VectorY Therapeutics has dosed the first participant in its Phase I/II PIONEER-ALS clinical trial, which is investigating VTx-002, a vectorised antibody targeting TDP-43 pathology, in individuals with amyotrophic lateral sclerosis (ALS).
The first participant received the treatment at the Sean M Healey & AMG Center for ALS at Mass General Brigham.
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The open-label, dose-escalation, multi-centre trial will assess two dose levels of VTx-002 and aims to enrol 12 adults with ALS across sites in Europe, the UK, and the US.
Its primary objective is to evaluate tolerability, safety, exploratory efficacy and pharmacokinetics of VTx-002 in ALS patients.
Secondary and exploratory endpoints have been set to optimise future pivotal clinical development. These include assessments of post-treatment Neurofilament light chain (NfL), TDP-43 pathway-related biomarker trajectories, along with clinical measures such as handheld dynamometry, slow vital capacity, survival rates, and amyotrophic lateral sclerosis functional rating scale-revised (ALSFRS-R) score.
VectorY Therapeutics plans to proceed with participant enrolment for the Phase I/II PIONEER-ALS trial across several sites.
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By GlobalDataVTx-002 uses a vector-based delivery platform and is designed for sustained antibody expression in the central nervous system to target underlying ALS biology.
VectorY Therapeutics’s chief medical officer Olga Uspenskaya-Cadoz said: “ALS is a fatal neurodegenerative disease with no cure and only limited symptomatic treatment options available.
“The initiation of dosing in the PIONEER-ALS trial is a significant milestone for VectorY as we strive to transform the neurodegenerative disease landscape with novel disease-modifying approaches.
“This trial marks the first ever clinical evaluation of a therapy designed to holistically target TDP-43 pathology in ALS, and thereby reduce TDP-43 aggregation, correct mis-splicing abnormalities, and restore normal nuclear function, and we are excited to advance this novel potential therapeutic strategy to a community of patients who are actively looking for hope.”
In December 2025, VectorY received clearance from the US Food and Drug Administration for its investigational new drug application to commence the PIONEER-ALS Phase I/II clinical trial of VTx-002.
