BioCardia Completes Phase III Randomized Double-Blind Controlled Trial of Autologous Cell Therapy for Ischemic Heart Failure
BioCardia has completed its Phase III CardiAMP HF trial, a randomized, double-blind, placebo-controlled study evaluating the CardiAMP Cell Therapy System for heart failure treatment. The trial enrolled 125 patients across 18 US hospitals, with 115 patients randomized 3:2 between treatment and control groups. The therapy, which received FDA Breakthrough Device Designation, aims to reduce deaths, hospitalizations, and improve quality of life for patients with heart failure of reduced ejection fraction (HFrEF). Top-line results are expected in Q1 2025. The company has submitted plans to the FDA and is pursuing approval discussions with both FDA and Japan's PMDA.
BioCardia ha completato il suo studio di Fase III CardiAMP HF, un trial randomizzato, in doppio cieco e controllato con placebo, che valuta il sistema di terapia cellulare CardiAMP per il trattamento dell'insufficienza cardiaca. Lo studio ha arruolato 125 pazienti in 18 ospedali degli Stati Uniti, con 115 pazienti randomizzati in un rapporto 3:2 tra i gruppi di trattamento e controllo. La terapia, che ha ricevuto la Designazione di Dispositivo Innovativo dalla FDA, punta a ridurre i decessi, i ricoveri ospedalieri e migliorare la qualità della vita per i pazienti con insufficienza cardiaca a frazione di eiezione ridotta (HFrEF). I risultati preliminari sono attesi nel Q1 2025. L'azienda ha presentato piani alla FDA e sta portando avanti discussioni per l'approvazione sia con la FDA che con il PMDA giapponese.
BioCardia ha completado su ensayo de Fase III CardiAMP HF, un estudio aleatorizado, doble ciego y controlado con placebo que evalúa el Sistema de Terapia Celular CardiAMP para el tratamiento de la insuficiencia cardíaca. El ensayo inscribió a 125 pacientes en 18 hospitales de Estados Unidos, con 115 pacientes asignados al azar en una proporción de 3:2 entre los grupos de tratamiento y control. La terapia, que recibió la Designación de Dispositivo Innovador por parte de la FDA, tiene como objetivo reducir muertes, hospitalizaciones y mejorar la calidad de vida de los pacientes con insuficiencia cardíaca con fracción de eyección reducida (HFrEF). Se esperan resultados preliminares en el primer trimestre de 2025. La empresa ha presentado planes a la FDA y está llevando a cabo discusiones para la aprobación tanto con la FDA como con el PMDA de Japón.
BioCardia는 심부전 치료를 위한 CardiAMP 세포 치료 시스템을 평가하는 무작위, 이중 맹검, 위약 대조 연구인 Phase III CardiAMP HF 시험을 완료했습니다. 이 시험은 18개의 미국 병원에서 125명의 환자를 모집했으며, 115명의 환자는 치료 그룹과 대조 그룹 간의 비율 3:2로 무작위 배정되었습니다. 이 치료법은 FDA의 혁신적인 기기 지정 승인을 받았으며, 심박출 분율이 감소된 심부전(HFrEF) 환자의 사망률, 입원율을 줄이고 삶의 질을 향상시키는 것을 목표로 하고 있습니다. 최종 결과는 2025년 1분기에 발표될 예정입니다. 이 회사는 FDA에 계획을 제출했으며, FDA 및 일본 PMDA와 승인 논의 중입니다.
BioCardia a terminé son essai clinique de Phase III CardiAMP HF, une étude randomisée, en double aveugle et contrôlée par placebo, évaluant le système de thérapie cellulaire CardiAMP pour le traitement de l'insuffisance cardiaque. L'essai a inclus 125 patients dans 18 hôpitaux aux États-Unis, avec 115 patients randomisés dans un rapport de 3:2 entre les groupes de traitement et de contrôle. La thérapie, qui a reçu la désignation de Dispositif Innovant par la FDA, vise à réduire les décès, les hospitalisations et à améliorer la qualité de vie des patients atteints d'insuffisance cardiaque à fraction d'éjection réduite (HFrEF). Les résultats préliminaires sont attendus au premier trimestre 2025. L'entreprise a soumis des plans à la FDA et mène des discussions d'approbation avec la FDA et le PMDA japonais.
