) involving healthy volunteers demonstrated that VES001 is both safe and well-tolerated across various doses. This treatment has shown promising pharmacokinetic properties and successful distribution to crucial brain areas, indicating the potential for effective once or twice daily dosing.

Crucially, participants administered VES001 exhibited significant elevations in progranulin levels, validating the drug's target engagement. Progranulin is essential for neuron survival, growth, and function, and individuals with FTD(GRN) typically have genetic mutations that lead to reduced progranulin levels.

Mads Kjølby, Chief Medical Officer of Vesper Bio, expressed enthusiasm over the initial results. "These positive early-stage outcomes are highly motivating, pushing us to advance VES001 as an effective treatment for FTD(GRN). Vesper Bio remains committed to translating this promising discovery into tangible benefits for patients and their families."

Kjølby further discussed the broader implications of sortilin inhibition, suggesting it might be a viable strategy not just for FTD(GRN) but also for other central nervous system diseases characterized by progranulin deficiency, neuroinflammation, and neuronal death, such as Parkinson's disease.

Sortilin, a receptor on neuron surfaces, competes with progranulin receptors. When progranulin binds to sortilin, it is internalized and degraded, potentially causing neuron damage. VES001 blocks sortilin receptors, allowing progranulin to be internalized by its own receptors, thus preventing neuronal damage.

Paul Little, Chief Executive Officer of Vesper Bio, praised the dedication of the Vesper team and the support from Lundbeckfonden BioCapital. "Our rapid progress is a testament to our team's relentless efforts and our partners' unwavering support. We are committed to translating groundbreaking science into a new class of CNS drugs to address significant unmet medical needs."

About Vesper Bio

Vesper Bio is a clinical-stage biotech company that leads in sortilin receptor biology research. The company's primary program involves a sortilin inhibitor aimed at rebalancing progranulin levels in patients. Progranulin is essential for cell growth, survival, and repair, and insufficient levels are linked to cellular dysfunction in various neurological conditions. Vesper believes that by normalizing progranulin levels, its compounds can have a disease-modifying effect, preserving existing cells.

VES001, Vesper's lead compound, is an innovative, brain-penetrant, oral treatment targeting progranulin deficiency, a significant cause of frontotemporal dementia (FTD). As an orally administered small molecule, VES001 can cross the blood-brain barrier, making it an optimal treatment option for patients experiencing rapid cognitive decline.

About Frontotemporal Dementia (FTD)

Frontotemporal dementia (FTD), also known as frontotemporal lobar degeneration (FTLD), encompasses brain disorders leading to degeneration in the frontal and temporal lobes. FTD affects behavior, judgment, communication, and daily activity participation. It is the most prevalent dementia type in people under 60 and is often misdiagnosed as Alzheimer's disease. FTD(GRN), a variant of FTD, arises from mutations in the progranulin gene (PGRN), resulting in low progranulin levels and accounting for about 25% of FTD cases.

Overall, Vesper Bio continues to make significant strides in developing treatments that address the underlying causes of neurodegenerative diseases, bringing hope to affected patients and their families.

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