Medipost(CEO Wonil Oh) announced the results of the domestic phase 2 clinical trial of Pneumostem, a treatment for bronchopulmonary dysplasia, on the 18th. Pneumostem is a treatment developed using allogeneic cord blood-derived mesenchymal stem cells as its main ingredient and is aimed at preventing and treating bronchopulmonary dysplasia (BPD) in premature infants.
Bronchopulmonary dysplasia is a chronic lung disease that occurs in premature infants who have received artificial ventilation and oxygen therapy. It is considered one of the main causes of death in premature infants and represents an unmet medical need, requiring intensive treatment for the survival and normal development of premature infants. It is an absolutely necessary disease.
Pneumostem was designated as an orphan drug by the U.S. Food and Drug Administration (FDA) in 2013 and the European Medicines Agency (EMA) in 2015.
After obtaining approval for domestic phase 2 clinical trials in December 2017, Medipost will provide 2 groups: a test group (Pneumostem group) and a control group (placebo group) for 60 extremely premature babies less than 25 weeks of gestation by January 2024. Superiority over placebo was evaluated by comparing the proportion of subjects who developed BPD or died related to lung disease at 36 weeks after administering the drug once through intratracheal intubation.
As a result of clinical trials, no clinically significant differences were observed in the safety evaluation of Pneumostem administration compared to the placebo control group, and as a result of the final judgment in connection with the results of previous clinical trials, it was confirmed that intra-airway administration of Pneumostem is safe. . In the analysis of the primary efficacy endpoint, the Pneumostem treatment group showed a lower incidence of severe BPD compared to the control group, but it was insufficient to confirm statistically significant effectiveness.
Severe BPD, a symptom of a very serious disease, showed an incidence rate of 22.58% in the Pneumostem group, according to the 2019 annual report of the Korean Neonatal Network (KNN), in patients with a gestational age of 24 weeks or less. In the case of premature infants, the incidence of severe BPD is reported to be 73.9%, and compared to the 60% incidence of severe BPD in the control group at 23 and 24 weeks of gestation in previous clinical trials, intra-airway administration of Pneumostem is effective in reducing severe BPD. It is believed to be effective in reducing the occurrence.
In addition, it was confirmed that the period of tracheal intubation and ventilator treatment was shorter in the test group compared to the control group, and statistical significance was secured (p=0.0296).
Regarding the clinical results, a Medipost official said, “Through the ongoing 5-year long-term follow-up study, Pneumostem observed in this clinical trial not only reduced the incidence of severe bronchopulmonary dysplasia but also shortened the period of tracheal intubation and ventilator treatment. “We plan to track the clinical significance.”
In addition, regarding future plans, “In accordance with the revision of the Act on Safety and Support of Advanced Regenerative Medicine and Advanced Biopharmaceuticals (Regenerative Medicine Act), which is scheduled to take effect in February next year, we are reviewing the development of Pneumostem as an advanced regenerative medicine treatment through the advanced regenerative medicine research and treatment track. “Based on the partially meaningful therapeutic effects shown in the Phase 2 clinical trial results, we plan to continue discussing technology export and partnering with global big pharma companies in advanced overseas markets that are interested in orphan drugs,” he said.
