GemBio announced research findings on the 72-week safety and efficacy of GV1001 for progressive supranuclear palsy (PSP) patients at the 2026 International Congress of Parkinson’s Disease and Movement Disorders (MDS 2026), thebell reported.
Clinical Results for GV1001 at MDS 2026
GemBio stated that the low-dose GV1001 group showed a trend toward improved neurological severity and disease progression inhibition compared to the placebo control group in the study. The findings were presented on October 6 at MDS 2026, which runs from October 4 through October 8 at COEX in Seoul, hosted in collaboration with the Korean Parkinson’s Disease and Movement Disorder Society.
The research evaluates safety and efficacy up to 72 weeks of GV1001 administration using domestic PSP Phase 2a clinical trials and open-label extension studies. The study was selected as an MDS 2026 abstract and featured in an oral presentation.
Low-Dose GV1001 Suppresses Disease Progression
Researchers examined changes in the PSP Rating Scale (PSPRS). Patients who received low-dose GV1001 in earlier clinical phases showed improved trajectories compared to patients who initially received a placebo before transitioning to GV1001 in the open-label extension. By the final 72-week evaluation, the neurological severity gap between the two patient groups remained statistically significant.
Subgroup analyses accounting for individual patient variables—including age, gender, and baseline disease severity scores—demonstrated consistent disease progression suppression. Investigators also presented findings on neurofilament light chain (NfL), a blood-based biomarker linked to nerve damage. Company officials noted that patients with relatively low baseline blood NfL levels experienced a pronounced slowing of disease progression following treatment.
GV1001 Shows Favorable Safety Results
Regarding safety, GV1001 maintained favorable results despite the advanced age and neurological severity of the study participants. Throughout the 72-week dosing period, researchers observed no serious adverse drug reactions, nor any adverse events leading to permanent treatment discontinuation or death.
A GemBio representative said the company continues to conduct diverse in-depth analyses to raise the success probability of subsequent PSP clinical trials. The company plans to establish refined development strategies for future large-scale confirmatory trials to clearly verify the efficacy and safety of GV1001.
Worth a look
