Successful next step for potential treatment of frontotemporal dementia

‘We are glad for our colleagues at Vesper Bio, and of course we really hope that this novel treatment will help reduce the burden of FTD’, says Dr Philip Kremer, Director of Neurology. FTD is a debilitating neurodegenerative disease with a relatively low age of onset, characterised by the degeneration of the brain’s frontal and temporal lobes. Based on research at Aarhus University, Danish biotech company Vesper Bio is exploring a possible oral treatment for FTD-GRN, a variant of FTD caused by genetically low levels of progranulin.

In CHDR’s 2024 Annual Report, Vesper Bio’s co-founder and Chief Medical Officer Dr Mads Kjolby told about his collaboration with CHDR: ‘We are a science-based company with strong roots in academia, and we were glad to meet a similar corporate culture at CHDR. It was a pleasure to collaborate.’ In this collaboration, the Neurology Group studied the investigational compound VES001 in healthy study participants. In addition to showing its safety and tolerability, the study confirmed target engagement and dose responsiveness for the pharmacodynamic effects.

Based on these findings, Vesper Bio conducted phase Ib/IIa studies in Rotterdam and London, focusing on healthy carriers of the GRN mutation, who have a very high risk of developing FTD later in life. According to Vesper Bio’s press release, VES001 has the potential to normalise progranulin levels both in asymptomatic individuals with GRN mutations and in symptomatic people. ‘We therefore think VES001 has great potential to slow or even arrest FTD-GRN disease progression’, said Kjolby in the press release. The company now plans to go on towards a Phase IIb/III clinical trial to evaluate VES001’s efficacy on clinical progression and biomarker endpoints in symptomatic FTD-GRN patients. Kremer: ‘We will follow the further development of this promising treatment, hoping that it will prove to be of significant value to both patients and asymptomatic carriers’.