VectorY Therapeutics, a leader in vectorized antibody therapies for neurodegenerative diseases, announced 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 ½ 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.

Source: Business Wire