Digital Outcome Measures Approved As Primary Endpoint In Cardiopulmonary Study

Bellerophon Therapeutics, a clinical-stage biotherapeutics company specializing in treatments for cardiopulmonary diseases, achieved a significant milestone by securing the first FDA endorsement of a novel wearable-derived primary endpoint — ultimately reducing the sample size required for their pivotal program by 50%.

In their Phase 2b trial, Bellerophon initially relied on traditional Clinical Outcome Assessments (COAs), including oxygen saturation (SpO₂) and the 6-Minute Walk Distance (6MWD), as primary endpoints. While these measures demonstrated clinical improvements, the results lacked statistical significance and were insufficient to support progression to a Phase 3 trial. However, the sponsor had also incorporated ActiGraph’s wearable data collection platform to capture exploratory endpoints related to Physical Activity (PA), including changes in Moderate to Vigorous Physical Activity (MVPA) from baseline.

Statistical analysis revealed that MVPA changes were both clinically meaningful and statistically significant. With ActiGraph’s support, Bellerophon presented this evidence to the FDA, leading to a groundbreaking regulatory endorsement. By integrating objective, continuous digital measures, uncover how Bellerophon not only salvaged their development program but also helped pave the way for a new era in clinical research.