First ALS patient dosed in study of experimental gene therapy VTx-002
Drug targets toxic protein aggregates believed to drive nerve cell damage
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- VTx-002 is an experimental gene therapy for ALS that has FDA fast-track designation.
- It targets toxic TDP-43 protein aggregates, a key driver of ALS progression.
- The therapy is in a Phase 1/2 clinical trial, with the first patient recently dosed.
The first patient has been dosed in a Phase 1/2 clinical study of VTx-002, an experimental therapy being developed by VectorY Therapeutics for amyotrophic lateral sclerosis (ALS).
The open-label PIONEER-ALS (NCT07287397) is enrolling up to 12 adults with ALS at sites in the U.S. and Europe. They will be randomly assigned to receive one of two doses of VTx-002 via injection into the cisterna magna, a large fluid-filled space at the base of the brain.
“The initiation of dosing in the PIONEER-ALS trial is a significant milestone for VectorY as we strive to transform the neurodegenerative disease landscape with novel disease-modifying approaches,” Olga Uspenskaya-Cadoz, MD, PhD, chief medical officer of VectorY, said in a company press release.
Developer expects VTx-002 to provide years of antibody protection
ALS is characterized by the progressive loss of motor neurons, the nerve cells that control movement. Toxic TDP-43 protein aggregates are present in the vast majority of ALS patients and are believed to be involved in nerve cell damage.
The goal with VTx-002 is to enable nerve cells to produce antibodies that target toxic TDP-43 aggregates, marking them for clearance by the body’s immune system. It uses an engineered adeno-associated virus to deliver the genetic information for the antibody directly to nerve cells in the brain and spinal cord. The therapy is given in a single administration, which the company expects to ensure years of antibody production.
“VTx-002 is engineered to enable sustained, localized delivery of an antibody targeting pathological TDP-43, a hallmark of ALS, with the goal of addressing a central driver of disease progression,” said James Berry, MD, director of the Neurological Clinical Research Institute at Mass General Brigham and the study’s global coordinating investigator.
PIONEER-ALS will evaluate VTx-002’s safety, tolerability, pharmacokinetics (how it moves into, through, and out of the body), and preliminary efficacy. Efficacy measures include changes in the levels of neurofilament light chain, a marker of neurodegeneration, and novel TDP-43 pathway-related biomarkers.
“This trial marks the [first-ever] clinical evaluation of a therapy designed to holistically target TDP-43 pathology in ALS, and thereby reduce TDP-43 aggregation,” Uspenskaya-Cadoz said. “We are excited to advance this novel potential therapeutic strategy to a community of patients who are actively looking for hope.”
This trial marks the [first-ever] clinical evaluation of a therapy designed to holistically target TDP-43 pathology in ALS, and thereby reduce TDP-43 aggregation.
The trial will also assess changes in clinical measures, including respiratory function, muscle strength, disease severity, health-related quality of life, and survival.
“The initiation of this study reflects an innovative approach aimed at one of the most important pathological features of the disease. I look forward to leading this exploration of VTx-002 and its potential to impact people living with ALS,” Berry added.
The treatment was recently granted fast-track designation by the U.S. Food and Drug Administration for ALS. The status is meant to accelerate the development and review of therapies for serious conditions with a significant unmet medical need by providing more frequent interactions with the FDA and potentially making VTx-002 eligible for priority review or accelerated approval.
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