CellProthera begins long-term observational study to demonstrate safety and efficacy of ProtheraCytes® cell therapy in acute myocardial infarction

The long-term PERFECT observational study follows patients from successful Phase I/IIb of ProtheraCytes for the repair and regeneration of damaged cardiac tissues after severe heart attack. 

Mulhouse, France, July 31, 2024 – CellProthera, a regenerative cell therapy company developing breakthrough treatments for ischemic diseases, has today announced the commencement of the ‘PERFECT’ study, a long-term follow-up observational study of the patients involved in the recent Phase I/IIb EXCELLENT trial, following success with patients having received ProtheraCytes after severe heart attack in the initial trial.

This new PERFECT observational study is expected to demonstrate the long-term safety and efficacy of ProtheraCytes for the regeneration and repair of cardiac tissue following acute myocardial infarction, on top of optimal standard of care.

In May, CellProthera announced positive results from the EXCELLENT trial, where the ability of ProtheraCytes to repair and regenerate heart tissue was observed with positive changes in prognostic markers including infarct size, left ventricular dimensions and NT-proBNP level, supporting the cell therapy’s anti-remodeling effect.

To confirm the long-term efficacy and safety of treatment with ProtheraCytes, the team will closely monitor the progression of biological markers, such as NT-proBNP, and evaluate the patient's clinical status over a period of 10 years. These findings will then be compared with data from patients having received standard of care.

As Matthieu de Kalbermatten, CEO, CellProthera explained: “We look forward to the clinical data of this long-term follow-up study. After demonstrating very promising improvements in the efficacy endpoints at six months, suggesting our therapy’s ability to act against adverse left ventricular remodeling, we are eager to analyze its impact on the long-term clinical outcome of the patients. This is precious information that will strengthen the body of evidence in favor of our drug candidate.”

With today’s standard-of-care, the mortality rate from ischemic heart failure and other complications within the first year following an acute myocardial infarction remains high, ranging between 5–10%. ProtheraCytes offer a promising solution, enhancing revascularization, controlling inflammation, preventing cell death and promoting the regeneration of heart cells, thus paving the way for the repair of damaged heart tissue. 

The study is being conducted in 10 participating clinical centers across France and the UK, including University Hospitals of Toulouse-Rangueil, Montpellier, Bordeaux, Dijon, as well as general hospitals such as Jacques Cartier in Massy and Mulhouse Ghrmsa. The Royal Infirmary of Edinburgh, St Barts Hospital in London, Queen Elizabeth University Hospital of Birmingham and Dundee Hospital will also participate as soon as regulatory approval is effective.

“The patients we enrolled in the EXCELLENT trial had prognostic risk factors for developing severe heart failure symptoms in the years following acute myocardial infarction. Therefore, we are naturally interested in the long-term follow-up of these patients in order to observe the effect of ProtheraCytes on the progress of their health status. I can’t wait to see the results, which I hope will be positive given the results observed at six months,” added Professor Jerome Roncalli, Professor of Cardiology at CHU de Toulouse and Vice President of the Fédération Française de Cardiologie.

The next steps for the company will be to continue the clinical trial development of ProtheraCytes cell therapy in acute myocardial infarction by conducting a Phase III study with a larger cohort.

About CellProthera 

CellProthera is a regenerative cell therapy developer specializing in cardiovascular diseases with a leading program in myocardial infarction. CellProthera has developed a unique GMP-compliant cell expansion process as well as a proprietary automation technology for in vitro production of large quantity of purified, CD34+ stem cells. Its lead therapy ProtheraCytes™, is an autologous cell therapy and has been developed for body regeneration and targeted to regenerate various damaged tissues, including cardiac tissue. ProtheraCytes is registered as an Advanced Therapy Medicinal Product by the European Medicines Agency (EMA). CellProthera’s proprietary technology platform comprises an automated expansion device called StemXpand® and its disposable kit StemPack®. CellProthera is headquartered in France.