Vesper Bio’s frontotemporal dementia (FTD) drug has increased progranulin levels in a Phase Ib/IIa trial, showing its potential in slowing or halting the development of the disease.
In the SORT-IN-2 trial (NCT06705192), oral VES001 was investigated in asymptomatic carriers with FTD caused by mutations in the progranulin gene (GRN).
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In the trial, mean progranulin levels increased by more than 95% compared to baseline in the high dose cohort, retuning them to a near normal level and confirming target engagement. Patients with FTD-GRN typically have progranulin concentrations that are around half that of people without GRN mutations.
The data also showed dose-dependent increases in progranulin protein levels in both plasma and cerebrospinal fluid (CSF).
Vesper’s co-founder and CMO Mads Kjolby said: “Based on these topline Phase Ib/IIa data, we believe VES001 has the potential to normalise progranulin levels not only in asymptomatic individuals with GRN mutations, but in symptomatic people too, without affecting other sortilin functions crucial for neuronal health.
“We therefore think VES001 has great potential to slow or even arrest FTD-GRN disease progression.”
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By GlobalDataThis trial data is the first validation of Vesper’s candidate in restoring normal progranulin levels.
VES001 remained well tolerated, with only a few mild reported adverse events (AEs). There were no severe AEs reported or discontinuations due to treatment-related effects.
Patients in the trial received daily oral doses of VES001, initially at a low dose for 28 days then a high dose for 56 days.
Full data from the trial, which is supported by the Alzheimer’s Drug Discovery Foundation (ADDF) and the Association for Frontotemporal Degeneration (AFTD) through the TreatFTD programme, is expected in Q1 2026.
Based on the full data, Vesper Bio will then prepare to initiate a Phase IIb/III clinical trial to evaluate VES001’s efficacy on clinical progression and biomarker endpoints in patients with symptomatic FTD-GRN.
VES001 is an oral inhibitor of sortilin, which regulates and promotes the degradation of progranulin, a pleiotropic protein that plays diverse roles in the brain. Pathogenic mutations in progranulin can lead to FTD.
This trial success will be welcome news for Vesper Bio, as there are currently no disease-modifying drugs approved for FTD.
There was a candidate in Phase III trials, Alector’s latozinemab; however, it failed to meet the primary endpoint in a Phase III trial last month, leading to the termination of its development.
According to GlobalData’s Pharmaceutical Intelligence Center, there are now 18 drugs in clinical trials for FTD, 11 in Phase I and seven in Phase II, including AviadoBio’s AVB-101.
GlobalData is the parent company of Clinical Trials Arena.
