Clinical

Mont-Saint-Guibert, Belgium, June 10, 2013 –The Belgian biotechnology company, Cardio3 BioSciences (C3BS), a leader in the discovery and development of regenerative, protective and reconstructive therapies for the treatment of cardiac diseases, announces it has treated its first patients in its Congestive Heart failure Cardiopoietic Regenerative Therapy (CHART-1) European Phase III trial for C-Cure®. The patients, who have been randomly allocated to either the treatment or sham arm of the trial, were treated at the CHU Charleroi, Belgium.

The CHART-1 trial represents the world’s first Phase III trial for a pre-programmed cellular therapy targeting heart failure.

The Cardio3 BioSciences’ most advanced therapy C-Cure®, involves taking cells from a patient’s bone marrow and through a proprietary process called Cardiopoiesis, re-programming those cells so that they become heart precursor cells with the aim of replicating the normal process of cardiac development in the embryo and healing the failing heart. The cells, known as cardiopoietic cells, are then injected back into the patient’s heart through a minimally invasive procedure using a proprietary catheter called C-Cath®ez ® , with the goal of repairing damaged tissue and improving heart function, clinical outcomes and quality of life. C-Cure® builds on research conducted at Mayo Clinic (Rochester, Minnesota, USA), Cardio3 BioSciences (Mont-Saint-Guibert, Belgium) and Cardiovascular Centre Aalst (Aalst, Belgium). This Phase III trial builds on the successful outcome of the Phase II trial of which the results were recently published in the Journal of the American College of Cardiology (JACC).

Dr Christian Homsy, CEO of Cardio3 BioSciences, said: “Heart failure remains a significant burden to patients, families and society in general. It is associated with high morbidity, mortality and escalating healthcare costs. We believe C-Cure® has the potential to become a treatment which goes beyond symptom relief towards healing heart tissue. It is a huge step for us to have the first patients undergo their cardiac injection in the Phase III trial and we are confident that we will be able to confirm the promising results we have already seen in our Phase II study.”

Dr Dariouch Dolatabadi, the physician that performed the first two procedures added: “Regenerative therapies have the potential to revolutionize the treatment of heart disease and other conditions and we are excited to be working with Cardio3 BioSciences on what is a ground breaking study. The results seen in the earlier trial were encouraging as was reflected in the recent publication in JACC. If repeated in this larger study, those results would bring nearer a potentially disruptive treatment for the expanding epidemic of heart failure.”

Prof. Jozef Bartunek, CHART-1 Study Principal Investigator, said: “Heart failure is a very common and very serious condition. A person living to age 40 years has a one in five risk of developing heart failure and, once the disorder is apparent, a one in three chance of dying within a year of diagnosis. Regenerative medicines offer the potential, for the first time, of repairing the failing heart. C-Cure® could therefore mark a significant step forward in treatment.”

The Phase III trial is a prospective, multi-centre, randomized, sham-controlled, blinded study (patients and evaluators) comparing treatment with C-Cure® to a sham treatment. The trial will recruit a minimum of 240 patients with chronic advanced heart failure of ischemic origin. The primary endpoint of the trial is a composite endpoint including mortality, morbidity, Six Minute Walk Test, quality of life and left ventricular structure and function at 9 months post-procedure. 

Studies in additional countries will commence once national regulatory approvals have been received and sites selected and engaged.

Trial Demonstrates Statistically Significant Improvements in Heart Function and Exercise Tolerance with Cardiopoietic Stem Cell Therapy

Mont-Saint-Guibert, Belgium, 11 April, 2013 – Cardio3 BioSciences (C3BS) announces today the advanced publication of C-CURE (Phase II) trial results in the on-line edition of the Journal of the American College of Cardiology (JACC).

The publication reported:

• Statistically significant improvement in cardiac function for the treated patients,
• Statistically significant improvement in 6-minute walk distance for the treated patients.

The publication concluded that the therapy with C3BS-CQR-1 (previously C-Cure) was feasible and safe with signals of benefit in chronic heart failure, meriting further definitive clinical evaluation.

The C-Cure trial was a prospective, multicenter, randomized study to evaluate the feasibility, safety, and efficacy of CQR-1 in the treatment of patients with chronic heart failure secondary to ischemic cardiomyopathy. CQR-1 consists of the patient’s own stem cells harvested from the bone marrow and engineered to become progenitors of new functional cardiac cells. Those cells behave identically to the cells lost to heart disease. In the C-Cure trial, all patients received optimal standard-of-care for heart failure, while treated group also received an intra-myocardial injection of CQR-1.

On the basis of these outcomes, C3BS has initiated a Phase III trial for CQR-1, called CHART-1 for Congestive Heart failure Cardiopoietic Regenerative Therapy. This is the first Phase III trial using organ specified cells for the treatment of ischemic heart failure and will recruit approximately 240 patients, with chronic advanced symptomatic heart failure underlining Cardio3 BioSciences’ dedication and leadership in bringing regenerative therapies to patients. The primary endpoint of the trial integrates cardiac and clinical endpoints as recommended by the European Medicines Agency.

