Biotechnology company Vesper Bio announces it has completed the single ascending dose stage in its clinical trial of its potentially disease-modifying treatment for FTD-GRN.

The goal of the single ascending dose stage was to determine how the body absorbs, distributes, and metabolizes the drug, as well as to monitor potential side effects. The study also allowed Vesper Bio to determine the highest safe dose that does not produce side effects.

VES001 is intended to treat FTD caused by mutations in the GRN gene by selectively inhibiting the protein sortilin. FTD-GRN causes abnormally low levels of another protein, progranulin, which plays an essential role in protecting neurons. Sortilin binds to progranulin and targets it for degradation, contributing to FTD-GRN. By inhibiting sortilin, Vesper Bio hopes to protect and preserve cells untouched by neurodegeneration.

According to Vesper Bio, data collected during the study demonstrates that VES001 is safe and well tolerated by participants. They also found the drug successfully entered the areas of the brain affected by FTD. Based on the current data, the company predicts the drug will require one to two daily doses.

“These excellent early-stage results are highly encouraging and spur us on to develop VES001 as a highly effective new treatment for FTD(GRN),” said Vesper Bio co-founder and chief medical officer Mads Kjølby, MD, PhD.

“Vesper Bio will continue to seek ways to bring this exciting discovery to benefit the lives of patients and their families. “We look forward to building on these exciting data and accumulating further evidence of the significant potential that sortilin inhibition has as a disease-modifying strategy.”

With the single ascending dose stage of the trial complete, Vesper Bio can now evaluate VES001 to determine the drug’s efficacy in people with FTD-GRN. In addition to gathering further safety data, this part of the first phase will determine if the drug shows enough promise to proceed to a larger-scale study.

Biotechnology and biopharmaceutical companies like Vesper Bio are making considerable progress in the search for effective interventions and diagnostic tools for FTD. Aprinoia Therapeutics and Transposon Therapeutics recently received Fast Track Designations for their respective PET tracer and FTD-GRN intervention.

Are you interested in getting involved in FTD research? The FTD Disorders Registry is a powerful tool that can connect you with opportunities to participate in research and let you guide researchers with your lived experiences of FTD.