VectorY Therapeutics has expanded the study size for its ongoing, global Phase I/II PIONEER-ALS clinical trial of VTx-002 for amyotrophic lateral sclerosis (ALS).
The dose-escalation, open-label, multicentre study is evaluating the tolerability, safety, pharmacodynamic, and pharmacokinetics effects of VTx-002 in individuals diagnosed with the disease.
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The trial is expanding from 12 to 36 participants across clinical sites located in Europe, the UK, and the US while assessing two dose levels of the therapy.
VectorY Therapeutics initiated dosing for its PIONEER-ALS clinical trial, starting with the first US participants in February 2026 at Mass General’s Sean M. Healey & AMG Center for ALS. Dosing subsequently expanded to Europe in September at the University Medical Center Utrecht.
According to the company, rapid participant recruitment and site activations facilitated the expansion to generate a broader clinical dataset.
VTx-002 is an unapproved, investigational vectorised antibody designed to target abnormal accumulations of the TDP-43 protein, which occurs in up to 97% of ALS cases.
The therapy uses a vector delivery system intended to sustain antibody expression within the central nervous system.
Alongside safety and pharmacological measures, the study is evaluating secondary and exploratory endpoints such as post-treatment Neurofilament light chain (NfL) trajectories and new TDP-43 pathway-related biomarkers.
It is also assessing clinical endpoints including the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) scores, slow-vital capacity, hand-held dynamometry, and survival.
ALS is a fatal neurodegenerative condition with no cure and limited treatment options for symptom management.
VectorY CEO Jim Scibetta said: “With VTx-002, we directly target TDP-43, which is the pathological driver in the vast majority of ALS cases.
“Tripling the study size significantly increases our opportunity to utilise its secondary and exploratory endpoints to obtain a better preliminary understanding whether VTx-002 is impacting the underlying biology of ALS in a way that may ultimately translate to clinical benefit and disease modification.”
VectorY is also developing candidates including VTx-002 for ALS, VTx-003 for Huntington’s disease, VTx-004 for frontotemporal dementia, and VTx-005 for Alzheimer’s disease.
