BioCardia Reports Completion of Low Dose Cohort Enrollment for CardiALLO Phase I/II Clinical Trial of BCDA-03 Allogeneic Mesenchymal Stem Cells to Treat Ischemic Heart Failure of Reduced Ejection Fraction (HFrEF)
BioCardia (NASDAQ: BCDA) has completed enrollment and dosing in the low dose cohort of its CardiALLO™ Allogeneic Mesenchymal Cell Therapy Phase I/II trial. The study targets patients with ischemic heart failure of reduced ejection fraction (HFrEF) who have elevated markers of heart stress and systemic inflammation.
The trial consists of a Phase I nine-patient dose escalation cohort, followed by a thirty-patient randomized double-blinded placebo procedure-controlled cohort. The Phase I study will test three escalating doses: 20 million, 100 million, and 200 million cells, using BioCardia's minimally invasive delivery system. The first Data Safety Monitoring Board review is expected in March 2025.
The therapy utilizes 'off the shelf' mesenchymal stem cells, manufactured at BioCardia's Sunnyvale facility, and is delivered using the FDA-approved Morph DNA steerable guide. The treatment aims to provide immunomodulatory benefits and enhance microvascular repair in heart failure patients.
BioCardia (NASDAQ: BCDA) ha completato l'arruolamento e la somministrazione nel gruppo a basso dosaggio della sua sperimentazione clinica di fase I/II CardiALLO™ per la terapia cellulare mesenchimale allogenica. Lo studio è rivolto a pazienti con insufficienza cardiaca ischemica con frazione di eiezione ridotta (HFrEF) che presentano marcatori elevati di stress cardiaco e infiammazione sistemica.
La sperimentazione consiste in un gruppo di fase I con nove pazienti per l'escalation del dosaggio, seguito da un gruppo randomizzato, in doppio cieco, controllato con placebo di trenta pazienti. Lo studio di fase I testerà tre dosi crescenti: 20 milioni, 100 milioni e 200 milioni di cellule, utilizzando il sistema di somministrazione minimamente invasivo di BioCardia. La prima revisione del Data Safety Monitoring Board è prevista per marzo 2025.
La terapia utilizza cellule staminali mesenchimali 'pronte all'uso', prodotte presso la struttura di BioCardia a Sunnyvale, e viene somministrata utilizzando il guidatore steerable Morph DNA approvato dalla FDA. Il trattamento mira a fornire benefici immunomodulatori e migliorare la riparazione microvascolare nei pazienti con insufficienza cardiaca.
BioCardia (NASDAQ: BCDA) ha completado el reclutamiento y la dosificación en el grupo de dosis baja de su ensayo clínico de fase I/II CardiALLO™ para la terapia celular mesenquimal alogénica. El estudio se dirige a pacientes con insuficiencia cardíaca isquémica con fracción de eyección reducida (HFrEF) que presentan marcadores elevados de estrés cardíaco e inflamación sistémica.
El ensayo consiste en un grupo de fase I con nueve pacientes para la escalada de dosis, seguido de un grupo controlado aleatorizado, doble ciego y controlado con placebo de treinta pacientes. El estudio de fase I probará tres dosis crecientes: 20 millones, 100 millones y 200 millones de células, utilizando el sistema de administración mínimamente invasivo de BioCardia. Se espera la primera revisión del Data Safety Monitoring Board en marzo de 2025.
La terapia utiliza células madre mesenquimatosas 'listas para usar', fabricadas en la instalación de BioCardia en Sunnyvale, y se administra utilizando la guía steerable Morph DNA aprobada por la FDA. El tratamiento tiene como objetivo proporcionar beneficios inmunomoduladores y mejorar la reparación microvascular en pacientes con insuficiencia cardíaca.
BioCardia (NASDAQ: BCDA)는 자사의 CardiALLO™ 동종 중간엽 세포 치료제 1/2상 임상 시험의 저용량 집단에서 환자 등록 및 투여를 완료했습니다. 이 연구는 심장 스트레스 및 전신 염증의 지표가 상승한 저유출량 심부전(HFrEF) 환자를 대상으로 합니다.
