BioCardia, Inc. has announced the completion of the last protocol-specified follow-up visit in its CardiAMP HF trial, a Phase III randomized, double-blind, placebo-controlled clinical trial evaluating the CardiAMP Cell Therapy System for the treatment of heart failure with reduced ejection fraction (HFrEF). The trial aims to assess the therapy's potential to reduce all-cause mortality, future hospitalizations, and improve functional capacity, symptoms, and quality of life for patients with HFrEF.
The CardiAMP HF trial enrolled 115 patients with advanced HFrEF across 18 hospitals in the United States. Participants, already on guideline-directed medical therapy (GDMT), were randomized in a 3:2 ratio to receive either the CardiAMP autologous cell therapy or a placebo procedure. An additional open-label roll-in cohort included ten patients, bringing the total number of patients in the study to 125. The primary effectiveness follow-up ranged from 12 to 24 months.
HFrEF represents the end-stage of ischemic heart disease, affecting over a million patients in the US and millions more worldwide. Despite advancements in therapies, premature death, recurrent hospitalizations, and declining quality of life remain significant challenges for patients, their families, and healthcare systems.
CardiAMP Cell Therapy System
The CardiAMP Cell Therapy System is an investigational device that has been granted Breakthrough Device Designation by the FDA. The autologous cell therapy is administered via a minimally invasive, catheter-based procedure, with patients typically discharged after an overnight stay. The therapy is designed to stimulate microvascular repair by increasing capillary density and reducing fibrosis, effects demonstrated in preclinical studies.
Peter Altman, PhD, CEO of BioCardia, stated that previous Phase I and II trials, along with interim Phase III results, have indicated trends toward patient benefit across various outcome measures. He expressed optimism that the CardiAMP cell therapy holds promise for providing meaningful benefits to patients with heart failure.
Upcoming Milestones
BioCardia anticipates completing close-out visits and data monitoring, including source data verification, this quarter to prepare for data lock. The final data will then be transferred to the independent Statistical Data Analysis Core at the University of Wisconsin. Top-line results are expected to be released in the first quarter of 2025.
BioCardia has submitted the Annual Report for the CardiAMP Heart Failure Trial to the FDA, outlining plans for completing patient follow-up. The company intends to request a meeting with the FDA to discuss the trial results and the potential approvability of the CardiAMP Cell Therapy System.
Regulatory Pathway in Japan
BioCardia has also submitted supplementary information to Japan’s Pharmaceutical and Medical Device Agency (PMDA) in response to previous inquiries regarding the approvability of the CardiAMP Cell Therapy System based on US data. A consultation with the PMDA is scheduled for late November, serving as preparation for a subsequent clinical consultation after the CardiAMP Heart Failure Trial results are available.
Mechanism of Action
CardiAMP Cell Therapy leverages a patient’s own bone marrow cells, delivered to the heart via a minimally invasive catheter procedure, to potentially stimulate the body’s natural healing response. This aims to increase capillary density, reduce tissue fibrosis, and address microvascular dysfunction. The therapy targets mechanisms such as fibrotic, inflammatory, apoptotic, and endothelial autonomic dysfunction through the production of growth factors, cytokines, chemokines, and other factors.
The CardiAMP Cell Therapy incorporates a pre-procedural cell population analysis for patient selection and treatment planning, a high target dosage of cells, and a proprietary delivery system designed for enhanced safety and cell retention. Clinical development to date has shown trends towards enhanced patient survival, reduced major adverse cardiac events, and improved quality of life. The CardiAMP clinical development program for heart failure is supported by the Maryland Stem Cell Research Fund and is reimbursed by the Centers for Medicare and Medicaid Services (CMS) for both treatment and control procedures.
