BioCardia’s CardiAMP Cell Therapy Heart Failure Trial Reports Interim Data

March 4, 2024—BioCardia, Inc. announced positive interim results from the phase 3 randomized controlled trial of its CardiAMP autologous cell therapy, which uses a patient’s own bone marrow cells delivered to the heart in a catheter-based procedure to potentially stimulate the body’s natural healing response. It has received the FDA’s Breakthrough Therapy designation.

The findings from the CardiAMP Cell Therapy Heart Failure trial included data from 110 randomized patients with advanced chronic heart failure with a mean 20-month follow-up. The results showed reductions in heart death equivalents and major adverse cardiac and cerebrovascular events (MACCE) with a magnified reduction among patients with elevated N-terminal pro-B-type natriuretic peptide (NT-proBNP), stated the company.

Amish Raval, MD, presented the data at THT 2024, the Technology and Heart Failure Therapeutics conference held March 4-6 in Boston, Massachusetts. Dr. Raval is Director of Clinical Cardiovascular Research and Professor of Medicine at the University of Wisconsin-Madison.

According to the company, patients with advanced chronic heart failure who received a single endomyocardial dose of autologous CardiAMP cell therapy while on maximal medical therapy had a 37% relative risk reduction in all-cause heart death equivalents and a 9% relative risk reduction in nonfatal incidence of MACCE (defined as heart attack, stroke, and hospitalization caused by heart failure) during a mean 20 months of follow-up.

Furthermore, patients treated with CardiAMP cell therapy had an approximately 5% lower rate of heart death equivalents at up to 2 years compared to control patients treated with heart failure medication alone (8.3% vs 13.2%, respectively). CardiAMP cell therapy was also associated with trends toward reduced ventricular tachyarrhythmias, enhanced heart function as measured by left ventricular ejection fraction, and improved NT-proBNP.

In a subgroup analysis of patients with elevated NT-proBNP at baseline—encompassing 59% of total enrolled randomized patients—patients treated with CardiAMP cell therapy experienced an 86.2% relative risk reduction in heart death equivalents and a 23.9% relative risk reduction in MACCE. These patients had a > 17% lower rate of heart death equivalents at up to 2 years compared to control patients treated with heart failure medication alone (2.9% vs 21.1%, respectively).

Dr. Raval, Coprincipal Investigator of the trial, commented on these findings in BioCardia’s press release.

“These positive results for CardiAMP cell therapy are very encouraging, especially for patients with elevated NT-proBNP, who encompass the majority of heart failure patients that we see in our daily practice,” stated Dr. Raval. “While the trial’s data safety monitoring board determined that the study would not meet its composite primary endpoint that included 6-minute walk distance per the trial design, the positive results for reduced heart death equivalents, reduced MACCE, and safety indicate potential for this therapy to improve outcomes for patients with advanced chronic heart failure.”

Dr. Raval continued, “Despite improvements with current medications and devices, heart failure remains at epidemic proportions, and we now have an exciting opportunity for a therapy to improve important, objective outcomes, such as mortality and hospital readmission rates. I am excited to be part of a terrific team to validate the promise of this therapy in this high responder group in the follow-on trial that is now FDA-approved and soon to treat its first patient.”

Additionally, the company announced that the first enrollment for the dose escalation safety phase of BioCardia’s phase 1/2 study of its CardiAllo allogeneic mesenchymal stem cell therapy in heart failure patients was reported at the THT scientific meeting. The cohort receiving the lowest dose of 20 million cells has been initiated with no treatment-emergent adverse events, arrhythmias, rejection, or allergic response. After completion of the dose escalation safety phase of the study, a phase 2 randomized double-blind controlled study is planned to assess efficacy.

The first author of the study is R. David Anderson, MD, Professor of Medicine at the University of Florida at Gainesville.

CardiAMP and CardiAllo therapies are considered investigational and limited by United States law to investigational use only, advised BioCardia.