Promethera Biosciences Receives Approval in Belgium to Enroll Patients in the Imminent HEP002 Phase 2b/3 Clinical Trial

Promethera Biosciences, a Belgian biotechnology company developing Promethera® HepaStem, a cell-based therapy for the treatment of both orphan liver-based metabolic diseases and acquired liver diseases, today announces that Belgium has authorized the conduct of a trial designed to treat paediatric patients with urea cycle disorders (UCD) in a Phase IIb/III study (HEP002).

This first agreement officially opens the start of the second clinical trial conducted by the company. Belgian patients will be recruited and treated in Cliniques Universitaires Saint-Luc in Brussels.

Most young patients affected by these very rare diseases have limited therapeutic options and may die at an early age. UCD are a group of eight inborn errors of metabolism that affect the transfer of nitrogen into urea. Although each specific disorder results in the accumulation of different precursors, hyperammonemia and hyperglutaminemia are the common biochemical hallmarks of these disorders. UCD are rare diseases, with an incidence between 1 in 8,000 and 1 in 44,000.

HEP002 is a prospective, open label, multicenter, efficacy and safety study with Promethera® HepaStem. The primary objective is to assess the efficacy of Promethera® HepaStem in patients with urea cycle deficiencies during the year following treatment. In the first clinical trial HEP001 Promethera® HepaStem demonstrated to be a safe treatment that can result in functional metabolic improvement in UCD patients.

“We are excited to pursue the development of this promising disease-modulating therapy.  It is urgently needed by the children living with these disabling metabolic diseases,” said Dr. Béatrice De Vos, Promethera Biosciences’ chief medical officer.

Professor Etienne Sokal, chief scientific officer and founder of Promethera Biosciences, paediatric hepatologist at Cliniques Universitaires Saint Luc and director of Cell Therapy Research Lab (in Belgium) said: “This is a new important achievement in the process of transferring original clinical research to industry and giving access to this innovative therapy to the maximum number of children.”

Eric Halioua, chief executive officer and co-founder of Promethera Biosciences said: “Obtaining the first authorization for our Phase 2b/3 trial in Belgium is a new major milestone reached by the company. Only five years since the company started we are about to begin our second clinical trial.”

About the ongoing clinical trials

Investigational study, Phase 1/2 (HEP001): Promethera Biosciences is currently completing a multicenter Phase 1/2 clinical study in five countries (Belgium, France, United Kingdom, Italy and Israel) involving 20 patients suffering from Crigler-Najjar syndrome and Urea Cycle Disorders and treated with HepaStem. Both pathologies are liver based inborn errors of metabolism. As of today, all patients have been treated and are in the follow-up period.

Observational study (OBS001): OBS001 is a retrospective and prospective observational study for paediatric patients suffering from Urea Cycle disorders or Crigler-Najjar syndrome. Patients are followed prospectively for a maximum of four years. This multicenter study is currently conducted in Belgium and France.

Long-term safety study (SAF001): This trial is proposed for patients having completed a study with HepaStem. The main objective of the SAF001 is to document the safety of HepaStem for a period of four years. The SAF001 protocol has been approved in Belgium, France, Italy, United Kingdom, Israel and Portugal.

About Promethera Biosciences:
Promethera Biosciences is a clinical stage pharmaceutical company that develops innovative therapies for the treatment of liver diseases. In addition, it is currently developing two products based on human liver progenitor cells:

Promethera® HepaStem is a cell therapy product based on the use of allogeneic stem cells isolated from healthy adult human livers (Heterologous Human Adult Liver Progenitor Cells, HHALPC). These cells can be used to treat a wide variety of liver diseases, from rare inborn metabolic diseases (which can be classified as ‘orphan diseases’ and mainly affect children), to acquired deficiencies affecting adults, such as fulminant hepatitis or liver fibrosis. This treatment has already received the orphan drug designation from the European Medicines Agency for the treatment of two very debilitating paediatric pathologies and from the Food and Drug Administration (USA) for both indications.

Promethera® H2Screen & H3Screen is a non-therapeutic in vitro product, based on a recently discovered and patented liver progenitor cell type, isolated from healthy adult human livers. This novel biotechnology tool is designed for early preclinical pharmaco-toxicological evaluation of drug candidates and new chemical entities.

Promethera Biosciences is a spin-off of the Université Catholique de Louvain (UCL), founded in 2009. Promethera Biosciences is located in Mont-Saint-Guibert, Belgium.

Since its creation in 2009, Promethera Biosciences has raised EUR 39 million in two financial rounds; in capital, grants and loans from the Walloon Region. The main investors are Vesalius Biocapital, Mitsui Global Investment, Boehringer Ingelheim Venture Fund, Shire and SRIW.

Promethera Biosciences has 44 employees led by an experienced management team.

For more information, please visit http://www.promethera.com