Medipost announced on Tuesday that it has completed its Phase 3 clinical trial in Japan for CARTISTEM®, an umbilical cord-derived allogeneic mesenchymal stem cell therapy. The company finalized the last patient’s final visit (LPO) in November of last year and has completed site closure procedures for all clinical trial institutions.
The Japanese Phase 3 trial for CARTISTEM involved 130 patients with moderate to severe knee osteoarthritis (Kellgren–Lawrence Grade 2-3) and was conducted across 13 medical facilities in Japan.
This open-label, randomized study used sodium hyaluronate (HA) injections as a control and evaluated safety and efficacy through a one-year (52-week) follow-up period after treatment.
A notable aspect of this trial was its dual primary efficacy endpoints: patient-reported outcomes (PROs) on pain reduction and functional improvement one year post-CARTISTEM surgery, coupled with arthroscopic examination to confirm cartilage regeneration.
If statistical significance is achieved for both primary endpoints, it would not only demonstrate CARTISTEM’s ability to alleviate pain and improve function in knee osteoarthritis patients but also prove its capacity for fundamental structural improvement. This could pave the way for CARTISTEM to be recognized as a disease-modifying osteoarthritis drug (DMOAD).
With the LPO completed last November, all final patient data has been collected. The company has now entered the data verification and analysis phase, preparing to draft the Clinical Study Report (CSR). They aim to receive the CSR in the first half of this year and announce results in the second quarter.
Medipost plans to submit a product approval application in the latter half of the year, targeting Japanese market approval by the end of 2027.
Lee Seung-jin, head of Medipost’s Global Business Division and Chief Executive Officer (CEO) of MEDIPOST K.K. in Japan, stated that the completion of CARTISTEM’s Phase 3 trial in Japan marks the entry into the product approval and commercialization stage. They’re poised to strengthen our leadership in innovative stem cell therapies for knee osteoarthritis, focusing on the Japanese and North American markets.
Medipost has also secured a foundation for business in Japan by signing an exclusive sales rights licensing agreement for CARTISTEM with Teikoku Pharmaceutical last December.
This deal includes an upfront payment of 11.8 billion KRW (about 8 million USD) and a regulatory milestone payment of 14.8 billion KRW (approximately 10 million USD), based on exchange rates from December 19 last year. The company also anticipates long-term sales milestone payments and revenue from supplying active pharmaceutical ingredients and finished drugs.
Japan, with its high proportion of elderly citizens aged 65 and over, is a strategically crucial market in the global degenerative arthritis treatment landscape. Medipost plans to leverage real-world evidence from roughly 560 patients treated in Korea over three years post-surgery to support its product approval and insurance listing processes in Japan.
Importantly, Medipost received approval for its Investigational New Drug (IND) application for CARTISTEM’s Phase 3 trial in the U.S. on Wednesday. The company aims to begin patient enrollment in the first quarter of this year, with plans to pursue commercial launch in the U.S. following anticipated product approval around 2031.
