Kolon TissueGene announced in a regulatory filing on the 20th that its cell and gene therapy for osteoarthritis, 'TG-C,' failed to meet the co-primary efficacy endpoint in topline data from its U.S. FDA Phase 3 clinical trial (TG-G15302 Study). However, a positive safety profile was confirmed in the 104-week safety analysis.

Clinical Trial Overview This trial (NCT03291470) was conducted in patients with Kellgren-Lawrence Grade 2 to 3 knee osteoarthritis. It was carried out across 27 U.S. institutions using a randomized, double-blind, placebo-controlled design, with a target enrollment of 510 patients (actual enrolled patients: 531). The first patient was enrolled in December 2021, and the last patient follow-up is scheduled to conclude in May 2026.

• *Efficacy Endpoint Not Met**
• According to the filing, TG-C failed to demonstrate statistically significant improvement over placebo at the 12-month time point for both co-primary efficacy endpoints: change in VAS pain score and WOMAC total score.
• VAS Pain Score: TG-C group -38.7 / Placebo group -39.2 (difference between groups: 0.5, 95% confidence interval -4.3 to 5.3, p=0.8322)
• WOMAC Total Score: TG-C group -27.61 / Placebo group -26.54 (difference between groups: -1.07, 95% confidence interval -4.75 to 2.62, p=0.5701)

Safety Evaluation and Future Plans In contrast, the safety evaluation showed that TG-C was overall safe and well tolerated, with no new safety signals identified. The incidence of treatment-emergent adverse events was 83.9% in the TG-C group and 78.1% in the placebo group, while serious adverse events were 10.3% and 8.6%, respectively, showing similar safety profiles between the two groups. The overall incidence of total knee arthroplasty was 0.6% in the TG-C group and 5.3% in the placebo group, appearing higher in the placebo group.

The company plans to comprehensively analyze the results of this trial along with the efficacy and safety results of another ongoing U.S. Phase 3 clinical trial (NCT03203330) to use as reference material for future consultations with the U.S. FDA.

Investment Considerations The company noted that the statistical probability of a clinical trial drug receiving final approval as a medicinal product is approximately 10%. If the results during the clinical trial and product approval process fall short of expectations, there is a possibility that commercialization plans may be altered or discontinued. Therefore, investors should comprehensively consider the risk factors detailed in regulatory filings and business reports and exercise caution in their investment decisions.