MEDIPOST said on the 4th that its degenerative knee cartilage stem cell therapy CARTISTEM® received U.S. Food and Drug Administration (FDA) approval for a phase 3 investigational new drug (IND) application.
With this, MEDIPOST will enter the final clinical stage for CARTISTEM in the United States and Canada. The phase 3 trial in the United States and Canada will compare a CARTISTEM treatment group with a control group undergoing surgical debridement of cartilage. The design calls for a two-year postoperative follow-up to evaluate efficacy and safety.
The trial will be conducted at about 60 clinical sites in the United States and Canada, with hundreds of patients with moderate to severe knee osteoarthritis expected to participate. The goal is to enroll the first patient in the first quarter of this year.
CARTISTEM has been on the market in Korea since it received product approval from the Ministery of Food and Drug Safety in 2012. MEDIPOST said that long-accumulated real-world treatment data have confirmed its safety and cartilage-regeneration effect.
The company plans to use this trial to demonstrate that CARTISTEM is effective and safe for treating knee osteoarthritis as a disease-modifying therapy based on cartilage regeneration. In parallel with the U.S. phase 3 trial, it is also pursuing business development collaborations with global pharmaceutical and biotech corporations in key regions including the United States and China.
In Japan, the phase 3 trial of CARTISTEM has already been completed, and the company is preparing the final clinical study report (CSR). The results will be announced in the second quarter of this year, and after submitting a product approval application in the second half, the goal is to obtain approval in 2027.
In Dec. of last year, the company signed an exclusive distribution agreement in Japan with Teikoku Pharma and secured a 11.8 billion won upfront payment and 14.8 billion won in regulatory milestone payments. It plans to strengthen the foundation for commercialization in Japan through additional milestone payments tied to future sales performance.
Lee Seung-jin, head of global business at MEDIPOST and co-CEO of the U.S. subsidiary, said, "This U.S. phase 3 IND approval is significant in that the clinical value of CARTISTEM and its technology as an allogeneic stem cell therapy, as well as its manufacturing and quality control capabilities, have been officially recognized in the United States," adding, "We will carry out the U.S. trial without a hitch and present a new option for treating knee osteoarthritis."