시험은 9명의 환자를 대상으로 하는 1상 용량 증량 집단으로 구성되며, 이후 30명의 환자를 대상으로 하는 무작위 이중 맹검 위약 대조 집단이 이어집니다. 1상 연구에서는 2천만, 1억, 2억 세포의 세 가지 증량 용량을 테스트하며, BioCardia의 최소 침습적 투여 시스템을 사용합니다. 첫 번째 데이터 안전성 모니터링 위원회의 검토는 2025년 3월로 예정되어 있습니다.
이 치료법은 BioCardia의 써니베일 시설에서 제조된 '즉시 사용' 가능한 중간엽 줄기 세포를 활용하며, FDA 승인된 Morph DNA 조절 가능한 가이드를 사용하여 전달됩니다. 이 치료의 목적은 면역 조절 이점을 제공하고 심부전 환자의 미세혈관 수리를 향상시키는 것입니다.
BioCardia (NASDAQ: BCDA) a terminé le recrutement et l'administration dans le groupe à faible dose de son essai clinique de phase I/II CardiALLO™ pour la thérapie cellulaire mésenchymateuse allogénique. L'étude cible les patients souffrant d'insuffisance cardiaque ischémique avec une fraction d'éjection réduite (HFrEF) qui présentent des marqueurs élevés de stress cardiaque et d'inflammation systémique.
L'essai se compose d'un groupe de phase I avec neuf patients pour l'escalade des doses, suivi d'un groupe randomisé, en double aveugle et contrôlé par placebo de trente patients. L'étude de phase I testera trois doses croissantes : 20 millions, 100 millions et 200 millions de cellules, en utilisant le système d'administration minimalement invasif de BioCardia. La première révision du Data Safety Monitoring Board est prévue pour mars 2025.
La thérapie utilise des cellules souches mésenchymateuses 'prêtes à l'emploi', fabriquées dans l'installation de BioCardia à Sunnyvale, et est administrée à l'aide du guide steerable Morph DNA approuvé par la FDA. Le traitement vise à fournir des avantages immunomodulateurs et à améliorer la réparation microvasculaire chez les patients souffrant d'insuffisance cardiaque.
BioCardia (NASDAQ: BCDA) hat die Rekrutierung und Dosierung in der Niedrigdosisgruppe seiner Phase I/II-Studie zur allogenen mesenchymalen Zelltherapie CardiALLO™ abgeschlossen. Die Studie richtet sich an Patienten mit ischämischer Herzinsuffizienz mit reduzierter Ejektionsfraktion (HFrEF), die erhöhte Marker für Herzstress und systemische Entzündungen aufweisen.
Die Studie besteht aus einer Phase-I-Gruppe mit neun Patienten zur Dosissteigerung, gefolgt von einer randomisierten, doppelt verblindeten, placebo-kontrollierten Gruppe mit dreißig Patienten. Die Phase-I-Studie wird drei steigende Dosen testen: 20 Millionen, 100 Millionen und 200 Millionen Zellen, unter Verwendung des minimalinvasiven Verabreichungssystems von BioCardia. Die erste Überprüfung durch das Data Safety Monitoring Board wird für März 2025 erwartet.
Die Therapie verwendet 'regalfertige' mesenchymale Stammzellen, die in der Einrichtung von BioCardia in Sunnyvale hergestellt werden, und wird mit dem von der FDA genehmigten steerable Morph DNA Guide verabreicht. Die Behandlung zielt darauf ab, immunmodulatorische Vorteile zu bieten und die mikrovasculäre Reparatur bei Patienten mit Herzinsuffizienz zu verbessern.
- Completed enrollment in low dose cohort of Phase I/II trial
- Using FDA-approved Morph DNA guide for enhanced delivery precision
- Previous clinical trials showed safety and efficacy trends without requiring immunosuppression
- Manufacturing process is commercially scalable
- None.
