Achievements

“First ever” injection of hALDMPC to three patients successfully performed at the Cliniques Universitaires Saint Luc of Brussels, Belgium.

Test doses of human adult liver stem cells hALDMPC (Heterologous Human Adult Liver-Derived Progenitor Cells) have already been injected in three patients at St Luc Hospital (Brussels, Belgium). The cells were produced in by the Liver Stem Cell Tissue Bank of Saint-Luc. 

The first patient was suffering from OTC (ornithine transcarbamylase deficiency) one of the 6 Urea Cycle defects, the second patient presented a Crigler-Najjar syndrome and the third patient was suffering from Glycogenosis type 1a.

The infusion of the liver progenitor cells occurred safely and was well tolerated by the patients.

The engraftment of the donor progenitor cells was evidenced in the recipient liver parenchyma by Fish technique on a liver biopsy 3 months post-infusion, and metabolic changes were detected.  

Achievements of the pre-clinical proof of concept in three different rodent models.

Two pre-clinical studies were undertaken in two different immunodeficient animal models with and without liver regeneration stimulus. These studies demonstrated that hALDMPC  (Heterologous Human Adult Liver-Derived Progenitor Cells), constitutive of Promethera^® HepaStem, are able to engraft and differentiate in the host liver parenchyma despite a xenogeneic environment. Similar results were obtained in both models meaning that a favorable niche for engraftment, as it is the case with liver regeneration stimulus, was not critical for the progenitor cells to engraft and differentiate.

A third study demonstrated the functional proof of concept through restoration of in vivo enzyme activity on Gunn rats, an animal model homologous to Crigler Najjar Syndrome. Gunn rats present a high level of unconjugated bilirubin in blood due to a mutation in UGT1A1 gene similar to the human condition. Transplanted Gunn rats responded to Promethera^® HepaStem therapy with a significant decrease of unconjugated bilirubin in blood after 6 months.

Successful technology transfer of manufacturing process from the Université Catholique de Louvain into a GMP-facility.

A comprehensive scale-up study of the manufacturing process has been performed and Promethera Biosciences has now the capability of producing progenitor cells in view of clinical trials to start beginning of 2012. Several batches have been produced, in the company’s 168m² GMP manufacturing unit (clean rooms), by Promethera Biosciences manufacturing team and successfully released by the Quality control team.

Patent granted by the European Patent Office in January 2011.

The first international patent application for hALDMPC has been submitted in December 2005. The patent (EP 1969118) has been granted by the European Patent Office in February 2011 protecting this valuable asset for the company. The patent is under review in US and other countries of the world.

Achievements of key Regulatory milestones and frequent interactions with the European and local regulatory bodies.

Promethera® HepaStem (under the name of Heterologous Human Adult Liver-Derived Progenitor Cells) was classified by the European Medicines Agency (EMEA) as a Medicinal Product in July 2007.

Orphan drug designation has been granted by the European Community for the treatment of Crigler-Najjar syndrome (Oct 2007) and for the treatment of Ornithinine Transcarbamylase deficiency, one of the six Urea Cycle disorders (Feb 2008).

Promethera Biosciences also received Scientific Advice from the EMA on the preclinical development of the product in March 2009 and on the quality of the product in June 2010.

Scientific Advice has been received from the Belgian authorities (FAMHP) on the preclinical development prior to phase I/II as well as on the quality of the product.

Finally, Promethera Biosciences has received Scientific Advice on the clinical development plan from the Paul Ehrlich Institute in Germany.

Closing in October 2009 of a 5.3 million euros A-round of funding complemented by more than 4 million euros of grants and loans from the Walloon Region.

Promethera Biosciences has been incorporated on February 2009. The company raises 5.3 million euros through series A capital round end October 2009 complemented by 4M€ of loans and grants from the Walloon region. Vesalius Biocapital is the lead investor of the company and is committed to support the company in Round-B

In January 2012, Promethera Biosciences represents an experimented and motivated team of 32 collaborators.