Dr Jozef Bartunek, Principal Investigator, said: “The Phase II trial demonstrates that cardiopoietic stem cell therapy is feasible, safe and with strong signals of efficacy. These results highlight the promise of such novel technology for optimized regenerative intervention in heart failure, bringing next generation therapies to patients. With Cardio3 BioSciences having started the follow-up Phase III trial, the cardiopoietic approach is at the forefront of this exciting field.”

Dr Christian Homsy, CEO of Cardio3 BioSciences, added: “Publication of the C-Cure trial results in a journal as prestigious as JACC highlights the quality of the science underlying our lead product, CQR-1. We look forward to confirming the promising Phase II results, in our Phase III trial. Worldwide, this is the first pivotal Phase III study assessing such advanced regenerative product forthe treatment of heart failure. We believe that the innovative science behind our product has the potential to revolutionize the treatment of this debilitating disease.”

Prof. Dr. André Terzic, lead regenerative medicine specialist at Mayo Clinic in Rochester (MN), USA and Co-Principal Investigator of the C-Cure Clinical Trial, commented: “Heart failure is a major global challenge with the aging of the population and the shortage of donor organs. By introducing lineage guidance into the cell therapy protocol, the C-CURE trial provides initial clinical evidence for a new approach in cardiovascular regenerative medicine. Clinical translation of cardiopoietic stem cell therapy indicates favorable impact on myocardial remodeling, left ventricular ejection fraction, and global wellness. The C-CURE trial thus advances the paradigm of stem cell therapy, providing a rationale for further clinical validation.” 

Mont-Saint-Guibert, Belgium, November 22, 2012 –The Belgian biotechnology company, Cardio3 BioSciences (C3BS), a leader in the discovery and development of regenerative and protective therapies for the treatment of cardiac diseases, today announces it has received authorization from the Belgian Federal Agency for Medicines and Health Products (FAMHP) to begin its Congestive Heart failure Cardiopoietic Regenerative Therapy (CHART-1) European Phase III trial for C3BS-CQR-1 in Belgium. This represents a world premiere for a regenerative medicine product targeting heart failure to be tested in the context of a Phase III trial. C3BS-CQR-1 is an autologous stem cell therapy for heart failure. 

The Phase III trial is a prospective, multi-centre, randomized, sham-controlled, patient-and evaluator-blinded study comparing treatment with C3BS-CQR-1 to a sham treatment. The trial will recruit a minimum of 240 patients with chronic advanced symptomatic heart failure. The primary endpoint of the trial is a composite endpoint including mortality, morbidity, quality of life, Six Minute Walk Test and left ventricular structure and function at 9 months post-procedure. 

Studies in additional countries will commence once national regulatory approvals have been received.

The Cardio3 BioSciences therapy, called C3BS-CQR-1, involves taking stem cells from a patient’s own bone marrow and through a proprietary process called Cardiopoiesis, re-programming those cells so that they go onto becoming heart cells. The cells, known as cardiopoietic cells, are then injected back into the patient’s heart through a minimally invasive procedure using a catheter called C-Cathez ® , with the aim of repairing damaged tissue and improving heart function and patient clinical outcomes. C3BS-CQR-1 is the outcome of multiple years of research conducted at Mayo Clinic (Rochester, Minnesota, USA), Cardio3 BioSciences (Mont-Saint-Guibert, Belgium) and Cardiovascular Centre in Aalst (Aalst, Belgium). This Phase III trial builds on the successful outcome of the Phase II trial conducted between 2009 and 2010 in multiple clinical sites in Belgium, Serbia and Switzerland.

Dr Christian Homsy, CEO of Cardio3 BioSciences, said: “Heart failure remains a significant unmet clinical need associated with high morbidity, mortality and escalating healthcare costs. We believe C3BS-CQR-1 has a potential to become an alternative to heart transplantation which is the only curative treatment for heart failure available today. Our Phase III trial is the first to begin anywhere in the world for a regenerative therapy for this indication. We look forward to confirming the promising results we have already seen in our Phase II study.”

Dr Jozef Bartunek, Principal Investigator, added: “Regenerative therapies have the potential to revolutionize the treatment of heart disease and other conditions and we are excited to be working with Cardio3 BioSciences on what is a ground breaking study. The results seen in the earlier trials were encouraging and if repeated in this larger study would bring nearer a potentially disruptive treatment for the expanding epidemic of heart failure.”

Cardio3 BioSciences has completed a Phase II trial with C3BS-CQR-1, which demonstrated that heart failure patients improved heart function and exercise capacity following treatment compared to patients receiving current standard of care. Moreover, feasibility and safety of the C3BS-CQR-1 treatment regimen were established. 

