April 28, 2026

The authorizations represent an expansion of the PIONEER-ALS clinical trial,
currently enrolling participants at clinical trial sites in the U.S.

VectorY Therapeutics, a leader in vectorized antibody therapies for neurodegenerative diseases, announced the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA), and the European Medicines Agency (EMA) have both granted the company clearance to initiate a first-in-human clinical trial of its investigational first-in-class vectorized antibody targeting TDP-43 pathology in people living with amyotrophic lateral sclerosis (ALS).

In the EU, the approval covers clinical trial sites in Belgium and The Netherlands, which will serve as primary European centers for the global study. The company recently announced that it has treated its first participant at its lead clinical trial site in the United States.

“Authorization from the MHRA and the EMA to initiate our Phase 1/2 clinical trial enables us to expand the clinical evaluation of our therapy globally and collaborate with leading investigators across numerous regions as we advance this potential new treatment for people living with ALS,” said Jim Scibetta, chief executive officer of VectorY. “These milestones build on the progress we’ve made in the U.S. where the study is already underway, and we’re excited to be able to offer clinical trial participation to people living with ALS in the UK and the EU.”

ALS is a rapidly progressive and fatal neurodegenerative disorder that affects motor neurons in the brain and spinal cord, leading to loss of muscle control, paralysis, and respiratory failure. Median survival is 3 to 5 years after a diagnosis. Despite advances in understanding the disease, there remains a significant unmet medical need for treatments that can meaningfully slow or halt its progression.

PIONEER-ALS is a multicenter, open label, dose-escalation Phase 1/2 clinical study that evaluates two dose levels of VTx-002 and is expected to enroll 12 adults with ALS across sites in the U.S., Europe and the U.K. The primary objective of this study is to evaluate VTx-002 safety, tolerability, pharmacokinetics, and exploratory efficacy in people living with ALS. To optimize the path to pivotal clinical development, secondary and exploratory endpoints include assessment of the post-treatment Neurofilament light chain (NfL) and novel TDP-43 pathway-related biomarker trajectories, as well as clinical endpoints, including ALSFRS-R, slow-vital capacity, hand-held dynamometry and survival.

About VTx-002

VTx-002 is a first-in-class vectorized antibody therapeutic designed to target pathological TDP-43, a protein that aggregates abnormally in up to 97 percent of ALS patients. By leveraging a vector-based delivery platform, VTx-002 is designed to enable sustained antibody expression within the central nervous system, with the potential to address the underlying biology of ALS.

Source: Press release VectorY Therapeutics

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