VectorY Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its investigational new drug (IND) application to commence the PIONEER-ALS Phase I/II clinical trial of VTx-002 in amyotrophic lateral sclerosis (ALS).
VTx-002 is a vectorised antibody targeting transactive response DNA-binding protein 43 kDa (TDP-43) pathology in ALS.
It is designed to target toxic forms of TDP-43 selectively, aiming to restore normal nuclear function, reduce aggregation, and correct mis-splicing abnormalities.
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The dose-escalation, open label Phase I/II clinical study will enrol 12 adult patients with ALS to assess two dose levels of VTx-002.
The primary aim is to evaluate the tolerability and safety, with exploratory and secondary endpoints focusing on post-treatment Neurofilament light chain and TDP-43 biomarker trajectories.
Clinical endpoints include slow-vital capacity, amyotrophic lateral sclerosis functional rating scale-revised (ALSFRS-R), survival, and hand-held dynamometry.
With the IND clearance, VTx-002 is expected to become the first clinical-stage therapeutic targeting the full spectrum of TDP-43 pathology in ALS.
In non-human primate trials, VTx-002 demonstrated a favourable safety profile, consistent with other therapies delivered using AAV5.
VectorY CEO Jim Scibetta said: “The FDA’s clearance to proceed with our Phase I/II study marks a pivotal milestone for VectorY, as we strive to transform the neurodegenerative disease landscape with our novel vectorised antibodies that are specifically designed to address the well-established biology driving disease manifestations.
“We are collaborating with PIONEER-ALS Global Coordinating Investigator James Berry, chief of the division of motor neuron diseases and director of the Neurological Clinical Research Institute (NCRI) at Mass General Brigham, and with the ALS patient advocate and physician community, as we actively work towards trial initiation.”
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