On August 18, GemVax & KAEL announced that it had confirmed statistically significant effects in an interim analysis of its extension clinical trial targeting patients with progressive supranuclear palsy (PSP).
Symptoms were maintained or improved in certain subdomain assessment indicators, such as mentation (cognitive function) and ocular motor function. In biomarker analyses conducted in previous clinical trials, a consistent trend was observed in major biomarkers relevant to neurodegenerative diseases.
The company analyzed the results of GV1001 administration at week 48, including six months of data from the preceding clinical trial and the extension study. GemVax is conducting the first domestic PSP clinical trial, comprising a six-month initial phase followed by a 12-month extension phase.
According to the interim analysis, for the low-dose group (0.56mg) of patients with progressive supranuclear palsy Richardson syndrome (PSP-RS), the change in the total PSP rating scale score at week 48, calculated as the least squares mean (LS mean) using the MMRM estimation method, showed a deterioration of only 4.37 points. The average change analyzed by T-test also indicated a deterioration of 2.89 points.
A clear difference was observed when compared to external control groups. In four representative PSP clinical trials previously conducted, the average change in the PSP rating scale total score for placebo-group PSP-RS patients worsened by 9.90 to 10.90 points. When compared with the score changes in the GV1001 low-dose group, each showed statistically significant differences. When pooling the results from the four studies and comparing them to the GV1001 low-dose group, the p-value was less than 0.0001.
In the low-dose group, the responder rate-defined as the proportion of patients whose PSP rating scale score was maintained or improved at week 48 compared to baseline (prior to first administration)-was 56%.
At week 48, meaningful changes were also observed in certain subdomain assessment indicators, including cognitive function and ocular motor function. According to the T-test, cognitive function actually improved by 0.22 points compared to baseline. Ocular motor function showed no difference from baseline (0.00 points). No adverse drug reactions were observed in this analysis, confirming the safety of the drug.
Alongside the interim analysis of the extension clinical trial, GemVax also disclosed the biomarker analysis results from the previous trial, which did not contradict the PSP rating scale analysis results.
In the low-dose group, a trend toward reduced plasma deterioration was observed for NfL (a marker of neural damage) and GFAP (a marker indicating astrocyte damage or activation), both of which are major biomarkers indicating the degree of neural damage in neurodegenerative diseases.
GemVax plans to proceed with a global phase 3 clinical trial, incorporating both the fully analyzed clinical trial data and biomarker results. The global phase 3 trial will benefit from the U.S. Food and Drug Administration (FDA) designations of orphan drug and fast track, as well as the European Medicines Agency (EMA) orphan drug designation.
A GemVax official stated, "It is highly encouraging that the efficacy of GV1001 has been sustained with long-term administration and that the safety of the drug has been confirmed. In particular, the difference shown by GV1001 in comparison with the external control group is highly significant." The official added, "We will thoroughly implement the global phase 3 clinical trial, fully reflecting the additional clinical trial data and biomarker analysis results."
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