The Association for Frontotemporal Degeneration (AFTD) and the Alzheimer’s Drug Discovery Foundation (ADDF) recently awarded $2.5 million as part of their shared funding opportunity called the Treat FTD Fund to Danish biotechnology company Vesper Bio for testing their drug VES001 in people who have variants in the progranulin gene (GRN), connected with FTD. 

Treat FTD Grant Comes Amid Positive News from VES001 Trial 

The Treat FTD Fund supports early-stage clinical trials (phases 1 and 2) testing new treatments or repurposed treatments for FTD. These treatments may be for genetic or sporadic FTD and are meant to answer new questions by being innovative in trial design or the metrics used to assess the treatment. AFTD’s portion of the Treat FTD Fund is generously provided by the Samuel I. Newhouse Foundation. 

The announcement of funding comes as Vesper Bio released positive interim results for its phase Ib/IIa study evaluating VES001 in people who have variants of the GRN gene but are asymptomatic for FTD. Study data support progressing the experimental drug into the next phase of clinical research. The Treat FTD award will therefore support further analysis of the phase Ib/IIa study data, as well as preparations for a possible next phase of the trial. 

“The Treat FTD Fund reflects AFTD’s commitment to advancing promising treatments like VES001 through the complex process of drug development for FTD,” said AFTD CEO Susan L-J Dickinson, MSGC. “With support from our donors and our partnership with the ADDF, we’re proud to help researchers move this work forward.” 

VES001 Led to Increased Progranulin Levels 

In FTD-GRN, production of the essential neuroprotective protein progranulin is impaired due to a variant of the GRN gene, which carries genetic instructions for making the protein. Progranulin can bind to a separate protein called sortilin, which sends it to be degraded, thereby further reducing production of the protein. Lower progranulin levels are associated with neuronal dysfunction and contribute to the abnormal accumulation of proteins that drives FTD.  

Vesper Bio’s experimental drug VES001 is an oral formulation designed to selectively inhibit sortilin, thereby promoting progranulin production without completely suppressing sortilin activity. According to Vesper Bio, interim results from the SORT-IN-2 study evaluating VES001 show that the drug successfully increases progranulin levels. Mean progranulin levels in participants increased by over 95% compared to baseline, reflecting a significant normalization of progranulin levels. 

“Progranulin is vital for maintaining neuronal health. However, progranulin levels in asymptomatic people with GRN mutations are typically half that of people without such mutations,” said Vesper Bio co-founder and chief medical officer Mads Kjolby. “Based on these topline Phase Ib/IIa data, we believe VES001 has the potential to normalize progranulin levels not only in asymptomatic individuals with GRN mutations, but in symptomatic people too, without affecting other sortilin functions crucial for neuronal health.”  

The current study included six participants from the UK and the Netherlands who carried GRN mutations associated with FTD. In addition to confirming that the desired drug mechanisms were taking place, the study demonstrated positive safety and tolerability for the drug. The only adverse effects reported were mild, and no participants had to discontinue VES001 due to side effects.  

Learn More About This and Other Clinical Trials 

Vesper Bio’s SORT-IN-2 trial entered phase Ib/IIa in January after previously announcing the completion of its phase 1 clinical studies in September 2024, demonstrating the drug was well-tolerated. The trial reached its enrollment milestone in June, with six participants successfully enrolled at the two study sites.  

For the most up-to-date information on this and other trials, refer to clinicaltrials.gov, (SORT-IN-2 specific page) and/or participate in the FTD Disorders Registry, which keeps the community informed of new and emerging research opportunities.