Boston — VectorY Therapeutics announced the expansion of its ongoing global Phase 1/2 PIONEER-ALS clinical trial evaluating VTx-002 in people with amyotrophic lateral sclerosis from 12 to 36 participants.
The study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamic effects of VTx-002, an investigational vectorized antibody therapy designed to target pathological TDP-43.
VectorY said the decision to triple enrollment was made before receiving data related to patient efficacy and was supported by rapid enrollment and global site activation.
The company said the larger study population will allow it to collect a broader set of clinical data and provide greater opportunity to evaluate secondary and exploratory endpoints related to ALS progression.
VectorY announced the first U.S. participants were dosed in February 2026 at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital. The first European participants were dosed in September at University Medical Center Utrecht.
“Expanding PIONEER-ALS from 12 to 36 participants is a direct reflection of the significant interest from people living with ALS, and the rapid pace of global site activation and study enrollment we’ve seen thus far,” said Jim Scibetta, Chief Executive Officer of VectorY.
“There remains a critical need for disease-modifying therapies in ALS,” Scibetta said. “With VTx-002, we directly target TDP-43, which is the pathological driver in the vast majority of ALS cases.”
VTx-002 is VectorY’s lead program and is designed to target pathological TDP-43, a protein that the company said aggregates abnormally in up to 97% of ALS patients.
The therapy uses a vector-based delivery platform intended to enable sustained antibody expression within the central nervous system.
VectorY said VTx-002 has the potential to address the underlying biology of ALS and modify disease progression. The therapy remains investigational and has not been approved by any regulatory authority.