BioCardia hat seine Phase-III-Studie CardiAMP HF abgeschlossen, eine randomisierte, doppelblinde, placebo-kontrollierte Studie zur Bewertung des CardiAMP Zelltherapiesystems zur Behandlung von Herzinsuffizienz. Die Studie umfasste 125 Patienten in 18 US-Krankenhäusern, wobei 115 Patienten im Verhältnis 3:2 zwischen Behandlungs- und Kontrollgruppen randomisiert wurden. Die Therapie, die von der FDA als bahnbrechendes Gerät ausgezeichnet wurde, zielt darauf ab, Todesfälle und Krankenhausaufenthalte zu reduzieren sowie die Lebensqualität von Patienten mit Herzinsuffizienz mit reduzierter Ejektionsfraktion (HFrEF) zu verbessern. Die ersten Ergebnisse werden im ersten Quartal 2025 erwartet. Das Unternehmen hat Pläne bei der FDA eingereicht und führt Genehmigungsdiskussionen sowohl mit der FDA als auch mit der japanischen PMDA.
- FDA Breakthrough Device Designation received for CardiAMP Cell Therapy System
- Phase I, II, and interim Phase III results showed positive trends across outcome measures
- Trial completion milestone achieved with 125 patients enrolled
- Advancing regulatory discussions with both FDA and PMDA for potential approvals
- Final trial results still pending and success not guaranteed
- Regulatory approval uncertainty remains
Insights
The completion of BioCardia's Phase III CardiAMP HF trial marks a significant milestone in cardiovascular therapeutics. The trial's robust design - randomized, double-blind, placebo-controlled with 115 patients - provides strong scientific validity. The 3:2 randomization ratio and 12-24 month follow-up period are particularly noteworthy for evaluating long-term efficacy.
The CardiAMP therapy's FDA Breakthrough Device Designation underscores its potential impact in addressing a critical unmet need in heart failure treatment. The autologous cell therapy's minimally invasive approach and overnight hospital stay requirement suggest a favorable risk-benefit profile. Previous Phase I/II results showing positive trends across outcome measures indicate promising therapeutic potential.
With data analysis imminent and topline results expected in Q1 2025, along with ongoing regulatory discussions with both FDA and PMDA, this represents a pivotal moment for BioCardia's commercialization pathway.
SUNNYVALE, Calif., Oct. 28, 2024 (GLOBE NEWSWIRE) -- BioCardia, Inc. [Nasdaq: BCDA], a global leader in cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary diseases, announced today the completion of its last protocol specified follow-up visit in CardiAMP HF, a prospective, randomized, double-blinded, placebo procedure controlled, multi-center pivotal clinical trial for the treatment of heart failure of reduced ejection fraction (HFrEF).
The CardiAMP HF Trial is assessing the safety and effectiveness of the CardiAMP® Cell Therapy System for HFrEF, with the potential to reduce all cause death, future hospitalizations, enhance functional capacity, as well as improve symptoms and quality of life for HF patients. The study enrolled 115 advanced HF patients on guideline directed medical therapy (GDMT), who were randomized 3:2 to either the CardiAMP autologous cell therapy treatment arm or a procedure placebo control arm. Patients were enrolled in eighteen hospitals in the United States. The outcomes analysis of the randomized cohort will soon take place with primary effectiveness follow-up ranging from 12 to 24 months. CardiAMP HF also includes an open label roll-in cohort of ten patients, for a total of 125 patients.
HFrEF is an end-stage of ischemic heart disease, affecting over a million US patients and millions more worldwide. Despite advances in therapy for ischemic HFrEF, premature death, recurrent hospitalizations, and deteriorating quality-of-life remain a large burden to patients, their families, and healthcare systems worldwide.
The CardiAMP Cell Therapy System is an investigational device system that has received Breakthrough Device Designation from the FDA. The CardiAMP autologous cell therapy is delivered during a standard minimally invasive catheter-based procedure. Patients are typically discharged after an overnight stay. The cell therapy is designed to promote microvascular repair through enhanced capillary density and reduced fibrosis, both of which have been demonstrated in small and large animal models of disease.
