VectorY Therapeutics has received authorization from the U.K. Medicines and Healthcare products Regulatory Agency and approval from the European Medicines Agency to initiate its Phase 1/2 PIONEER-ALS clinical trial of VTx-002 in people living with amyotrophic lateral sclerosis.
The approvals expand a study already underway in the United States and cover European clinical sites in Belgium and the Netherlands, which will serve as primary European centers.
PIONEER-ALS is a multicenter, open-label, dose-escalation study evaluating two dose levels of VTx-002 and is expected to enroll 12 adults across the U.S., Europe and the U.K. The trial’s primary objectives are to evaluate safety, tolerability, pharmacokinetics and exploratory efficacy; secondary and exploratory measures include neurofilament light chain and TDP-43 pathway biomarker trajectories and clinical endpoints such as ALSFRS-R, slow vital capacity, hand-held dynamometry and survival.
VTx-002 is a first-in-class vectorized antibody designed to target pathological TDP-43 and enable sustained antibody expression in the central nervous system, and the company claims that TDP-43 aggregates are present in up to 97 percent of ALS patients. The company also reports it recently treated its first participant at its lead U.S. trial site.
