VectorY Therapeutics Receives FDA Clearance of IND to Proceed with the PIONEER-ALS Phase 1/2 Trial of VTx-002, a First-in-Class Vectorized Antibody Targeting TDP-43 Pathology in ALS
December 3, 2025 – Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical – FDA clearance, VectorY Therapeutics, amyotrophic lateral sclerosis, clinical trial, neurodegenerative disease, vectorised antibody therapies
3 December 2025 — Amsterdam, The Netherlands and Massachusetts, US — VectorY Therapeutics, a leader in vectorized antibody therapies for neurodegenerative diseases, announced today that the U.S. Food and Drug Administration (FDA) has cleared the company to proceed with the PIONEER-ALS Phase 1/2 Trial of VTx-002. VTx-002 is a first-in-class vectorized antibody targeting TDP-43 pathology in amyotrophic lateral sclerosis (ALS).
TDP-43 pathology drives up to 97 percent of ALS cases. VTx-002, VectorY’s lead vectorized antibody program, delivers an engineered antibody designed to selectively target toxic species of TDP-43. By binding only to pathological forms, the therapeutic aims to reduce TDP-43 aggregation, correct mis-splicing abnormalities, and restore normal nuclear function. The VTx-002 antibody is produced continuously within target cells following a single intracisterna magna (ICM) administration of its genetic payload, delivered via an AAV5.2 capsid, a vector with a well-established clinical safety profile.
“The FDA’s clearance to proceed with our Phase 1/2 study marks a pivotal milestone for VectorY, as we strive to transform the neurodegenerative disease landscape with our novel vectorized antibodies that are specifically designed to address the well-established biology driving disease manifestations” said Jim Scibetta, chief executive officer. “We are collaborating with PIONEER-ALS Global Coordinating Investigator James Berry, MD, PhD, chief of the Division of Motor Neuron Diseases and director of the Neurological Clinical Research Institute (NCRI) at Mass General Brigham, and with the ALS patient advocate and physician community, as we actively work towards trial initiation.”
The PIONEER-ALS Phase 1/2 Trial of VTx-002 in ALS
PIONEER-ALS, an open label, dose-escalation Phase 1/2 clinical study, will evaluate two dose levels of VTx-002 and is expected to enroll 12 adults with ALS. The primary objective of this study is to evaluate VTx-002 safety and tolerability. 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 VectorY Therapeutics
VectorY Therapeutics is dedicated to changing the trajectory of neurodegenerative diseases by developing precision-targeted, durable therapies that have the potential to arrest and ultimately reverse disease progression. With deep expertise in neurobiology, antibody engineering, and AAV-based delivery, VectorY is building a pipeline that includes VTx-002 targeting TDP-43 in ALS, VTx-001 targeting oxidized phospholipids in ALS, VTx-003 targeting mutant HTT and TDP-43 in Huntington’s disease, VTx-005 targeting phosphorylated tau in Alzheimer’s disease, and VTx-004 targeting TDP-43 in frontotemporal dementia (FTD). Visit: www.vectorytx.com.
