GemVax Secures PSP Extension Trial CSR... Significant Efficacy Confirmed in Low-Dose Group

GemVax & KAEL (hereafter "GemVax") announced on April 9 that it has received the final Clinical Study Report (CSR) for the domestic Phase 2 extension clinical trial of progressive supranuclear palsy (PSP).

The extension clinical trial results showed that GV1001 demonstrated positive data in terms of drug tolerability and safety, further confirming its safety profile. No serious adverse drug reactions (Serious ADRs) related to the drug were reported, and there were no ADRs leading to clinical trial discontinuation or death.

In the overall evaluation of deaths, serious adverse events (SAEs), and other significant adverse events, no new safety issues were identified. Laboratory tests—including hematological, blood chemistry, and urinalysis—also showed no clinically meaningful differences in changes between treatment groups or deviations from reference values.

This CSR includes not only the safety data from the extension clinical trial, but also the combined results of both the preceding and extension clinical trials over a total period of 72 weeks. It also contains comparative analyses with external control groups and significant findings in certain subdomains.

The PSP clinical trials were conducted over a total period of 72 weeks, consisting of a preceding clinical trial of 24 weeks and an extension clinical trial of 48 weeks. The preceding trial was divided into three groups: placebo, GV1001 0.56mg (low dose), and GV1001 1.12mg (high dose). The extension trial was conducted by administering GV1001 1.12mg to patients from the preceding trial who wished to continue treatment.

According to GemVax, when the change in the total PSP rating scale score for the PSP-Richardson's syndrome (PSP-RS) patient group was compared with an external control group in the 72-week clinical trial, statistical significance was confirmed in the low-dose group of the preceding trial.

The external control group was based on placebo group data from three major overseas PSP clinical trial publications, and the MAIC (Matching Adjusted Indirect Comparison) method was applied to enhance the objectivity of the comparison.

While the external placebo groups were analyzed based on 52-week outcomes, GV1001 was evaluated over a 72-week period. As a result, the external control group worsened by 10.66 points, whereas the low-dose group from the preceding trial worsened by only 3.31 points over a longer period (72 weeks), demonstrating statistical significance.

Additionally, the analysis of subdomains in the PSP rating scale after 72 weeks was also released. In the mentation domain for the PSP-RS patient group, an LS mean analysis using the MMRM estimation method found a statistically significant difference in favor of the low-dose group compared to the placebo group in the preceding trial.

In the T-test-based mean analysis, the low-dose group also showed meaningful differences compared to the placebo group in the mentation, ocular motor, and limb motor domains.

In particular, higher scores in the mentation domain indicate a worsening of condition. In the PSP-RS low-dose group, the average score improved by decreasing 0.56 points from baseline, confirming a therapeutic effect.

A GemVax representative stated, "Considering that PSP is a disease in which the rating scale score typically worsens by about 11 points per year, the fact that the low-dose group worsened by only 3.31 points over 18 months is an unprecedented and encouraging result. This significantly enhances the potential for GV1001 as a therapeutic agent."

The representative added, "Based on these results, we plan to smoothly proceed with confirmatory clinical trials for commercialization approval targeting global regulatory agencies such as those in the United States and Europe. The CSR will also be provided to investors and partner companies with whom we are currently in discussions."

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