CardiAMP, a Heart Failure Cell Therapy, Misses Mark in Phase 3 Trial

Despite use being associated with increased survival and reduced major adverse cardiac and cerebrovascular events, CardiAMP failed to achieve statistical significance for the primary endpoint of the phase 3 CardiAMP-HF study.

A novel cell therapy for heart failure, which uses a patient’s own marrow cells delivered to the heart in a minimally invasive, catheter-based procedure, results of the trial indicate a single dose of CardiAMP cell therapy failed to provide a statistically significant difference for the primary composite endpoint, but subgroup analyses demonstrate potential benefit in patients with elevated NTproBNP.

“This clinical trial was conducted because there remains a large group of heart failure patients today who are insufficiently responsive to optimized heart failure medication. In this rigorous CardiAMP-HF Trial, patients who received the novel cell therapy adjunctive to medication experienced decreased mortality and MACCE, with improved quality of life, when compared to those on medication alone. These benefits appeared to be greater in patients with elevated NTproBNP - comprising fully half of treated patients - reaching statistical significance in the composite measure of these outcomes,” said Amish N. Raval, MD, FACC, professor of medicine at the University of Wisconsin School of Medicine and Public Health.2 “The trial shows us that CardiAMP therapy has the potential to safely and significantly improve survival and quality of life for heart failure patients in distress, encompassing a large group of patients we see in daily practice.”

Debuted at the American College of Cardiology (ACC) 2025 Annual Scientific Sessions, CardiAMP-HF was a double-blind, placebo-controlled, randomized clinical trial designed to assess the safety and efficacy of CardiAMP cell therapy among patients with HFrEF. Previously awarded a Breakthrough Therapy designation by the US Food and Drug Administration, the therapy boasts 3 proprietary elements not used in previous cardiac cell therapies, including a pre-procedural cell analysis to identify likely responders, a high target dosage of cells, and a minimally-invasive system for cell therapy delivery to the damaged area of the heart that has been shown to be safer and promote greater cell retention than other intramyocardial delivery systems.1,2

Investigators enrolled 115 patients in the trial. Inclusion criteria for the study required patient to have chronic ischemic cardiomyopathy with a left ventricular ejection fraction (LVEF) from 20% to 40%, NYHA class II or III heart failure on maximally tolerated guideline-directed therapies, a 6-minute walking test (6MWT distance of more than 100 and up to 450 meters, and a flow cytometry-based cell population analysis by core lab.1

The primary efficacy outcome of interest for the trial was a Comparison b/t groups of the hierarchical composite ranking of:1

1. All-cause mortality, cardiac transplant, left ventricular assist device (LVAD)
2. All non-fatal heart failure hospitalizations, myocardial infarction or stroke
3. Change in 6MWT distance from baseline to the last follow-up

Analysis of primary outcome data suggested use of CardiAMP cell therapy did not provide a statistically significant benefit for the composite outcome through 24 months (Win ratio, 1.01; P = .954). However, an analysis of major adverse cardiovascular events suggested such an event occurred in less than 25% of the treatment arm and more than 30% of individuals in the control group.1

A post-hoc subgroup analysis of patients with an NTproBNP or BNP exceeding 500 pg/mL at baseline suggested the treatment group (n=35) experienced a lower incidence of all MACE compared to the control group (n=22). A Finkelstein-Schoenfeld analysis was used to evaluate outcomes across three tiers: Tier 1 included death, heart transplant, or LVAD implantation, tier 2 included nonfatal major adverse cardiovascular events, and tier 3 assessed the Minnesota Living with Heart Failure Questionnaire scores—this analysis achieved statistical significance (P = .020).1

“The group of high responders to CardiAMP therapy represents a market of approximately one million patients in the United States alone, who today cost the healthcare system $30 billion per year for their care, most of which is inpatient care. These figures highlight that CardiAMP cell therapy also has potential to make a significant contribution toward reducing the cost of heart failure to society by improving the health of these patients,” said Peter Altman, PhD, president and chief executive officer of BioCardia.2 “We look forward to sharing the CardiAMP-HF two-year data with both the US FDA and Japan PMDA soon to align on the pathways that could make it available for physicians and their patients as soon as possible.”

References:

1. Raval AN. A Double Blind, Randomized Controlled Trial Of An Autologous Cell Therapy In Patients With HFrEF: Principal Results From The CardiAMP-HF Trial. Presented at: American College of Cardiology (ACC.25) Annual Scientific Session. March 29 – 31, 2025. Chicago, Il.

2. BioCardia Inc. BioCardia Phase 3 CardiAMP-HF Trial Of Novel Cardiac Cell Therapy For Ischemic Heart Failure Shows Increased Survival, Decreased Cardiac Events, And Improved Quality Of Life At Two Years. Biocardia.com. Published March 31, 2025. Accessed March 31, 2025. https://www.biocardia.com/investors/press-releases/id/1037?pressReleaseId=217.