GemVax & KAEL Co. Ltd. announced topline results from its Phase 2a clinical trial of GV1001, an investigational peptide drug for progressive supranuclear palsy (PSP), at the Neuro2024 International Research Symposium in Toronto. The study suggests GV1001 could be a potential treatment for PSP, a neurodegenerative disease with no current cure. The results support moving to a Phase 3 trial.
The Phase 2a trial was a 24-week, randomized, double-blind, placebo-controlled study conducted across five centers in Korea, involving 78 PSP patients. Participants were randomized to receive either placebo, GV1001 0.56 mg, or GV1001 1.12 mg, administered subcutaneously. The primary endpoint was the change from baseline in the PSP-Rating Scale total score after 24 weeks.
Primary Endpoint Analysis
The primary endpoint analysis showed a deterioration of 2.14 points in the GV1001 0.56 mg dose group compared to 4.10 points in the placebo group, representing a 48% reduction in disease progression. While this result did not reach statistical significance, it indicates the potential of GV1001 as a PSP treatment.
Subgroup Analysis in PSP-RS Patients
Clinically, PSP-RS is the most common and aggressive form of PSP. A subgroup analysis focused on PSP-RS patients revealed a more pronounced effect. The GV1001 0.56 mg dose group showed a deterioration of only 0.25 points compared to a 5.19-point deterioration in the placebo group, demonstrating a 4.94-point difference, or a 95% reduction in disease progression. Furthermore, 58.33% of PSP-RS patients in the 0.56 mg GV1001 group experienced symptom stabilization or improvement.
Safety and Tolerability
The safety profile of GV1001 in the Phase 2a trial was consistent with previous data, with the drug being generally well-tolerated and no serious adverse events reported.
Expert Commentary
Hyungsik Moon, CSO of GemVax, stated that the Phase 2a trial helped determine the optimal dosage and understand how the peptide works on different subgroups. Peter Schüler, MD, Senior Vice President of Drug Development at ICON, noted that the observed trends are plausible and consistent in motor performance and cognitive function, favoring the lower dose group. Dr. Günter U. Höglinger, Head of the Department of Neurology, LMU Hospital, Munich, expressed excitement about participating in the Phase 3 study.
About GV1001
GV1001 is a synthetic peptide consisting of 16 amino acids based on the key sequence of telomerase. It has shown potential in modulating glial cell phenotypes and regulating neuroinflammation. A Phase 2 Alzheimer’s disease clinical trial of GV1001 is ongoing in the U.S. and Europe.
