VectorY Therapeutics, a biopharmaceutical company, has been cleared by the FDA to proceed with its PIONEER-ALS Phase I/II clinical trial of VTx-002, an antibody targeting toxic forms of TDP-43, a protein strongly linked to the underlying disease process in ALS. Preclinical studies showed that VTx-002 reduced harmful TDP-43 aggregates and restored normal RNA processing, and improved neuronal function. The trial will consist of 12 adult patients with ALS, with two doses of the drug. The primary goal is to assess safety and tolerability, while the secondary and exploratory measures track changes in neurofilament light chain and TDP-43 biomarkers after treatment.
According to VectorY’s CEO Jim Scibetta: “The FDA’s clearance to proceed with our Phase I/II study marks a pivotal milestone for VectorY, as we strive to transform the neurodegenerative disease landscape with our novel vectorised antibodies that are specifically designed to address the well-established biology driving disease manifestations. We are collaborating with PIONEER-ALS Global Coordinating Investigator James Berry, 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.” See the official press release here.