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VES001

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Brain-penetrant sortilin ligand with completed Phase 1 testing and company-reported Phase 1b/2a results in presymptomatic GRN mutation carriers.

Modality
oral brain-penetrant small-molecule sortilin ligand
Availability (US)
ClinicalTrials.gov Phase 1 study Completed; company reported positive Phase 1b/2a topline results in October 2025
Biomarkers
treatment-emergent adverse events; pharmacokinetics; plasma and cerebrospinal-fluid progranulin; sortilin pathway engagement
Hallmark targeted
lysosomal dysfunction, impaired autophagy, loss of proteostasis
Kind
investigational drug
Clinical trials
NCT06226064 — Phase 1, Completed on August 22, 2024 registry update; company-reported Phase 1b/2a proof-of-concept study