お知らせ • Sep 11
Healios K.K. Announces Agreement with the FDA on Pivotal, Global Phase 3 "REVIVE-ARDS" Clinical Trial
Healios K.K. announced that as disclosed in press release "Development Plan for Acute Respiratory Distress Syndrome (ARDS)" on August 8, 2024, held an End-of-Phase 2 consultation with the FDA (Food and Drug Administration) on September 6, 2024 (U.S. time) regarding the launch of a pivotal, global Phase 3 study to demonstrate and confirm the efficacy and safety of MultiStem®? for acute respiratory distress syndrome (ARDS) caused by pneumonia, primarily in the United States (the "REVIVEARDS" study). The company reported that as a result of the meeting, the company have agreed with the FDA on the design of the REVIVE-ARDS study in accordance with request. As for the study design, the company agreed with the FDA on the use of a primary endpoint based on VFD (Ventilator Free Days: the number of days a patient does not require mechanical ventilation out of 28 days post administration in REVIVE-ARDS study, which is consistent with that utilized in the ONE-BRIDGE study previously completed in Japan). Interim analyses will be conducted at the 300 and 400 patient stages, and the REVIVE-ards study can be completed when statistical significance is confirmed. The maximum number of patients is 550. The company also confirmed the framework for utilizing 3D investigational product in this study. The specific REVIVE-ARDS study protocol and operational details will be finalized, and the study will be initiated as soon as possible. Further details will be announced in due course. With this agreement with the FDA, the company will consult with the regulatory authorities in Japan regarding the application for conditional and time-limited approval, based on the positive results of the already completed Phase 2 study (ONE-BRIDGE study) and the initiation of REVIVE-ARDS as a confirmatory study. Future Outlook. This matter has no impact on consolidated financial results of the fiscal year ending December 31, 2024 at this time. The company will promptly announce any matters that should be disclosed in the future. Company Name: HEALIOS K.K., Representative: Hardy TS Kagimoto, Chairman & CEO (TSE Growth Code: 4593), Contact: Richard Kincaid, Executive Officer CFO. Healios has a long history developing MultiStem. It originally added MultiStem to its pipeline in 2016 through an exclusive license to develop and distribute the product to treat ischemic stroke in Japan. Further, in 2018 Healios expanded its license to include development and distribution to treat ARDS in Japan, and in 2023 it expanded its ARDS license to include global territories. Having acquired the full technology platform in April 2024, Healios is seeking to advance MultiStem on a global basis for ischemic stroke, ARDS, and ARDS has received Fast Track and RMAT designation from the FDA, which allows for expedited approval of drugs that meet certain criteria for serious or life-threatening diseases or those for which no treatment is available. Acute Respiratorydistress Syndrome (ARDS)ARDS is a general term for respiratory failure that occurs suddenly in a variety of critically ill patients. Although there are many causes of ARDS, approximately one-third of ARDS cases are caused by pneumonia, and it has been confirmed that ARDS also occurs in critically ill patients with COVID-19. There is currently no approved drug therapy that can directly improve the prognosis of patients with ARDS, and respiratory failure is treated with mechanical ventilation. The mortality rate after the onset of ARDS is 30 to58%a, and there is a need for new therapies that can improve the prognosis of patients With ARDS. Currently, the number of patients in Japan is estimated to be approximately 28,000b per year, and ARDS is designated as a rare disease. However, it is estimated that 262,000c patients in the United States, 133,000*d in Europe, 670,000 in China, and more than 1.1 million people worldwide are affected annually. (Source) ARDS Diagnostic Guidelines 2016.