MEDIPOST's CARTISTEM® Meets All Primary and Secondary Endpoints in Japan Phase 3 Trial and Demonstrates Statistically Significant Efficacy Gains Versus Active Control
- Achieved statistical significance versus standard-of-care (SOC) control across all key efficacy endpoints in the 52-week study
- Japan Phase 3 results strengthen CARTISTEM's® global commercial prospects, with Japan biologics license application (BLA) scheduled for 2H 2026
SEOUL, South Korea and TOKYO, May 14, 2026 /PRNewswire/ -- MEDIPOST has met all primary and secondary efficacy endpoints in the Japan Phase 3 clinical trial of CARTISTEM®, demonstrating clinical improvements in pain and function as well as cartilage regeneration. Based on the results, MEDIPOST is expected to accelerate preparations for its biologics license application (BLA) filing in Japan later this year.
MEDIPOST (CEO Wonil Oh), a global leader in stem cell biotherapeutics, announced today that the Japan Phase 3 clinical trial of its knee osteoarthritis treatment CARTISTEM® achieved statistical significance across all primary and secondary efficacy endpoints. The results showed improvements not only in symptoms, including pain and function, but also in cartilage regeneration, marking an important milestone in the company's global market expansion strategy.
The trial was a randomized, controlled study conducted at 13 sites(hospitals) in Japan enrolling 130 participants. Subjects were randomized into the CARTISTEM® treatment group, comprising 59 patients, and the hyaluronic acid (HA) control group, comprising 61 patients. Efficacy and safety were evaluated over a 52-week follow-up period.
The trial adopted a randomized design to evenly distribute baseline demographic and clinical characteristics between the two groups, enhancing the objectivity of the results. The mean age of patients was 61.3 years in the CARTISTEM® treatment group and 62.2 years in the control group. Other baseline clinical parameters, including Kellgren-Lawrence (K&L) Grade for osteoarthritis severity, International Cartilage Repair Society (ICRS) Grade for cartilage defect severity, and baseline pain scores, were well balanced, with no statistically significant difference between the two groups, ensuring the reliability of the clinical findings.
CARTISTEM® demonstrated statistically significant improvements over the active control group on both co-primary endpoints: Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC), a knee pain and function index, and improvements in International Cartilage Repair Society (ICRS) grade against control(WOMAC p <0.0001 and ICRS p =0.0002).
Statistically significant improvements were also confirmed across all secondary endpoints. Notable improvements were observed in key indicators including the Visual Analog Scale (VAS), which measures pain intensity, as well as the International Knee Documentation Committee (IKDC) and Knee Injury and Osteoarthritis Outcome Score (KOOS), both of which assess knee function. The p-values for VAS, IKDC, and KOOS were all less than <0.0001. Marked improvements were likewise demonstrated in the WOMAC subscale indicators for pain, function, and stiffness (p <0.0001).
CARTISTEM® maintained an excellent safety profile throughout the trial. All reported serious adverse events (SAEs) were assessed as having no causal relationship to CARTISTEM®.
In particular, results for WOMAC (p-value <0.0001) and the 100 mm VAS (p-value <0.0001), which are the identical primary endpoints utilized in the ongoing U.S. Phase 3 trial, showed statistically significant superiority over the active control. These findings serve as positive indicators for MEDIPOST's U.S. clinical development efforts.
Antonio Lee, Global President of MEDIPOST and CEO of MEDIPOST K.K. (Japan) & Co-CEO of MEDIPOST Inc. (U.S.), stated, "The Japan Phase 3 results have reaffirmed CARTISTEM®'s strong efficacy and safety profile." He added, "The consistency observed across all co-primary and secondary endpoints demonstrates significant clinical implications. In particular, the cartilage regeneration effect of CARTISTEM® that was demonstrated in the Korea Phase 3 trial has now been reconfirmed in the Japan Phase 3 study. We believe these results will provide a solid foundation for CARTISTEM®'s global market entry as a Disease-Modifying OA Drug (DMOAD)."
Meanwhile, MEDIPOST plans to accelerate its entry into the Japanese market by submitting a marketing authorization application later this year, targeting regulatory approval by end of 2027. The company has already established a commercial partnership through an exclusive distribution agreement with Teikoku Seiyaku Co., Ltd.. Following regulatory approval and commercial launch, MEDIPOST expects to generate tangible business outcomes in Japan through finished product supply revenues and sales-based milestone payments.
SOURCE MEDIPOST
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