Healios KK's MultiStem Therapy Advances to Global Phase 3 Trial for ARDS

Key Insights

• Healios KK has secured FDA agreement to proceed with a pivotal global Phase 3 trial, REVIVE-ARDS, evaluating MultiStem therapy for Acute Respiratory Distress Syndrome (ARDS).
• The REVIVE-ARDS trial will enroll up to 550 patients and use Ventilator Free Days as the primary endpoint, with interim analyses planned at 300 and 400 patient milestones.
• Healios intends to pursue conditional and time-limited approval in Japan, leveraging prior Phase 2 results and the forthcoming confirmatory Phase 3 study.

Healios KK has announced the advancement of its MultiStem cell therapy into a global Phase 3 clinical trial for Acute Respiratory Distress Syndrome (ARDS), following an agreement with the U.S. Food and Drug Administration (FDA). The REVIVE-ARDS trial aims to evaluate the efficacy and safety of MultiStem in patients with ARDS, a severe inflammatory lung condition.

Trial Design and Endpoints

The REVIVE-ARDS trial is designed as a pivotal, randomized, controlled study. It will enroll up to 550 patients globally. The primary endpoint is Ventilator Free Days, a measure of the number of days a patient can breathe without mechanical ventilation within a specified period. The trial incorporates interim analyses at the 300 and 400 patient enrollment milestones, allowing for early assessment of efficacy trends.

Regulatory Strategy in Japan

Building on positive Phase 2 results, Healios plans to engage with Japanese regulatory authorities to explore conditional and time-limited approval pathways for MultiStem in ARDS. This strategy hinges on the confirmatory data expected from the REVIVE-ARDS trial.

MultiStem Therapy

MultiStem is a cell therapy product developed by Healios. It is designed to modulate the immune system and promote tissue repair. The therapy holds promise for treating various inflammatory conditions, including ARDS. ARDS is characterized by widespread inflammation in the lungs, leading to impaired oxygen exchange and often requiring mechanical ventilation. Despite advances in critical care, ARDS remains a significant cause of morbidity and mortality.

ARDS: An Unmet Medical Need

ARDS represents a critical unmet medical need, with limited effective treatment options currently available. The initiation of the REVIVE-ARDS trial signifies an important step forward in the development of a potential new therapy for this life-threatening condition. The results of this trial could have a significant impact on the management of ARDS patients worldwide.