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Innovative Heart-Specified Stem Cell Therapy Opens a New Chapter in Cardiovascular Regenerative Medicine

Mont-Saint-Guibert, Belgium, May 16, 2011 – The Belgian biotechnology company, Cardio3 BioSciences, a leader in discovery and development of regenerative and protective therapies for the treatment of cardiovascular diseases, will present Phase II clinical trial data of the flagship product C3BS-CQR-1 (C-Cure®), at the EuroPCR Meeting in Paris on Wednesday, 18th May. This is the First-in-man use of lineage specified stem cells for the treatment of heart failure.

On behalf of the C-Cure international program, data will be presented by Dr. Dariouch Dolatabadi, Civil Hospital, Charleroi, Belgium and Investigator of the C3BS-CQR-1 (C-Cure) trial.

The trial demonstrates that heart failure patients improved heart function and exercise capacity at 6 months following treatment of C-Cure, an innovative stem cell therapy based on the Company’s “Cardiopoiesis” proprietary technology. Moreover, feasibility and safety of the C-Cure treatment regimen were established.

Data strongly suggest that C-Cure is a promising treatment for heart failure, one of the world’s greatest unmet medical needs. A person living to the age of 40 has a one-in-five risk of developing heart failure and, once the disorder is apparent, a one-in-three chances of dying within a year of diagnosis. 

More detailed information on www.c3bs.com/fr/products-pipeline/clinical.html

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Innovative Heart-Specified Stem Cell Therapy Improves Heart Function and Fitness in Heart Failure Patients

• C-Cure is the next generation heart-specified regenerative product developed from the patients’ own bone marrow cells to enable heart repair
• At 6 months post-therapy, C-Cure treated heart failure patients walked 73 meters more than patients that received optimal standard of care
• C-Cure therapy improved heart function with an increase in left ventricular ejection fraction of 18.1%

Mont-Saint-Guibert, Belgium, April 5, 2011 – The Belgian biotechnology company, Cardio3 BioSciences, a leader in discovery and development of regenerative and protective therapies for the treatment of cardiovascular diseases, today revealed detailed data from the Phase II clinical trial of its novel stem cell therapy for heart failure, C3BS-CQR-1 (C-Cure®), at the 60th annual American College of Cardiology in New Orleans, USA. This newest innovation builds on a proprietary “cardiopoietic platform”, and the ongoing collaborations with investigators at Mayo Clinic and Aalst Cardiovascular Center.

The clinical study was presented by Dr. Jozef Bartunek, Associate Director of the Cardiovascular Center in Aalst, Belgium and Co-Principal Investigator of the C-Cure trial. The results show that, beyond existing best standard of care, patients saw improvements in heart function and exercise capacity when treated with C-Cure. This novel stem cell therapy involves taking a patient’s own bone marrow stem cells and guides them to repair heart tissue when introduced into a damaged heart area.

Heart failure affects over 117 million people worldwide, and cannot be cured today by currently available therapies. Regenerative therapies, such as C-Cure, offer the potential of a lifesaving treatment providing choice to patients with limited options and potentially avoiding the need for heart transplantation.

The Phase II trial recruited 45 patients with severe heart failure of ischemic origin in Belgium and Serbia who were treated with optimal standard of care (Control group) or optimal standard of care plus C-Cure (C-Cure group). 

Patients receiving C-Cure saw an 18.1% increase in left ventricular ejection fraction (LVEF), a measure of heart function, over baseline, as measured by echocardiography, while the mean LVEF improved only marginally in patients enrolled in the control group. This difference in LVEF between the C-Cure treated and control patients was significant (p

Importantly, signs of functional heart improvement were supported by improved fitness, shown by a clinically meaningful mean difference in the 6-minute walking distance test between the C-Cure treatment and control groups. After 6 months, patients treated with C-Cure were able to walk an average of 52 metres further in six minutes whereas control patients treated with current optimal standard of care were able to walk 21 metres less on average. The difference of 73 meters between both groups suggests heart failure patients treated with C-Cure could expect to return to a more active lifestyle, as they recover their ability to perform daily activities.

Dr. Jozef Bartunek explained: “Data presented today strongly suggest that C-Cure is a promising treatment for heart failure, one of the world’s greatest unmet medical needs. A person living to the age of 40 has a one-in-five risk of developing heart failure and, once the disorder is apparent, a one-in-three chances of dying within a year of diagnosis. With the C-Cure trial, we show improved left ventricular and clinical performance consistent with a generalized therapeutic benefit. Moreover, we proved feasibility and safety of the C-Cure treatment regimen. The overall signs of efficacy in C-Cure treated patients are indeed encouraging and open a new chapter in cardiovascular regenerative medicine.”

Dr. Christian Homsy, CEO of Cardio3 BioSciences, added: “We are very pleased to be presenting this important data at one of the most prestigious cardiology conferences. The positive outcome of the C-Cure study reiterates our belief that C-Cure treatment using heart-specified stem cells can make a real difference to patients suffering from heart failure. We are currently planning the next stages of product development, and are committed to taking the steps needed to successfully bring this new and important treatment to patients with heart failure, a condition where current therapies do not address the underlying cause of disease.”

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