Kolon TissueGene’s stock has dropped after a Phase III trial of its knee osteoarthritis gene-modified cell therapy failed to meet any of its endpoints.
In the Phase III ACTiVION-II study (NCT03291470), 532 patients from 27 US sites were treated with either a single intraarticular injection of TG-C or placebo. Those enrolled had Kellgren–Lawrence (KL) Grade 2 or 3 osteoarthritis of the knee.
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TG-C did not demonstrate statistically significant improvements versus placebo in pain and function, and did not meet the co-primary endpoints of change from baseline in VAS (Visual Analogue Scale) pain and WOMAC (Western Ontario and McMaster Universities Osteoarthritis Index) total score after 12 months of treatment. Additionally, the study did not meet any of the key secondary endpoints.
TG-C was generally safe and well-tolerated with no new or unexpected safety signals identified, with the majority of events reported as Grade 1 or Grade 2 in severity. The observed safety findings were consistent with the known safety profile of TG-C and did not indicate any clinically meaningful safety concerns.
After the failure was announced, Kolon TissueGene’s stock, listed on the KOSDAW exchange in South Korea, dropped 29.9% from Won61,200 at close on 20 July to Won42,900 at market close on 21 July.
Dr Moon Jong Noh, co-CEO of Kolon TissueGene, said: “While we are disappointed by the results from the first of two Phase III clinical trials of TG-C, we remain committed to advancing cell and gene therapy innovation targeting osteoarthritis of the knee.”
Topline results from ACTiVION-I, the other Phase III clinical trial of TG-C (NCT03203330) targeting osteoarthrosis in the knee, are anticipated in October 2026, after which Kolon TissueGene will determine next steps for the candidate.
TG-C is an allogeneic cell therapy involving human cartilage cells. The cells are genetically modified to produce the therapeutic growth factor transforming growth factor beta 1 (TGF-beta1).
Currently, care for knee osteoarthritis includes low-impact exercise (like walking or cycling), targeted muscle strengthening, and weight management. If pain persists, treatments include over-the-counter NSAIDs, such as ibuprofen or topical Voltaren gel, and corticosteroid injections for acute flare-ups. If the damage to the joint is severe, patients may undergo a knee replacement.
According to GlobalData analysis, the osteoarthritis market across the seven major markets (7MM: US, France, Germany, Italy, Spain, UK, and Japan) is set to be worth $3.8bn in 2031, up from $2.4bn in 2021.
GlobalData is the parent company of Clinical Trials Arena.
