BioCardia has paused its pivotal phase III CardiAMP Cell Therapy Heart Failure trial to allow time for one-year follow-up outcomes to be analyzed. The interim data are promising, but the news caused BioCardia shares to plummet 46% to a new 52-week low.
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CardiAMP Cell Therapy, believed to be the first cardiac cell therapy to receive FDA Breakthrough Device status, uses the patient’s own bone marrow cells to potentially stimulate the natural healing response. When the cells are injected into the heart muscle, it is believed that they release proteins that signal other cells to repair themselves. The company says it’s a different mechanism of action from other therapies that have sought to transform stem cells into new heart cells, a method that has presented patient risks, such as rhythm abnormalities and cell rejection.
This trial is the first multicenter clinical trial using stem cell therapy to prospectively screen cells for therapeutic potency.
“Past trials of cell therapy delivered intramyocardially showed benefit in improving heart function,” BioCardia CEO Peter Altman, Ph.D., “but they did not reach statistical significance. We learned from these earlier approaches and added three elements that we believe improve our probability of success: a pre-procedural diagnostic for patient selection, a high target dosage of cells, and a proprietary delivery system that has been shown to be safer than other intramyocardial delivery systems and exponentially more successful in cell retention.” This approach, he says, allows the patient to be discharged from the hospital the morning after the procedure.
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The pause — unrelated to any emergent safety events — was recommended by the independent data safety monitoring board. The board’s interim review was based on available data for 132 procedures involving 111 patients from both treatment and control groups. The prespecified statistical analysis plan provided that the monitoring board would use a composite endpoint that considered survival, major adverse cardiac and cerebrovascular events, and six-minute walk distance at 12 months. It did not include other endpoints that are part of the trial, such as quality of life and heart function assessment.
The blinded data show that the survival rate at follow-up is greater than that observed in recent similar large pivotal trials in patients with heart failure with reduced ejection fraction. The blinded data also show improved health outcomes in aggregate for patients followed through all key visit dates over 12 months. The data don’t disclose differences in outcomes between the treated and control groups.
According to a Biocardai press release, the board recommended that the blind not be broken, to protect the integrity of the outcomes yet to be collected and “to ensure that the study may be restarted without compromise after completion of the one-year data analysis.” It also recommended that the company continue to treat patients already enrolled.
“We are pleased that there appear to be no treatment emergent safety issues and that the health outcomes measured in the trial show improvement for patients a compromise group,” said Altman. “We will utilize this pause to accelerate our CardiAMP Chronic Myocardial Ischemia Trial and our CardiALLO Heart Failure trial with our world-class clinical partners.”