“Phase I and II trial results and the interim Phase III trial results have shown trends toward patient benefit across most outcome measures. These results support our belief that the CardiAMP autologous, minimally invasive, cell therapy has great promise to provide a meaningful benefit for patients suffering from heart failure,” said Peter Altman, PhD, CEO of BioCardia. “We will soon complete data monitoring and then unblind the study to perform analysis for safety and effectiveness.”
Close out visits and data monitoring with source data verification to prepare for data lock are expected to be completed this quarter. Final data transfer to the independent Statistical Data Analysis Core at the University of Wisconsin is expected soon thereafter. Results are on track for top line release in the first quarter of 2025.
BioCardia has submitted the Annual Report for the CardiAMP Heart Failure Trial to FDA detailing plans for completing patient follow-up and intends to request a meeting with FDA to discuss the results with respect to approvability of the CardiAMP Cell Therapy System.
BioCardia also completed a supplementary submission to Japan Pharmaceutical and Medical Device Agency (PMDA) providing answers to PMDA’s previous responses on the approvability of the CardiAMP Cell Therapy System based on US data and requested a consultation. This PMDA consultation is scheduled to take place in late November as preparation for a subsequent clinical consultation with PMDA after results from the CardiAMP Heart Failure Trial are available.
About CardiAMP Cell Therapy
Granted FDA Breakthrough designation, CardiAMP Cell Therapy uses a patient’s own marrow cells delivered to the heart in a minimally invasive, catheter-based procedure to potentially stimulate the body’s natural healing response to increase capillary density, reduce tissue fibrosis, and ultimately treat microvascular dysfunction. The mechanisms that lead to microvascular dysfunction, including fibrotic, inflammatory, apoptotic, and endothelial autonomic dysfunction, are all targets of CardiAMP cell therapy, largely through production of growth factors, cytokines, chemokines, and other factors that directly counteract each of these mechanisms.
CardiAMP Cell Therapy incorporates three proprietary elements not previously utilized in investigational cardiac cell therapy: a pre-procedural cell population analysis for patient selection and treatment planning, 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. 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 for heart failure is supported by the Maryland Stem Cell Research Fund and is reimbursed by Centers for Medicare and Medicaid Services (CMS) for both treatment and control procedures.
CAUTION - Limited by United States law to investigational use.
About BioCardia®
BioCardia, Inc., headquartered in Sunnyvale, California, is a global leader in cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary disease. CardiAMP® autologous and CardiALLO™ allogeneic cell therapies are the Company’s biotherapeutic platforms with three cardiac clinical stage product candidates in development. These therapies are enabled by its Helix™ biotherapeutic delivery and Morph® vascular navigation product platforms.
Forward Looking Statements
This press release contains forward-looking statements that are subject to many risks and uncertainties. Forward-looking statements include, among other things, references to filings and communications with the FDA and Japan’s Pharmaceutical and Medical Device Agency, FDA and Japanese product clearances, the efficacy and safety of our products and therapies, regulatory timelines, and other statements regarding our intentions, beliefs, projections, outlook, analyses, or current expectations. Such risks and uncertainties include, among others, the inherent uncertainties associated with developing new products or technologies, regulatory approvals, unexpected expenditures, the ability to raise the additional funding needed to continue to pursue BioCardia’s business and product development plans, the ability to enter into licensing and partnering arrangements and overall market conditions.
We may use terms such as “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “approximately” or other words that convey the uncertainty of future events or outcomes to identify these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained herein, we caution you that forward-looking statements are not guarantees of future performance and that our actual results may differ materially from the forward-looking statements contained in this press release. As a result of these factors, we cannot assure you that the forward-looking statements in this press release will prove to be accurate. Additional factors that could materially affect actual results can be found in BioCardia’s Form 10-K filed with the Securities and Exchange Commission on March 27, 2024, under the caption titled “Risk Factors,” and in our subsequently filed Quarterly Reports on Form 10-Q. BioCardia expressly disclaims any intent or obligation to update these forward-looking statements, except as required by law.
FAQ
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