BOSTON — VectorY Therapeutics said it has dosed the first participant in its Phase 1/2 PIONEER-ALS clinical trial evaluating VTx-002, a first-in-class vectorized antibody designed to target TDP-43 pathology in people with amyotrophic lateral sclerosis.
The first participant was treated at the Sean M. Healey & AMG Center for ALS at Mass General Brigham, which is serving as the lead clinical trial site. VTx-002 recently received Fast Track designation from the U.S. Food and Drug Administration, a status intended to expedite the development and review of therapies for serious conditions with unmet medical needs.
“ALS is a fatal neurodegenerative disease with no cure and only limited symptomatic treatment options available,” said Olga Uspenskaya-Cadoz, M.D., Ph.D., chief medical officer of VectorY. “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.”
PIONEER-ALS is a multicenter, open-label, dose-escalation Phase 1/2 study that will evaluate two dose levels of VTx-002 and is expected to enroll 12 adults with ALS across clinical sites in the United States, Europe, and the United Kingdom. The primary objective of the trial is to assess safety, tolerability, pharmacokinetics, and exploratory efficacy. Secondary and exploratory endpoints include post-treatment measurements of neurofilament light chain and TDP-43 pathway-related biomarkers, as well as clinical measures such as ALSFRS-R, slow vital capacity, hand-held dynamometry, and survival.
“There remains a critical need for disease-modifying therapies in ALS,” said James Berry, MD, MPH, global coordinating investigator for the trial and chief of the Division of Motor Neuron Diseases and director of the Neurological Clinical Research Institute at Mass General Brigham. “VTx-002 is engineered to enable sustained, localized delivery of an antibody targeting pathological TDP-43, a hallmark of ALS, with the goal of addressing a central driver of disease progression. The initiation of this study reflects an innovative approach aimed at one of the most important pathological features of the disease.”
VectorY said it plans to continue enrolling participants in the PIONEER-ALS trial across multiple clinical sites.
VTx-002 is designed to target pathological TDP-43, a protein that aggregates abnormally in up to 97 percent of people with ALS. The therapy uses a vector-based delivery platform intended to enable sustained antibody expression within the central nervous system, with the goal of addressing the underlying biology of the disease.
