Safety Confirmed in Both Low-Dose and High-Dose Groups
Efficacy Confirmed in Low-Dose Group Compared to High-Dose Group
However, Statistical Significance Compared to Placebo Group Not Achieved
"Placebo Group, Mild Cases and Small Sample Size Cause Interpretation Confusion"
[Asia Economy Reporter Lee Chun-hee] Kangstem Biotech announced the topline results of the Phase 1/2a clinical trial of its rheumatoid arthritis treatment ‘Purestem-RA’ on the 13th.
This clinical trial involved administering an umbilical cord blood-derived stem cell therapy to 30 patients with moderate or higher rheumatoid arthritis who were unresponsive to disease-modifying antirheumatic drugs (DMARDs). The trial assigned the low-dose group (5.0x10^7 cells), high-dose group (1.0x10^8 cells), and placebo group in a 2:2:1 ratio (12, 13, and 5 patients respectively), administering three doses at 4-week intervals followed by a 24-week observation period.
The company confirmed safety in both the low-dose and high-dose groups of Purestem-RA, and observed efficacy of the stem cell therapy in the low-dose group compared to the high-dose group.
In terms of safety, the tolerability of Purestem-RA was confirmed, with no toxic reactions or adverse drug reactions observed.
Regarding efficacy, the low-dose group was identified as the more effective therapeutic dose compared to the high-dose group. Even patients unresponsive to conventional DMARDs (cDMARDs) and biological DMARDs (bDMARDs) showed excellent improvement in rheumatoid arthritis (ACR) response in the low-dose group. Measurement of 10 cytokines showed a tendency for most inflammatory cytokines to decrease, and X-ray (mSHS) results confirmed that the bone erosion phenomenon known as ‘golmiran’ was halted or alleviated.
However, the ACR response rate in the low-dose group did not show a statistically significant difference compared to the placebo group. The company explained, “The placebo group had only 5 subjects, and the mildest patients at baseline were assigned to this group,” adding, “It is believed that the placebo effect was excessively expressed in some patients, causing confounding.” The company also emphasized that this clinical trial was “an exploratory study aimed at identifying the appropriate therapeutic dose and evaluating treatment potential at an early stage,” and that statistical significance testing was not the primary objective.
Baeyo-han, Head of Clinical Development at Kangstem Biotech, stated, “It is meaningful to have secured exploratory efficacy and safety for the first domestic stem cell therapy for rheumatoid arthritis,” and added, “Based on these results, we plan to promptly prepare follow-up clinical trials to fully evaluate efficacy and submit an Investigational New Drug (IND) application within this year.”
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