Kangstem Biotech’s Stem Cell for Knee Osteoarthritis Phase 2a Reports Significant Results
The Korean biotech company reported improved pain, joint function, and MRI-based structural measures versus placebo at 24 weeks, with no drug-related serious adverse events.
Key Points
- OSCA met statistical significance versus placebo across four patient-reported symptom measures at 24 weeks.
- The Phase 2a trial enrolled 113 patients with moderate knee osteoarthritis, with 108 completing follow-up.
- No treatment-related serious adverse events were reported, while MRI analyses showed statistically significant structural findings on selected measures.
Kangstem Biotech has reported topline results from a Phase 2a trial of OSCA, its umbilical cord-derived mesenchymal stem cell therapy, in patients with moderate knee osteoarthritis. The study compared medium and high doses of OSCA with placebo over 24 weeks.
The trial enrolled 113 patients, assigned 38 to the medium-dose group, 38 to the high-dose group, and 37 to placebo. A total of 108 patients completed the 24-week follow-up.
The Phase 2a assessed safety, efficacy, and dose selection for subsequent trials. Safety outcomes did not differ significantly between groups, and no treatment-related serious adverse events were reported.
Symptom and Imaging Results
Both OSCA dose groups showed statistically significant differences from placebo across the following patient-reported measures:
- 100 mm visual analog scale (VAS) for pain: Medium dose, p<0.0001; high dose, p<0.0001
- International Knee Documentation Committee (IKDC) score: Medium dose, p=0.0003; high dose, p=0.0002
- Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) total score: Medium dose, p=0.0304; high dose, p=0.0002
- Knee injury and Osteoarthritis Outcome Score (KOOS) pain score: Medium dose, p=0.0018; high dose, p<0.0001
Structural changes were evaluated using MRI and X-ray measures, including the Magnetic Resonance Observation of Cartilage Repair Tissue (MOCART) 2.0 score, Whole-Organ Magnetic Resonance Imaging Score (WORMS), and Kellgren-Lawrence grade.
At week 24, the mean MOCART total score increased by 2.70 points, or 4.48%, in the medium-dose group. It increased by 1.64 points, or 3.67%, in the high-dose group. The placebo group declined by 0.90 points, or 1.62%. The difference between the medium-dose and placebo groups was statistically significant at p=0.0263.
Additional MRI findings included:
- The medium-dose group showed a significant difference from placebo for integration into adjacent cartilage, a MOCART submeasure, at p=0.0377.
- The high-dose group showed a significant difference from placebo for the surface of repair tissue at p=0.0424.
- The high-dose group showed a significant difference from placebo for marrow abnormality in the lateral femorotibial joint subregion of WORMS at p=0.041.
Kangstem Biotech CEO Jong-cheon Na said the findings reproduced the rapid onset and clinical results observed in Phase 1. The company expects the data to inform subsequent trial design, development strategy, and ongoing partnering discussions with pharmaceutical and biotechnology companies.
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