Key findings
- FDA gives the nod for a Phase 3 trial of CARTISTEM, an umbilical cord blood-derived allogeneic mesenchymal stem cell (MSC) therapy for knee osteoarthritis.
- The randomized, double-blind study plans to enroll several hundred patients across about 60 sites in the US and Canada, with 2 years of follow-up.
- Medipost is targeting first patient in Q1 2026.
The FDA has approved Medipost’s Investigational New Drug (IND) application to start a Phase 3 clinical trial of CARTISTEM, an umbilical cord blood-derived allogeneic mesenchymal stem cell (MSC) therapy for Knee Osteoarthritis (KOA).
This may be a historic moment for the regenerative medicine industry soon.
Short history of CARTISTEM
In 2012, I believe it became the world’s first approved umbilical cord blood product (note that it was only approved in Korea, where MEDIPOST is based). They’ve published a few studies with before/after arthroscopic images, MRIs, and post-administration biopsies to confirm that cartilage cells were generated. Based on this and their US Phase 1/2a results, the FDA gave them a Phase 2 waiver, and they’re now heading into Phase 3.
They also wrapped up Phase 3 in Japan in 2025, expect to see those results in the next few months. It’s been marketed in Korea since receiving product approval from the Ministry of Food and Drug Safety in 2012, and may have a true Disease-Modifying Osteoarthritis Drug (DMOAD) for cartilage regeneration on their hands.
Trial design
The Phase 3 trial will be a randomized, double-blind, multinational study comparing CARTISTEM with surgical debridement. The trial will track efficacy and safety over a 2-year follow-up period after surgery.
The trial is expected to run at approximately 60 clinical sites across the US and Canada. Medipost will enroll several hundred patients with moderate to severe knee osteoarthritis (Kellgren–Lawrence Grade 2~3), randomized into a CARTISTEM treatment group and a control group, and they’re aiming for Q1 2026.
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