GemVax & KAEL recently announced results from its Phase 2a clinical trial evaluating GV1001, a drug for progressive supranuclear palsy (PSP). While the trial did not achieve statistical significance in its primary endpoint, the data showed enough promise for the company to proceed to Phase 3 trials. The study, presented at the Neuro2024 PSP and CBD International Research Symposium, garnered positive feedback from experts, fueling excitement about the potential of GV1001 as a novel treatment for this debilitating neurodegenerative disorder.
Promising Signals in PSP-RS Subgroup
The Phase 2a trial enrolled participants with PSP-Richardson’s syndrome (PSP-RS) and PSP-Parkinson’s (PSP-P), administering either a 0.56 mg dose, a 1.12 mg dose, or a placebo via weekly injection for one month, followed by bi-weekly intervals for five months. The primary endpoint was the impact of GV1001 on disease progression, measured by changes in the PSP-Rating Scale score. Although all groups experienced disease progression, the 0.56 mg dose group showed a 48% reduction in progression compared to placebo.
Notably, a subgroup analysis revealed a significant difference in outcomes for participants with PSP-RS. This group experienced a mean increase of only 0.25 on the PSP-Rating Scale, representing a 95% decrease in disease progression compared to the 4.10 increase observed in the placebo group. GemVax reported that some PSP-RS participants even experienced symptom stabilization or improvement during the trial.
Safety and Tolerability
In addition to the encouraging efficacy signals, GV1001 demonstrated a favorable safety profile. Researchers reported no serious adverse effects, and the drug was generally well-tolerated by participants, aligning with previously gathered safety data.
Expert Commentary and Future Directions
Hyungsik Moon, CSO of GemVax, stated, "This Phase 2a trial was an exploratory study to determine the optimal dosage and find out how the peptide works on different subgroups. Although the topline result did not achieve statistical significance, the evidence is strong enough to consider moving forward to a pivotal trial and shows potential to develop GV1001 as the world’s first treatment option for PSP."
Peter Schüler, MD, Senior Vice President of Drug Development at ICON, commented on the trial's findings: "This pilot study was not fully powered, and the treatment duration with 6 months was short. Thus, statistically significant confirmatory results could not be expected. Nonetheless, the observed trends are very plausible and consistent in two domains: motor performance and cognitive function, both favoring the lower dose group...[it] demonstrated clinically meaningful benefits, namely full stabilization of the disease compared to the placebo group."
GemVax is now planning a Phase 3 trial to further investigate the potential of GV1001 as a treatment for PSP, particularly in the PSP-RS subtype.
