BOSTON — VectorY Therapeutics said regulators in Europe and the United Kingdom have authorized the company to begin a first-in-human clinical trial of its experimental ALS therapy, expanding an ongoing study already underway in the United States.
The company said both the U.K. Medicines and Healthcare Products Regulatory Agency and the European Medicines Agency have cleared the Phase 1/2 PIONEER-ALS trial of VTx-002, a vectorized antibody targeting TDP-43 pathology in patients with amyotrophic lateral sclerosis.
The approvals allow the study to move forward at sites in Belgium and the Netherlands, which will serve as primary European hubs for the global trial. The company recently announced it has treated its first participant at a U.S. site.
“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.”
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease that affects motor neurons in the brain and spinal cord, leading to muscle weakness, paralysis, and ultimately respiratory failure. Median survival after diagnosis is typically three to five years, underscoring the need for new treatments.
The PIONEER-ALS study is a multicenter, open-label, dose-escalation trial evaluating two dose levels of VTx-002. It is expected to enroll 12 adults with ALS across sites in the U.S., Europe, and the U.K.
The primary goal is to assess safety, tolerability, and pharmacokinetics, along with early signals of efficacy. Secondary and exploratory measures include biomarkers such as neurofilament light chain and TDP-43-related indicators, as well as clinical outcomes including functional rating scores, respiratory capacity, muscle strength, and survival.
Company officials said the expansion into Europe and the U.K. is intended to accelerate development of the therapy and broaden access to clinical trial participation for patients.
