[Bio Talk] Could Korea Pioneer the World’s First Fundamental Osteoarthritis Therapy?

Kolon TissueGene to Announce Topline Results of U.S. Phase 3 This Month

Three Korean Companies Compete with Distinct Strategies

Long-Term Follow-Up Essential for Validation... Market Patience Key to Success

Can the "Invossa" osteoarthritis treatment, which sent shockwaves through Korea's bio industry after its approval was revoked seven years ago, finally make a comeback? The story centers on Kolon TissueGene's osteoarthritis therapy "TG-C" (formerly Invossa). Kolon TissueGene is scheduled to release the topline results of its U.S. phase 3 clinical trials for TG-C at the end of this month. Two trials were conducted, enrolling a total of 1,066 patients.

This announcement is drawing attention because it will serve as a key indicator of how close Kolon TissueGene has come to developing an unprecedented disease-modifying osteoarthritis drug (DMOAD). Unlike existing treatments that only alleviate pain, a DMOAD is considered a "fundamental therapy" because it is expected to regenerate damaged cartilage. No global pharmaceutical or biotech company, including major multinational corporations, has managed to develop such a drug to date.

In the past 15 years, all 11 DMOAD candidates that advanced to phase 2 or 3 clinical trials have failed. This is because no drug has succeeded in demonstrating both pain relief and cartilage regeneration. Sprifermin by Merck KGaA managed to reduce cartilage loss, but failed to improve pain. Tanezumab, developed by Pfizer and Eli Lilly, improved pain but was found to cause side effects such as osteonecrosis of the knee and rapid disease progression.

Kolon TissueGene is tackling this challenge with its gene-cell therapy TG-C, which injects growth factors that induce cartilage production via cells into the joint cavity. The topline result set to be released will focus on the primary endpoint: improvements in pain and knee function. Whether or not cartilage is regenerated will be determined by changes observed in magnetic resonance imaging (MRI), which is the secondary endpoint.

If the primary endpoint is not met, the therapy will not be approved. Even if it is, that will not be the end of the road. As seen with tanezumab, success in pain improvement can still be derailed by side effects; moreover, in order to be recognized as a DMOAD, the therapy must also satisfy the secondary endpoint. Most candidates that failed before did not overcome this critical hurdle. That moment of suspense is now very near.

Including Kolon TissueGene, there are three companies in Korea attempting to compete in this field. In May, Medipost met both the primary and secondary efficacy endpoints for pain relief and cartilage regeneration in a Japanese phase 3 trial with its product Cartistem, which uses umbilical cord-derived mesenchymal stem cells applied surgically to the injured cartilage area. IPECS is currently conducting early clinical trials of "MIUChon," which involves injecting chondrocytes differentiated from induced pluripotent stem cells (iPSC). After publishing research on the therapy’s mechanism in the prestigious international journal "Science Advances," IPECS recently completed administration to a third patient.

The fact that these three Korean companies are pursuing the world's first DMOAD using different approaches, validating their results step by step, is in itself a testament to the progress and capability of Korea’s bio industry. Depending on the outcome, the topline results that Kolon TissueGene will announce could become another significant milestone.

The development of DMOADs is a long-term battle. For example, it will take at least two years just for Kolon TissueGene’s secondary endpoint results and Medipost’s U.S. phase 3 outcomes to be revealed in sequence. While pain can be assessed within weeks, cartilage structure changes require years of observation and follow-up. The critical factor is financial endurance: during the R&D process, a company’s ability to secure long-term funding is its basic strength. If short-term successes or failures cause instability in share prices and funding channels, it can jeopardize the completion of clinical trials themselves. The market’s patience to see through the accumulation of clinical data to the end is therefore essential.

This content was produced with the assistance of AI translation services.