GemVax Publishes Paper Showing GV1001 Reduces Tau Pathology and Neuroinflammation Markers

GemVax & KAEL has published a research paper in an international academic journal supporting the mechanism of action of its progressive supranuclear palsy (PSP) treatment candidate, GV1001.

On March 18, GemVax announced that a paper providing scientific evidence for the clinical results of PSP was published in the international journal "Scientific Reports." This journal is published by Nature Portfolio.

The paper, titled "GV1001 reduces pathological 4R tau and functional deficits in models relevant to progressive supranuclear palsy," was a collaborative effort between GemVax's Research Institute and leading research institutions in Korea.

Tauopathy refers to a group of neurodegenerative diseases characterized by abnormal aggregation of tau proteins. In particular, PSP is known to be primarily caused by the abnormal accumulation of 4R (4-repeat) tau. The researchers explained that GV1001 may slow the progression of PSP by regulating the overexpression of 4R tau protein.

According to the paper, in animal experiments, behavioral changes were observed indicating overall improvement in motor skills and cognitive functions after administration of GV1001. In cellular experiments, the level of 4R tau protein was regulated to near-normal levels.

Additionally, in cellular experiments, it was found that GV1001 also affected the reduction of TSPO, a marker of microglial activation. TSPO is a marker associated with neuroinflammation and glial cell activation, and research continues on its use as an imaging biomarker for PSP diagnosis.

In this regard, GemVax previously confirmed through an animal model study of PSP conducted in collaboration with Professor Matthias Brendel's research team at Ludwig Maximilian University (LMU) Hospital in Munich, Germany, that GV1001 can reduce neuroinflammation and tau pathology, including TSPO and other indicators. This research was presented at the Alzheimer's Association International Conference (AAIC 2025).

A company representative stated, "This paper is a meaningful achievement that proves the action of GV1001 in PSP," adding, "It provides a scientific foundation for interpreting clinical trial results in connection with pathological protein changes, functional markers, and cellular-based evidence."

Meanwhile, GemVax has received Fast Track and Orphan Drug Designation for GV1001 from the US Food and Drug Administration (FDA), as well as Orphan Drug Designation from the European Medicines Agency (EMA). The company is scheduled to receive the 72-week results of its domestic Phase 2 clinical trial next month and plans to pursue a global Phase 3 clinical trial.

This content was produced with the assistance of AI translation services.