· The phase 3 clinical trial will evaluate the safety and efficacy in 90 patients with mild to moderate knee osteoarthritis and determine the optimum dosage
· Synergy with CARTISTEM^®, the world’s first allogeneic cord blood-derived knee osteoarthritis treatment, is expected

The injectable drug for knee osteoarthritis developed by MEDIPOST will enter phase 2 of clinical trials in Korea.

MEDIPOST (CEO: Yang Yoon-sun, KOSDAQ 078160) announced on the 27th that the company received approval to proceed with its phase 2 clinical trial for SMUP-IA-01, a next-generation injectable drug for knee osteoarthritis. The request for approval was submitted to the Ministry of Food and Drug Safety in July.

SMUP-Cell, the active ingredient of SMUP-IA-01, is a highly efficacious allogeneic cord blood-derived mesenchymal stem cell produced with the next-generation stem cell platform technology independently developed by MEDIPOST. SMUP-IA-01 not only has excellent therapeutic efficacy but also can be mass-produced through a bioreactor, making it possible to reduce production time and cost. Furthermore, SMUP-IA-01 is rated to have overcome the difficulties in the commercialization and entry into the global market processes that the first-generation cell therapy products had in the past; these difficulties were resolved by using a frozen formulation, which solves issues in the drug’s storage and distribution.

In the phase 2 clinical trial in Korea, 90 patients with mild to moderate knee osteoarthritis (K&L Grade 2-3) will be administered a single injection SMUP-IA-01 in the knee joint. The company plans to evaluate the safety and exploratory efficacy through a 1-year follow-up period and confirm the optimum dosage for the phase 3 clinical trial. The clinical trial will be conducted at three sites: Seoul National University Hospital, Severance Hospital, and Seoul National University Bundang Hospital.

According to the estimation by the Health Insurance Review and Assessment Service, the number of knee osteoarthritis patients in Korea reached 2.78 million last year and is increasing every year as the country enters an aging society.

Currently, patients with mild to moderate (K&L grades 2-3), who account for 65% of the knee osteoarthritis patients, can only expect temporary symptom relief by taking painkillers or conservative treatment such as intra-articular hyaluronic acid (HA) injections. In recent years, regenerative medicine including stem cell therapy, such as SMUP-IA-01, is emerging as an innovative strategy for developing fundamental therapeutics.

A MEDIPOST official said, “SMUP-IA-01 is expected to become an alternative for patients with a treatment gap who may not experience improvement with drug treatment or are hesitant to undergo surgery,” and, “With CARTISTEM^®, a blockbuster first-generation cell therapy, SMUP-IA-01 will contribute to improving the quality of life of osteoarthritis patients.”

In SMUP-IA-01’s phase 1 clinical trial in Korea, 12 patients with knee osteoarthritis were given a single injection into their knee joint cavity at Seoul National University Hospital. The response to the drug was then evaluated for 6 months to prove the safety and efficacy in pain reduction and improvement of knee function.

[Glossary]

· CARTISTEM^®: The world’s first allogeneic cord blood-derived stem cell therapy product that received product approval in 2012. CARTISTEM^® is the only product with a proven effect of cartilage regeneration which requires a single administration through surgery. Its sales have grown at an average annual rate (CAGR) of 43% to date. This is MEDIPOST’s blockbuster treatment that has sold over 21,000 vials cumulatively through June 2021.

· SMUP-IA-01: This is an injectable drug for knee osteoarthritis developed with SMUP-Cell, a next-generation “high-efficiency and low-cost” cell culture platform technology that enables the selection, separation, and mass-production of high-efficiency cells through bioreactors, as well as storage under normal refrigeration for easy transportation and long-term storage. This has allowed the company to overcome limitations in production, storage, and commercialization that existed for the first-generation cell therapy.

· Kellgren Lawrence (K&L) Classification: The degree of osteoarthritis symptoms is evaluated through a knee X-ray. Depending on the gap between the knee bones, the symptom is rated in grades from 0 to 4. When the cartilage between the knee bones is normal, the symptom is rated as 0. The grade increases as the cartilage wears out and the gap between the knee bones narrows. Grade 1 can be treated with drugs, and grades 2-3 can be treated by injection, surgery, or CARTISTEM^®. Grade 4 requires arthroplasty.