Kolon’s Cell-Based Gene Therapy Fails Phase 3 Osteoarthritis Trial

Key Points

• Kolon TissueGene’s phase 3 trial of TG-C in knee osteoarthritis missed its co-primary endpoints and all key secondary endpoints.
• The study enrolled 531 patients across 27 U.S. sites and compared intra-articular TG-C injections against placebo.
• Kolon plans to decide the future of the program after data from a second phase 3 trial expected in October.

Kolon TissueGene, a Korean-based biotech company, announced that its cell-based gene therapy, TG-C, failed to meet the main goals of a phase 3 trial in osteoarthritis of the knee. After one year, patients who received TG-C showed no statistically significant improvement in knee pain or function versus placebo. The trial also missed all key secondary endpoints.

For context, TG-C contains human allogeneic (donor derived) chondrocytes and an irradiated human embryonic kidney-derived cell line stably expressing TGF-β1. South Korean regulators approved the therapy in 2017, but withdrew that approval two years later after a mix-up over the type of cells used in the treatment.

Trial design and what comes next

Kolon enrolled 531 patients at 27 sites in the U.S. The trial compared intra-articular injections of TG-C with placebo. Enrollment began four years after the company registered the study, following an FDA hold.

Attention now shifts to Kolon’s second phase 3 trial, which is expected to report data in October this year. That study has the same treatment arms and co-primary endpoints as the failed trial, along with nearly identical inclusion and exclusion criteria. Kolon said it will decide the next steps for the program after reviewing those results.

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