BioCardia unveils interim data that led it to stop PhIII enrollment of heart failure stem cell therapy

BioCardia disclosed that its heart failure stem cell therapy after an interim analysis failed to hit on all three measures that comprised the primary endpoint of a Phase III study.

In 102 patients, there was no statistically significant difference in rates of death, major cardiovascular events and performance on the six-minute walk test (6MWT) at one year between those who got BioCardia’s treatment and a sham, according to BioCardia’s press release Tuesday morning.

BioCardia said that based on the unblinded results from the one-year interim analysis, the full study, which is supposed to enroll 250 patients, likely won’t meet its final efficacy goals. The announcement is unsurprising: In July, the biotech stopped enrollment on the study after an independent review board said the trial was not likely to succeed and pointed out that the study was enrolling very slowly.

BioCardia’s treatment delivers a high dose of a patient’s own bone marrow cells to the heart via a catheter, and the biotech was testing the treatment for systolic heart failure.

At one year, 5.6% of patients who received BioCardia’s stem cell therapy either died, received a heart transplant or a heart pump called a left ventricular assist device (LVAD) — a cardiac death equivalent — compared to 5.3% in the control group, which received sham surgeries. In terms of cardiovascular events, 16.7% in the treatment group experienced hospitalization from heart failure, stroke or heart attack, compared to 15.8% in the control group at one year.

As for the 6MWT, both groups saw improvements but the difference between the two groups was not statistically significant, BioCardia said. Those who received BioCardia’s treatment saw their scores increase by 36.1 meters (with a standard deviation of ±70 meters), while those who received the sham surgery saw their scores increase by 33.4 meters.

Peter Altman

The future of BioCardia’s therapy is unclear. “We are actively engaged in identifying patients who responded the most to the therapy and are considering other learnings with respect to trial design to inform this program and our other two ongoing clinical programs,” BioCardia president and CEO Peter Altman said in a press statement. He also noted that patients enrolled on the trial would be completing treatment in the next six weeks.

BioCardia is also testing a bone marrow treatment for chronic myocardial ischemia and an “off-the-shelf” therapy for heart failure.

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