Preparation for an application to expand the indication for recurrent malignant glioma and make partial changes

「For recurrent malignant glioma, we entered into agreements with Osaka Medical and Pharmaceutical University and Sumitomo Heavy Industries concerning a domestic Phase III clinical trial designed as a randomized comparative study of BNCT in patients with recurrent glioblastoma, establishing a framework for cooperation in the investigator-initiated clinical trial. The trial is scheduled to begin in spring 2026, and we intend to seek approval based on its results.」

「Following the selection in March 2025 of a research and development project for a randomized, open-label, comparative study of BNCT in patients with recurrent glioblastoma proposed by Osaka Medical and Pharmaceutical University for AMED's public call for applications for the FY2025 Innovative Cancer Medical Research and Development Project, we decided to change our development policy to cooperate in the Phase III investigator-initiated clinical trial and seek approval based on the results of that trial.」

「Following the selection of a research and development project for a randomized, open-label, comparative study of BNCT in patients with recurrent glioblastoma proposed by Osaka Medical and Pharmaceutical University for AMED's public call for applications for the FY2025 Innovative Cancer Medical Research and Development Project, we have decided to change our development policy to cooperate in the Phase III investigator-initiated clinical trial and seek approval based on the results of that trial.」

「In September 2024, the Ministry of Health, Labour and Welfare designated recurrent malignant glioma and recurrent meningioma as orphan diseases. We are also considering expanding the indication to newly diagnosed glioblastoma by cooperating in the investigator-initiated clinical trial launched by the University of Tsukuba.」

「We are also considering expanding the indication to newly diagnosed glioblastoma by cooperating in the investigator-initiated clinical trial planned by the University of Tsukuba.」

「We entered into an agreement concerning the investigator-initiated Phase I clinical trial for newly diagnosed glioblastoma planned by the University of Tsukuba.」

「Based on these developments, we will reconsider the future direction while also considering expanding the indication to newly diagnosed malignant glioma.」

「In response to this comment, we had planned to obtain the additionally requested clinical data through a retrospective study and had continued consultations with PMDA. We have discontinued the study and will reconsider the future direction.」

「In response to this comment, we had planned to obtain the additionally requested clinical data through a retrospective study and had continued consultations with PMDA. We have discontinued the study and will reconsider the future direction.」

「In response to this comment, we had planned to obtain the additionally requested clinical data through a retrospective study and had continued consultations with PMDA regarding the plan. We have now discontinued the study and will reconsider the future direction.」

「As of the filing date, we have decided to discontinue the planned retrospective study and change the development plan for recurrent malignant glioma, which had been premised on filing an application for approval based on the results of the Phase II clinical trial. Going forward, we will also consider conducting a new trial and continue developing a trial protocol in consultation with the relevant parties.」

「As a result, the one-year survival rate among 24 patients with recurrent glioblastoma was 79.2%, exceeding the prespecified expectation of 60% before the trial began. Based on these trial results, we are consulting with the Pharmaceuticals and Medical Devices Agency under the SAKIGAKE designation system toward filing a partial-change application. However, because survival, the primary endpoint of the trial, is affected by patient background factors such as age, tumour histology (grade) and preoperative general condition, we believe additional clinical data are needed to demonstrate efficacy after eliminating differences in these factors. We are currently continuing consultations on the possibility of seeking approval by submitting additional data from a retrospective study, among other measures.」

「We are currently continuing consultations on the possibility of seeking approval by submitting additional data from a retrospective study, among other measures.」

「We have since been consulting with the Pharmaceuticals and Medical Devices Agency (PMDA) toward filing a partial-change application and are preparing for an early filing while considering the possibility of conducting an additional clinical trial.」

「The Phase II clinical trial results showed a one-year survival rate of 79.2% among 24 patients with recurrent glioblastoma, exceeding the prespecified expectation of 60% before the trial began. We continue consultations with PMDA, while also preparing a trial protocol in parallel in case an additional clinical trial is required.」