Kolon TissueGene apologizes for Phase 3 miss, pledges independent CRO for second analysis

Kolon TissueGene has issued a formal apology to shareholders after TG-C, its cell-gene therapy for knee osteoarthritis, failed to meet statistical significance in the first study of its US Phase 3 trial. The company also announced it will hand all analysis of the upcoming second study to an external, internationally accredited contract research organization to strengthen objectivity and transparency.

Co-CEOs Noh Moon-jong and Jeon Seung-ho issued a joint letter to shareholders Monday, saying they "sincerely and humbly apologize for the deep disappointment and concern caused by the announcement of the first results of the TG-C US Phase 3 trial." They said the company would "thoroughly investigate from the ground up why this study failed to demonstrate statistical significance, unlike previous clinical results."

Kolon TissueGene released the top-line results of the first study (15302 Study) of the TG-C US Phase 3 trial on Tuesday. Patients who received TG-C experienced meaningful pain relief (VAS score: -38.6) and improved joint function (WOMAC score: -26.34), and the rate of total knee replacement surgery was 8.8 times lower in the TG-C group than in the placebo group — trends the company said demonstrated clinical relevance. However, the placebo group showed an unexpectedly strong improvement response, and the study ultimately failed to meet the pre-specified threshold for statistical significance against the control group.

The company said it has accepted shareholders' criticism and concerns over its in-house analysis of the first study results and will significantly strengthen the transparency of subsequent procedures. Analysis of results from the ongoing second study (12301 Study) will be conducted independently and rigorously through an external contract research organization (CRO) with globally recognized credibility.

To that end, Kolon TissueGene has appointed Dr. Andy Wayman — an orthopedic surgeon who served as chief medical officer at global healthcare company Smith & Nephew for 14 years — as its new chief medical officer to lead the investigation into the cause of the trial outcome. The company said it will analyze additional clinical data using objective scientific standards and consult with leading external experts to chart a new development direction.

The company also addressed allegations that its share price fell and clinical data may have been leaked before the trial results were announced. While the company said it had maintained a ban on share trading by employees and kept security around its clinical data division at the highest level, it announced plans to establish an independent investigative body — going beyond an internal probe — to resolve the allegations.

"We have walked a single path for nearly 30 years, dedicated solely to developing TG-C, and now face an unexpected challenge — but we will never stop or give up," co-CEOs Noh and Jeon said. "We will not rely on excuses or vague hope, but will communicate with the market and restore trust through scientific data and responsible action alone."

Kolon TissueGene plans to release results from the second US Phase 3 study of TG-C in October. That study was conducted under the same protocol but with an independent institution and a separate patient group.