Groundbreaking therapy for advanced heart failure: Outcomes of the BIOVAT-HF clinical trial published in NEJM

Repairon Announces Publication of Clinical Trial Results for Engineered Human Heart Muscle Tissue in the New England Journal of Medicine

GÖTTINGEN, Germany and PROVIDENCE, R.I., May 28, 2026 (GLOBE NEWSWIRE) -- Repairon, a biotechnology company developing regenerative cardiac therapies, today announced that clinical results evaluating its engineered human heart muscle tissue for patients with advanced heart failure have been published in the New England Journal of Medicine (NEJM). The publication concludes that restoration of heart muscle function in patients with advanced heart failure is achievable, resulting in improved health and quality of life. Publication details: Zimmermann WH, Ensminger S, Kutschka I, et al. Stem-Cell-Derived Biologic Ventricular Assist Tissue in Heart Failure. N Engl J Med. May 28;394(20):1991-2001 DOI: 10.1056/NEJMoa2513525

The data were generated in the BioVAT-HF clinical trial (BiologicalVentricularAssistTissue in TerminalHeartFailure). Designed to assess safety and preliminary efficacy, this Phase 1-2 study involved implanting fully functional heart muscle patches engineered from human induced pluripotent stem cell-derived terminally differentiated heart muscle cells onto the weakened left ventricular muscle of patients with advanced heart failure with reduced ejection fraction (HFrEF), alongside guideline-directed medical therapy. The trial began with a dose-escalation phase to establish the safe maximum dose, followed by treatment at that dose to further evaluate safety and efficacy. Of the 20 patients enrolled, 16 received the safe maximum dose. At the time of reporting, the last enrolled patient had completed 3 months of follow-up, and follow-up across these patients ranged from 6 to 52 months.

Key safety findings from the NEJM Publication indicate that 3 patients died during the trial from causes that the Data Safety Monitoring Board graded as unrelated to the BioVAT. Severe adverse events were mostly related to underlying heart and concurrent diseases as well as to immunosuppression requiring adaptation of the immunosuppression regimen. 3 patients experienced episodes of ventricular tachycardia which were found to be unrelated to the BioVAT transplant. No patients had ventricular fibrillation. Among the 16 safe maximal dose patients, there were heart failure hospitalizations for 2 patients.

The efficacy findings from the NEJM Publication indicate that for those patients who received the safe maximal dose, from baseline:

• Target heart wall thickness increased 4.5 mm at 3 months and 2.9 mm at 12 months follow-up.
• Left ventricular ejection fraction increased 3.9% at 3 months and 6.9% at the latest timepoint.
• Quality of Life as measured by KCCQ-OSS increased 6.7 points at 3 months and 15 points at 12 months follow-up.
  
  

The study outcomes support preclinical findings that engineered heart muscle can integrate with damaged myocardium, form a vascularized layer, and contract in synchrony with native tissue. This was further confirmed by analysis of an explanted heart from a patient in the dose-finding cohort who later underwent cardiac transplantation, providing clear evidence of human heart remuscularization and associated increases in wall thickness, ejection fraction, and quality of life. The authors concluded that further clinical investigations with longer follow-up times are warranted.

Wolfram-Hubertus Zimmermann. MD, professor and director of the Institute of Pharmacology and Toxicology at the University Medical Center Goettingen, Germany, and principal author of the NEJM Publication, remarked that “heart failure therapies available today can often slow the progression of the disease, but they cannot replace destroyed heart muscle. Our goal, therefore, is to generate new, functional heart muscle tissue and thereby provide targeted support to the weakened heart.”

Lothar Germeroth, Ph.D., Repairon’s CEO, stated: “We are highly encouraged by these Phase II results, which we believe validate the therapeutic potential of our regenerative cardiac patch platform. Heart failure remains one of the leading causes of morbidity and mortality worldwide, and we believe these findings may open a new chapter in myocardial regeneration and restorative cardiovascular medicine.”

High medical need in advanced heart failure: Approximately 5% of the global population suffers from chronic heart failure of any severity, and it remains one of the most common causes of death. In the US, heart failure represents the most common cause of hospitalization and mortality in the senior population, and over 6 million people are affected. As heart failure progresses to advanced stages, patients experience weakness with discomfort during all physical activities and at rest, sometimes even requiring constant bed rest. For these severely ill patients, the only treatment options currently available are mechanical pump devices or heart transplantation.

About Repairon: Repairon GmbH is a German biotech company based in Göttingen, Germany, focused on developing regenerative cell therapies for cardiac medicine. The company was founded in 2014 based on research by Wolfram-Hubertus Zimmermann, MD and his team at the University Medical Center Goettingen, who have developed several tissue engineering technologies with proven applicability for organ repair and drug development. Repairon's lead therapeutic candidate, the human engineered heart muscle patch, is currently being evaluated in the BioVAT-HF Phase 2 clinical trial as a biological ventricular assist tissue (BioVAT) for end-stage heart failure.

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