Medipost Administers First Patient in U.S. Phase 3 Trial of CARTISTEM for Knee Osteoarthritis

On July 8, Medipost announced that it has completed the first patient enrollment and first patient treatment (FPT) in the U.S. Phase 3 clinical trial of its umbilical cord blood-derived allogeneic mesenchymal stem cell therapy, CARTISTEM®.

This U.S. Phase 3 trial targets 300 patients with knee osteoarthritis and is being conducted at approximately 70 clinical trial sites across the United States and Canada. Patient enrollment and dosing will take place over about two years, followed by another two years of follow-up observation to comprehensively assess both structural improvements and clinical symptom relief. After the clinical trial is completed, Medipost plans to sequentially prepare the Clinical Study Report (CSR) and submit the Biologics License Application (BLA). With this trial design, Medipost aims to establish proof of disease-modifying osteoarthritis drug (DMOAD) efficacy based on cartilage regeneration.

This U.S. Phase 3 trial is particularly noteworthy because it was granted exceptional “single trial” approval by the U.S. Food and Drug Administration (FDA). Typically, at least two independent Phase 3 trials are required for FDA new drug approval. Medipost was able to secure this single trial approval by leveraging robust clinical data already obtained in Korea and Japan, as well as real-world evidence (RWE) from about 560 Korean patients who have been treated and followed for more than three years. The well-crafted protocol design, which satisfies the FDA’s requirement for “adequate and well-controlled clinical trials,” also played a critical role.

CARTISTEM is already a commercialized stem cell therapy for knee osteoarthritis in Korea, and Medipost has recently completed Phase 3 clinical trials in Japan and is preparing for subsequent regulatory approval there. Medipost’s strategy is to develop CARTISTEM into a global blockbuster therapy through entry into the U.S. market, actively pursuing local business development (BD) activities in parallel with the trial process. Furthermore, after obtaining approvals in the U.S. and Japan, the company plans to sequentially expand into other major global markets, including Europe and China.

Seungjin Lee, Head of Global Business at Medipost, stated, “The initiation of first patient enrollment and treatment in the U.S. Phase 3 clinical trial is not only a key milestone for global market entry but also a moment when leading groups of renowned orthopedic key opinion leaders (KOLs) in the U.S. are showing great interest in the cartilage regeneration outcomes following CARTISTEM surgery through tissue repair and remodeling.” He added, “We aim to demonstrate the therapeutic value of CARTISTEM in the world’s largest pharmaceutical market and change the paradigm of knee osteoarthritis treatment.”

Meanwhile, Medipost has completed the Phase 3 clinical trial of CARTISTEM in Japan and secured the final Clinical Study Report (CSR). The company released the clinical results in May and is proceeding with the Japanese regulatory submission process, aiming to apply for product approval by the end of this year and to obtain regulatory approval next year. Previously, in December of last year, Medipost signed an exclusive sales agreement for CARTISTEM in Japan with Teikoku Seiyaku, securing a signing fee of 11.8 billion won and regulatory milestone payments of 14.8 billion won. Additional milestone payments tied to finished product supply sales and sales performance are also expected in the future.

This content was produced with the assistance of AI translation services.