Promethera Biosciences Opens the French Part of its Clinical Trial in Cell Therapy

Promethera Biosciences has announced that it has successfully treated a patient with liver progenitor cells enrolled in its Phase I/II trial. The intervention was performed in January 2013 at the University Children’s Hospital Jeanne de Flandre in Lille, France.

Promethera Biosciences is currently conducting a Phase I/II clinical study involving Crigler-Najjar syndrome and Urea Cycle Disorders patients that has been approved by FAMHP (Belgian Authorities), MHRA (British Authorities), ANSM (French Authorities) and ISS (Italian Authorities).

This patient is the first one treated in France and the first one treated outside of Belgium where the study was launched.

The trial, a prospective, open label, multicenter Phase I/II study is a dose escalation study designed to evaluate the safety and the preliminary efficacy of Promethera® HepaStem in Urea Cycle Disorders and Crigler-Najjar Syndrome pediatric patients.

These diseases are extremely rare. Most young patients have only limited therapeutic options and may die at an early stage.

On January 25, 2013, a 14-year-old adolescent suffering from Ornithine Transcarbamylase Deficiency (OTCD, one of the Urea Cycle Disorders) was treated with liver progenitor cells. The patient is the first to have been treated in France.

The administration of the cells took place at Hospital Jeanne de Flandre in Lille, France. It was coordinated by the reference center of Inherited Metabolic Diseases of Child and Adulthood of Dr. Dries Dobbelaere in collaboration with the Paediatric Clinical Investigation Center of Prof. Frederic Gottrand.

The treatment was successfully applied over three days. The cell formulation was performed both on clinical site in Promethera’s mobile formulation unit and at the premises in Mont-Saint-Guibert, Belgium.

The patient was discharged in good condition and is doing well. He is being closely monitored by Dr Dobbelaere’s medical team.

Administration of Promethera® HepaStem was already successfully performed in four more patients at the Cliniques Universitaires Saint-Luc in Brussels, Belgium.

The safety data collected so far supports the good tolerability of Promethera® HepaStem in the phase I/II clinical study and warrants further enrolment of new patients.

“We expect that this innovative treatment will address an important unmet need for physicians and patients. It will improve their quality of life and ease the daily disease management,” says Dr Dries Dobbelaere.

“We are very pleased with the progress of this Phase I/II trial. With the treatment of a patient abroad, using our mobile formulation unit, we have demonstrated the feasibility of such a treatment anywhere in Europe,” says Eric Halioua, co-founder and chief executive officer of Promethera Biosciences.

Professor Etienne Sokal, co-founder of Promethera Biosciences and chief scientific officer, paediatric hepatologist at Cliniques Universitaires, Saint Luc, Belgium and director of UCL’s Cell Therapy Research Lab, Belgium adds: “With this first patient treated outside the founding clinical site, Promethera Biosciences has shown that liver stem cell therapy could become a widely accessible medicinal therapy, which can be applied for all patients in need within their own metabolic treatment center.”

Dr. Beatrice De Vos, chief medical officer of Promethera Biosciences concludes: “A close and profound collaboration between the clinical teams in Brussels, in Lille and at Promethera is the cornerstone of this successful outcome. We are very keen to share this expertise with other centers in Belgium, France, UK and Italy participating in this trial.”

The clinical centers in UK and Italy are preparing to welcome the next patients for this clinical trial.

The major innovation of Promethera® HepaStem is in the simplicity of the treatment - a simple injection into the vein leading to the liver. Promethera® HepaStem could be used to treat a wide variety of liver pathologies.

Thanks to the industrial development, the potential for treating hundreds of patients from a single liver could become a reality.