Insights
The completion of low-dose cohort enrollment in BioCardia's CardiALLO trial marks a significant milestone in the development of allogeneic MSC therapy for heart failure. The trial's innovative approach incorporates three key differentiators that enhance its potential for success:
1. Precision Medicine Strategy: By specifically targeting patients with elevated markers of heart stress and inflammation, BioCardia is employing a biomarker-driven approach that could significantly improve response rates. This patient selection strategy represents a sophisticated evolution in regenerative medicine, moving away from the traditional one-size-fits-all approach.
2. Enhanced Delivery Mechanism: The integration of the FDA-approved Morph DNA steerable guide for therapeutic delivery addresses one of the historical challenges in cardiac cell therapy - precise and controlled cell delivery to target areas. This technological advancement could improve treatment efficacy and standardization.
3. Scalable Manufacturing: BioCardia's in-house manufacturing capability for both cells and delivery systems positions them favorably for commercial scale-up, particularly significant given the recent first U.S. MSC therapy approval, which validates the regulatory pathway for this class of treatments.
The trial's design, incorporating both dose-escalation and randomized controlled phases, provides a robust framework for safety and efficacy evaluation. The previous clinical evidence showing safety without immunosuppression requirements is particularly noteworthy, as it could significantly reduce treatment complexity and costs.
The company's dual development strategy with CardiALLO (allogeneic) and CardiAMP (autologous) therapies creates multiple paths to market, while their manufacturing platform's versatility enables expansion into additional indications like PulmAllo for respiratory distress. The potential for conditional approval in Japan, known for its accelerated regenerative medicine pathway, could provide earlier market access and revenue generation opportunities.
Focus on Patients with Elevated Markers of Heart Stress and Inflammation
Procedural enhancement using FDA Approved Morph DNA steerable guide for therapeutic delivery
SUNNYVALE, Calif., Feb. 13, 2025 (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 completion of enrollment and dosing in the low dose cohort in its CardiALLO™ Allogeneic Mesenchymal Cell Therapy Phase I/II trial. The trial is designed to treat patients with ischemic heart failure of reduced ejection fraction (HFrEF) and is believed to be the world’s first prospective trial of allogeneic mesenchymal stem cells (MSC) intended for treating HFrEF patients having elevated markers of heart stress and systemic inflammation.
“This novel investigational cell therapy has great potential to help these patients, who suffer from significant lifestyle limitations despite receiving guideline directed medical therapy,” said Carl Pepine, M.D., Professor of Medicine at the University of Florida Division of Cardiovascular Medicine at Gainesville, and National Principal Investigator for the study. “The higher dosing and precision medicine for patients most likely to be responsive to this novel allogeneic therapy are very promising enhancements.”
“The treatment procedures have gone well, and we look forward to the first independent safety review,” said R. David Anderson, M.D., Professor of Medicine in the University of Florida Division of Cardiovascular Medicine and the Director of Interventional Cardiology and Cardiac Catheterization, and Principal Investigator for the study at the University of Florida at Gainesville. “As part of this therapy, we are utilizing the Morph DNA guide to navigate, enhancing physician control for cell delivery. Based on experience to date in this study, it is a compelling product improvement.”
The CardiALLO Heart Failure Trial is a Phase I/II study with an open label Phase I nine-patient dose escalation cohort, followed by a thirty-patient randomized double-blinded placebo procedure-controlled cohort, intended to develop support for safety and efficacy of this treatment. For Phase I, three patients are to be treated at one of three escalating doses of 20 million cells, 100 million cells, and 200 million cells, using BioCardia’s minimally invasive delivery system which enters the heart through a blood vessel. A two-week waiting period between each dose cohort is built into the study design with a formal Data Safety Monitoring Board (DSMB) review following each dose. These proprietary CardiALLO human cells and their dedicated proprietary catheter delivery system are manufactured at BioCardia’s facility in Sunnyvale, California. The first DSMB review is expected to take place in March 2025.
