GemVax Announces Results of GV1001 Imaging Biomarker Study in PSP Animal Model

On July 31, GemVax & KAEL announced that it has once again demonstrated the potential of GV1001, currently under development as a treatment for progressive supranuclear palsy (PSP), to modulate neuroinflammation and tau pathology through positron emission tomography (PET) imaging.

GemVax disclosed the results of its study titled "Imaging Biomarker Study of GV1001 in a PSP Animal Model" at the Alzheimer's Association International Conference (AAIC 2025), the world's largest academic conference on dementia and neurodegenerative diseases, held in Toronto, Canada, from July 27 to July 31.

The study, conducted by Professor Matthias Brendel's team at Ludwig Maximilian University Hospital in Munich, investigated the modulatory effects of GV1001 on neuroinflammation and tau pathology in a PSP mouse model. The effects were assessed by PET imaging, focusing on the activation of microglia and astrocytes following administration of GV1001.

PET is a nuclear medicine imaging technique that visualizes specific metabolic activities in vivo and is mainly used for diagnosing cancer, brain diseases, and heart diseases. Biomarkers measured and detected using imaging technology can be monitored in a non-invasive and real-time manner.

Professor Brendel's team used microglial activation marker (TSPO), astrocyte activation marker (MAO-B), and tau marker as PET imaging biomarkers. They conducted longitudinal follow-up according to disease progression. The results showed that GV1001 could reduce neuroinflammation and tau pathology in tauopathy mouse models.

Professor Matthias Brendel, who led the study at LMU Hospital, stated, "Astrocyte activation was extensively observed in the brains of the control group, but in the GV1001-treated group, it was limited to localized regions within the brain." He added, "Microglial activation was also reduced by GV1001 administration, with notable differences observed particularly in the hippocampus and cortex."

Gunther U. Hoglinger, a world-renowned expert in the PSP field and scientific advisor to GemVax, as well as the head of the Department of Neurology at Munich LMU Hospital, commented, "The observed reduction in TSPO and MAO-B binding after GV1001 administration suggests suppression of tau pathology and neuroinflammation, indicating its potential as a disease-modifying biomarker."

He further evaluated, "The signal reduction in the brainstem, the initial pathological site of PSP, not only supports the mechanistic validity of GV1001 but also raises expectations for the success of future PSP clinical trials."

A GemVax representative stated, "The significance of this study lies in once again demonstrating the modulatory potential of GV1001 on neuroinflammation and tau pathology by longitudinal monitoring using PET."

GemVax has completed the first domestic Phase 2 clinical trial for PSP and is preparing for a global Phase 3 clinical trial based on these results. For Alzheimer's disease, all dosing in the global Phase 2 clinical trial has been completed and data analysis is underway. Recently, the company announced preclinical results for amyotrophic lateral sclerosis (ALS) and continues research to expand indications for neurodegenerative diseases.