Kolon TissueGene (950160) Corporate presentation summary

Product overview and mechanism

• TG-C is a cell and gene therapy targeting osteoarthritis, utilizing a combination of chondrocytes and genetically modified 293 cells to deliver TGF-B1 and IL-10, aiming to reduce inflammation and improve joint structure and function.

• The therapy interrupts the inflammatory cycle in joints, promoting M2 macrophage differentiation and structural improvement.

Clinical development and regulatory progress

• Clinical hold was imposed by the FDA in 2019 due to concerns over the origin of 293 cells, but was lifted in April 2020 after extensive safety data and cell characterization were provided.

• Phase 3 trials in the US resumed, with 1,066 patients enrolled after screening over 6,800 candidates across 80+ sites.

• Two pivotal phase 3 studies (TG-C 15302 and TG-C 12301) are ongoing, with data readouts expected in 2026 and BLA submission planned after follow-up and analysis.

Safety and monitoring

• An Independent Data Monitoring Committee (IDMC) has overseen the trial for over two years, ensuring ongoing safety and scientific validity.

• FDA has confirmed no outstanding safety concerns regarding the 2nd component cell line based on current data.