PPMX-T003 Phase 1 Trial for Polycythemia Vera Enters the Patient Stage

「For this reason, the company first selected polycythemia vera (PV), a disease characterized by abnormally increased red blood cells, as the target indication, conducted a domestic Phase I trial, and completed it in June 2024.」

「Regarding the results of this Phase I trial, the company reported at the 66th American Society of Hematology (ASH) Annual Meeting held in December 2024 that a 12-week phlebotomy-free period had been achieved in 5 of the 6 subjects, excluding 1 case discontinued for subject-related reasons, and that efficacy was suggested by red blood cell parameters, including hematocrit and hemoglobin, in all 6 subjects.」

「The company conducted a domestic Phase I trial targeting PV and completed it in June 2024. As of the date of submission of this document, all trial data had been locked and preparation of the clinical study report was underway. At The 14th JSH International Symposium 2024, held in July 2024, the company reported that safety and pharmacokinetics, the primary endpoints, had been confirmed in the 5 subjects whose data had been locked, and that an extension of the phlebotomy-free period, a secondary endpoint, had been suggested.」

「The scheduled completion date for the trial was changed from March 2024 to June 2024. However, as announced on 10 May 2024, the trial is now expected to be completed by the end of July 2024 because the final patient received a high dose based on the physician's overall assessment, taking into account the patient's individual circumstances.」

「As announced on 2 February 2024, based on the status of the trials for the 2 patients currently participating, completion of post-dose observation is expected to take longer than initially anticipated. The trial is expected to be completed in June 2024.」

「As of the date of submission of this document, all 6 PV patients targeted for enrollment in the trial had been enrolled. The trials for 3 of these patients have been completed, and initial dosing has also been completed for the remaining 3 patients. The trial is scheduled for completion during the current fiscal year.」

「The trial is designed to enroll 6 PV patients, and enrollment is being promoted by increasing the number of clinical trial sites to 8. As of the date of submission of this document, the enrollment of 1 additional patient brought cumulative enrollment to 4 patients. In an interim report presented by the principal investigator in May, all 3 patients who had completed dosing were reported to have had no serious adverse reactions, with safety and therapeutic effects comparable to those in healthy individuals. Preparations are currently underway to enroll the remaining 2 patients, and the trial is scheduled for completion during the current fiscal year.」

「The company is currently conducting a Phase I trial in 6 patients with polycythemia vera, and 3 patients received dosing during the fiscal year ended March 2023. No serious adverse reactions have been observed to date, and the trial is progressing smoothly. Additional clinical trial sites are being added to enroll the remaining 3 patients as soon as possible, bringing the total to 7 sites as of the date of submission of this document. Procedures for 1 additional site are also underway, and the trial is scheduled for completion during the fiscal year ending March 2024.」

「The trial is designed to enroll 6 patients, but recruitment has been more difficult than initially anticipated, so the eligibility criteria were reviewed to reflect real-world clinical practice. As a result, dosing had begun for 3 patients as of the date of submission of this quarterly report. However, because it is no longer possible to foresee dosing all 6 patients during the fiscal year ending March 2023, the trial is expected to be completed during the fiscal year ending March 2024. To complete enrollment of the remaining 3 patients as soon as possible, additional clinical trial sites are being added. Registration of 1 site has already been completed, and procedures for the other sites are underway.」

「Following a review of the eligibility criteria for PV patients to reflect real-world clinical practice, dosing began for the first and second patients in August 2022. Preparations are also underway to add clinical trial sites, and there is no change to the original schedule for completing the Phase I trial in PV patients.」

「For recruitment of patients with PV, after reviewing the eligibility criteria to reflect real-world clinical practice, the first patient is now expected to receive the drug during the second quarter of the current fiscal year. We are also preparing to add clinical trial sites, and the planned completion date for the Phase 1 trial in PV patients remains unchanged.」

「We will amend the clinical trial protocol to broaden the eligibility criteria for patients and aim to complete the Phase 1 trial during fiscal 2022.」

「After confirming safety and pharmacological effects in healthy volunteers in March 2021, we are currently recruiting patients with PV in preparation for administration. Recruitment has been slightly delayed compared with the initial expectations, but the planned completion date for the Phase 1 trial in PV patients remains unchanged.」

「After completing the Phase 1 trial in healthy volunteers, the clinical trial notification submitted to the Pharmaceuticals and Medical Devices Agency (PMDA) on 31 May 2021 was accepted in June 2021, and we signed contracts with the clinical trial sites. We are currently preparing to administer the drug to patients with PV.」

「In March 2021, we confirmed both safety in healthy volunteers and a reduction in erythroblasts. To conduct the next-step Phase 1 trial in patients with polycythemia vera, we submitted a clinical trial notification to the Pharmaceuticals and Medical Devices Agency (PMDA) on 31 May 2021.」

「We began a Phase 1 trial in November 2019 for polycythemia vera treatment, and completed the Phase 1 trial in healthy volunteers in March 2021. After confirming safety in healthy volunteers, we submitted a clinical trial notification to the Pharmaceuticals and Medical Devices Agency (PMDA) on 31 May 2021 to administer the drug to patients with polycythemia vera and complete the Phase 1 trial as the next step.」