Completion of an Investigator-Initiated Clinical Trial and Generation of Data for a Marketing Authorization Application

「During the consolidated fiscal year under review, our directors and employees worked together on the marketing-approval application and submitted an application for marketing approval for a regenerative medical product to the Ministry of Health, Labour and Welfare in April 2025.」

「During the interim consolidated accounting period under review, our directors and employees worked together on the marketing-approval application. In preparing the application materials, we incorporated long-term data into the materials in order to improve the prospects of obtaining an appropriate assessment and approval, taking into account the condition of patients with severe heart failure targeted by the investigator-initiated clinical trial.」

「In addition to publication of the above paper, a research group at Osaka University presented the results of the investigator-initiated clinical trial, the first human transplantation of a human iPS cell-derived cardiomyocyte sheet, at the American Heart Association’s scientific sessions in November 2023.」

「As described above, we are evaluating the efficacy and safety of the human iPS cell-derived cardiomyocyte sheets transplanted into 8 subjects. In September 2023, a research group at Osaka University published a paper summarizing its analysis of 3 Cohort A subjects. Following one year of observation after transplantation, the group analyzed changes in cardiac function, cardiac blood flow, the condition of heart failure and immune responses. The paper concluded that no adverse effects, worsening of the disease or serious adverse events related to the investigational product were observed, although more cases would be needed to assess therapeutic effects and immune responses. It also suggested that improvements in cardiac function had been observed.」

「We are currently evaluating efficacy and safety. In September 2023, a research group at Osaka University published a paper summarizing the analysis results for Cohort A, the first part of the investigator-initiated clinical trial, involving 3 subjects. After a one-year observation period following transplantation of the human iPS cell-derived cardiomyocyte sheet, the group analyzed changes in cardiac function, cardiac blood flow, the condition of heart failure and immune responses. The paper concluded that no adverse effects, worsening of the disease or other safety problems were observed, although the number of cases would need to be increased to assess the relationship with therapeutic effects and immune responses. It also suggested that improvements in cardiac function had been observed.」

「During the cumulative first-quarter period under review, we conducted 26-week efficacy assessments and safety assessments through Week 52. We also began preparing the application documents for marketing approval.」

「In Cohort B, one subject each underwent transplantation at Juntendo University Hospital in August and December 2022, followed by transplantation at Kyushu University Hospital and Osaka University Hospital in January 2023 and Tokyo Women’s Medical University Hospital in March 2023. This completed transplantation in the planned 5 subjects. As a result, transplantation was completed in all 8 planned subjects in the investigator-initiated clinical trial.」