Repairon Announces Positive Clinical Outcomes of Engineered Heart Muscle as Reparative Therapy for Advanced Heart Failure Reported at the Scientific Sessions of the American Heart Association

NEW ORLEANS, Nov. 11, 2025 /PRNewswire/ -- Yesterday at the Ernest N Morial Convention Center, latest clinical trial findings were disclosed on the use of human engineered heart muscle as biological ventricular assist tissue (BioVAT) transplants in patients with advanced heart failure. Wolfram Zimmermann, MD, professor and director of the Institute of Pharmacology and Toxicology at the University Medical Center Göttingen, Germany, presented the first-in-patient, first-in-class "Safety and Efficacy of Induced Pluripotent Stem Cell-derived Engineered Human Myocardium as Biological Ventricular Assist Tissue in Terminal Heart Failure - BioVAT-HF-DZHK20 - Phase II Interim Data Report".

Designed to evaluate safety and initial efficacy, BIOVAT-HF is an investigator-initiated, open label, Phase 1-2 study where functional heart muscle patches engineered from human induced pluripotent stem cells are transplanted on the weakened left ventricle muscle of patients with advanced heart failure with reduced ejection fraction (HFrEF). Following a dose escalation stage, the study included a pre-specified interim analysis after completion of 3-month follow-up after 16 patients were treated with the predetermined safe maximal dose. Safety end points included major adverse cardiac events, arrhythmic events, and frequency of immune rejection. Primary efficacy end points included change of target heart wall thickness, change of left ventricular ejection fraction, and change in quality of life determined by the Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS).

Today's reported interim analysis indicates that the BioVAT transplants were tolerated well with an acceptable safety profile. At the prespecified 3-month interim time point, the treated heart wall was thickened by 4.5 mm while left ventricular ejection fraction increased by 3.9% and KCCQ-23 OSS increased by 6.7 points. Long-term follow-up for up to 36 months showed sustained augmentation of the target heart wall by 2.7 mm, improved left ventricular ejection fraction by 6.9% and improved KCCQ-OSS by 17 points aligned with the anticipated mode of action of the living BioVAT-transplant.

Considering the unmet need in patients with advanced heart failure with reduced ejection fraction, the ability to augment heart wall thickness, improve heart function and improve quality of life by BioVAT transplantation offers a promising new option for patients refractory to the standard of care. Lothar Germeroth, Ph.D., the company's CEO, remarked that "the outcomes reported at the Scientific Sessions of the American Heart Association provide further evidence of the attractiveness of Repairon's approach to heart muscle repair. We look forward to expanding the clinical studies and the location of trial sites to benefit patients globally and ultimately demonstrate success by real world evidence".

The presented clinical observations are aligned with data collected over decades of research, including pivotal preclinical study in Rhesus macaques as well as first-in-human proof for cardiac remuscularization, published earlier this year in Nature (Jebran et al. 2025). Now, the interim data from 16 patients treated with BioVAT assembled from 20 EHM and 800 million induced pluripotent stem cell derived cardiomyocytes and stromal cells adds important evidence for safety and efficacy in heart failure patients refractory to guideline-directed therapies. Prof. Zimmermann pointed out that although the conclusions from the interim analysis must be considered early evidence "We are highly encouraged by what we have seen thus far and look forward to the transitioning of BioVAT-HF Phase II into a pivotal Phase III trial"

High medical need in advanced heart failure:  Approximately 5% of the global population suffers from heart failure, a most common cause of hospitalization and mortality. 6 million people are affected in the US. As heart failure progresses, patients experience weakness with discomfort during all physical activities and also at rest, despite optimal care. Approximately 50% of advanced heart failure patients die within 1 year. For these severely ill patients, the only treatment options currently available are mechanical pump devices or heart transplantation.

About Repairon: Repairon GmbH is a clinical-stage biotech company based in Göttingen, Germany, focused on developing reparative cell therapies for heart failure. The company was founded in 2014 based on research by Wolfram Zimmermann, MD and his team at the University Medical Center Goettingen, who have developed several tissue engineering technologies with proven applicability in organ repair and drug development. Repairon holds an exclusive license from the University Medical Center Göttingen to develop engineered heart muscle into a viable heart failure therapeutic. Together with the German Center for Cardiovascular Research (DZHK), Repairon GmbH is financing the BioVAT-HF study, which is conducted at the University Medical Centers in Göttingen and Lübeck. Additional centers in and outside Germany will join in the near future.

SOURCE Repairon GmbH