[Edaily Reporter NA EUN-KYUNG ] KANGSTEM BIOTECH CO., LTD.(217730)announced on the 28th that it had confirmed the safety and efficacy of its osteoarthritis treatment, the “Purestem-OA Kit,” based on topline results from a domestic Phase 1/2a clinical trial. Improvements in cartilage structure were statistically significant compared to the placebo in some evaluation metrics. The company plans to proceed with follow-up clinical trials.

This trial was a domestic Phase 1/2a study involving 113 patients with knee osteoarthritis, comparing a medium-dose group, a high-dose group, and a placebo group. Phase 1 confirmed safety and the recommended dose (RP2D), while Phase 2a exploratively evaluated improvements in pain and function, as well as changes in cartilage structure.

In the safety evaluation, no serious adverse drug reactions (SADRs) occurred in any group. A total of three serious adverse events (SAEs) occurred, but all were cases of hospitalization or extended hospital stays; no deaths, life-threatening events, or permanent disabilities were reported.

Improvements in pain and function were also confirmed. At the 24-week mark, the KOOS pain score showed statistically significant improvement in both the medium-dose group (p=0.0018) and the high-dose group (p<0.0001) compared to the placebo group. Both treatment groups also demonstrated statistical significance compared to the placebo in key pain and function assessment measures such as WOMAC, VAS, and IKDC.

Improvements in cartilage structure were observed only in some measures. The MOCART score, which assesses the degree of cartilage regeneration, showed a statistically significant improvement in the medium-dose group compared to the placebo group (p=0.0263), but the high-dose group did not achieve statistical significance (p=0.1305). Regarding the WORMS total score—another structural assessment indicator—neither dose group showed a statistically significant difference compared to the placebo group; however, in a subgroup analysis, the high-dose group demonstrated a statistically significant improvement in the “marrow abnormality” category compared to the placebo group.

KANGSTEM BIOTECH CO., LTD. plans to prepare the Clinical Study Report (CSR) by the fourth quarter of this year, then design a follow-up clinical trial in collaboration with its domestic partner, Yuyoung Pharmaceutical, and, following consultation with the Ministry of Food and Drug Safety, begin the follow-up trial during the third quarter of 2027.