The “off the shelf” mesenchymal stem cells utilized for the study treatment are believed to act by providing immunomodulatory benefits in inflammatory disease mediated heart failure as well as microvascular repair promoting enhanced capillary density and reduced fibrosis. This program follows two previous clinical trials of allogeneic MSC delivered intramyocardially for patients with ischemic heart failure sponsored by BioCardia1,2. These completed trials provide evidence for safety and trends of efficacy for these allogeneic cells which have not required immunosuppression or produced arrhythmias.
“We have confidence in the CardiALLO MSC program whose current study is intended to position this therapy for pivotal clinical studies in the United States and conditional time limited approval in Japan,” said Peter Altman, PhD, BioCardia President and CEO. “Our enhanced manufacturing of these MSC is expected to be commercially scalable. The CardiALLO MSC development is synergistic to our autologous CardiAMP mononuclear cell therapy development, which has shown great promise and has potential to be many years closer to market, particularly if the upcoming data to be presented at the American College of Cardiology meeting in March confirms the interim results.”
BioCardia’s allogeneic MSC manufacturing for the CardiALLO MSC program is able to support partnering for its PulmAllo™ MSC acute respiratory distress IND3, an indication where a conditional approval in Japan is expected for MSC4, as well as other preclinical and clinical indications where partners may have interest. A first MSC therapy was recently approved in the United States5.
About BioCardia
BioCardia, Inc., headquartered in Sunnyvale, California, is developing 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 for the treatment of heart disease. BioCardia also acts as a biotherapeutic delivery partner supporting therapies for the treatment of heart failure, chronic myocardial ischemia, and acute myocardial infarction. For more information visit: www.BioCardia.com.
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, statements relating to the mechanisms of action of CardiALLO MSC therapy, future enrollment in our ongoing clinical trial, anticipated milestones and events, the likelihood of safety and patient benefit, future regulatory approvals, and the ultimate success of our clinical cell therapy programs. These forward-looking statements are made as of the date of this press release.
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. Factors that could cause or contribute to such differences include, but are not limited to, the Company’s liquidity position and its ability to raise additional funds, as well as the Company’s ability to successfully progress its clinical trials. 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 its 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.
References:
- Hare JM, Fishman JE, Gerstenblith G, DiFede Velazquez DL, Zambrano JP, Suncion VY, Tracy M, Ghersin E, Johnston PV, Brinker JA, Breton E, Davis-Sproul J, Schulman IH, Byrnes J, Mendizabal AM, Lowery MH, Rouy D, Altman P, Wong Po Foo C, Ruiz P, Amador A, Da Silva J, McNiece IK, Heldman AW, George R, Lardo A. Comparison of allogeneic vs autologous bone marrow–derived mesenchymal stem cells delivered by transendocardial injection in patients with ischemic cardiomyopathy: the POSEIDON randomized trial. JAMA. 2012 Dec 12;308(22):2369-79. Erratum in: JAMA. 2013 Aug 21;310(7):750. George, Richard [added]; Lardo, Albert [added]. PMID: 23117550; PMCID: PMC4762261.
- Florea V, Rieger AC, DiFede DL, El-Khorazaty J, Natsumeda M, Banerjee MN, Tompkins BA, Khan A, Schulman IH, Landin AM, Mushtaq M, Golpanian S, Lowery MH, Byrnes JJ, Hendel RC, Cohen MG, Valasaki K, Pujol MV, Ghersin E, Miki R, Delgado C, Abuzeid F, Vidro-Casiano M, Saltzman RG, DaFonseca D, Caceres LV, Ramdas KN, Mendizabal A, Heldman AW, Mitrani RD, Hare JM. Dose Comparison Study of Allogeneic Mesenchymal Stem Cells in Patients With Ischemic Cardiomyopathy (The TRIDENT Study). Circ Res. 2017 Nov 10;121(11):1279-1290.
- NCT 05491681: AllogeneiC Expanded Human MSC Therapy in Patients Recovering From COVID-19 Acute Respiratory Distress Trial (ACE_CARD)
- Helios Press release January 15, 2025: “Status of Conditional and Time-Limited Approval Application for ARDS in Japan (Clinical Part)”
- https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/ryoncil
FAQ
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