GemVax & KAEL’s GV1001 fails to show effects in P2a trial of progressive supranuclear palsy

GemVax & KAEL's telomerase-based peptide drug GV1001 failed to demonstrate significant efficacy in a phase 2a clinical trial in Korea in patients with progressive supranuclear palsy (PSP).

On Thursday, GemVax & KAEL disclosed topline data from the Korean phase 2a trial of GV1001 in people with PSP.

The trial was conducted in a multicenter, randomized, double-blind, placebo-controlled, parallel design at five hospitals in Korea, including SMG-SNU Boramae Medical Center. Its primary objective was to explore the efficacy and safety of GV1001 in improving severity in PSP patients.

The phase 2a trial, which began after its investigational new drug (IND) plan won approval from the Ministry of Food and Drug Safety (MFSD) in March 2023, has been conducted until Oct. 2, when the last subject visit was completed. The trial targeted 75 subjects but ultimately recruited 78. Of those, 70 were included as complete analysis subjects (FAS), according to the company.

Two doses of GV1001 were administered subcutaneously (0.56 mg/day and 1.12 mg/day). The primary endpoint was the change in the PSP-rating scale total score after 24 weeks of treatment with the investigational product compared to baseline.

Results showed that the mean change in PSP-rating scale total score (LS Mean) from baseline to 24 weeks post-treatment for each treatment arm was +2.14 points in the GV1001 0.56 mg arm, +6.46 points in the GV1001 1.12 mg arm, and +4.10 points in the control arm.

The difference in PSP-rating scale total score change between treatment groups was -1.96 points between GV1001 0.56 mg and control and +2.36 points between GV1001 1.12 mg and control, with no statistically significant differences observed (GV1001 0.56 mg p=0.4254, GV1001 1.12 mg p=0.3184).

GemVax & KAEL plans to receive a complete study report (CSR) in the first half of 2025. The report will include primary and secondary efficacy analyses, subgroup analyses, and biomarker analyses.

In a separate release on the same day, GemVax & KAEL argued that the LS mean, calculated using the MMRM estimator, showed a 2.14-point worsening in the GV1001 0.56 mg arm over six months compared to a 4.10-point worsening in the placebo arm, resulting in a 48 percent delay in disease progression in the GV1001 arm.

“The phase 2a study was designed to explore optimal dosing and did not show statistical significance, it confirms the high potential of GV1001 as a first-in-class treatment for PSP. However, considering that there are no treatments now and past clinical studies for PSP drug development have not even shown a trend, the domestic trial has demonstrated a high possibility of the first global treatment,” a company official said. “It also enabled us to secure successful clinical design data for a global phase 3 trial for commercialization.”