The promise of cell therapy for liver diseases: hurdles and success in drug development Beatrice De Vos, MD, PhD, BCPM (CMO PB)

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1 The promise of cell therapy for liver diseases: hurdles and success in drug development Beatrice De Vos, MD, PhD, BCPM (CMO PB) Research Symposium on digestive and liver diseases European Parliament, Brussels September 18, 2012 Tel:

2 Promethera Biosciences is a cell therapy company dedicated to the treatment of liver diseases using an in vitro expanded cell. 2 ONE SINGLE MISSING ENZYME CAN HAVE A DRAMATIC CONSEQUENCE ON THE QUALITY OF LIFE INBORN ERRORS OF LIVER METABOLISM : Orphan diseases: < 1/5000 to 1<2000 Most young patients have only very limited therapeutic options and may die at an early age Only a few % more of the missing enzymatic activity makes a dramatic difference in the quality of life

3 From organ transplantation to advanced therapy Liver transplantation Liver cell transfusion Somatic cell therapy medicinal product Industrial processing 3

4 Promethera Biosciences Proprietary Technology: Hundreds of patients can be treated with one liver. Raw Material DRUG SUBSTANCE DRUG PRODUCT INJECTION Normal Adult Human Liver Human Hepatocytes HHALPC in culture Larger Volume of HHALPC HepaStem final product Intraportal Injection into Patient Isolation of human hepatocytes from the liver Enzymatic and mechanical dissociation of parenchymal cells Purification of HHALPC (1) from mature liver cells In vitro expansion Cell resistant to cryopreservation Washing, formulation and final packaging of HepaStem Optimized system of cryopreservation / storage and quality control 4 Treatment with healthy liver cells can potentially cure any single liver-based inborn error of metabolism, as each cell contains all liver enzymes required for normal functioning *Note: Method subject to an international patent application (1) hhalpc = Heterologous Human Adult Liver-derived Progenitor Cells

5 Promethera Biosciences develops two products with the same technology: HepaStem and HepaScreen. HepaStem Innovative medicinal products to treat miscellaneous liver diseases Orphan status obtained from EMEA and FDA Proof of concept completed and three patients injected HepaScreen In vitro tool for toxicity and metabolic studies for new chemical entities Unlimited access to the cell product Used by Pre-clinical departments of Pharmaceutical companies Spin-off from the UCL (Université Catholique de Louvain) lab of Professor Etienne Sokal, one of the leading experts in hepatic cell transplantation 5

6 Acquired liver disorders Liver based Metabolic Diseases Promethera Biosciences Pipeline. Indications Research & Development Pre-clinical Phase I/II Phase II/III Next steps Crigler-Najjar Urea Cycle diseases Start phase I/II pediatrics in Q Start phase I/II pediatrics in Q HepaStem Phenylketonuria Glycogene Storage Disease Type-1 Start exploratory trial in adults in 2013 Start exploratory trial in adults in 2013 Liver Fibrosis Start animal tests Q HepaScreen Product Cell Based Assay Development/ Characterization Cell-based Assay Optimisation Market Next steps Full repertoire of metabolic activities 6

7 Major achievements since the company creation in 2009 Achievement of key milestones nearly a year ahead of the initial schedule First clinical trial authorization by regulatory authorities in Belgium and the UK for HepaStem for the treatment of Crigler-Najjar syndrome and urea cycle disorders in a paediatric setting Between 2009 and 2011, injections of HHALPC 1 safely administered to three patients - a world first - at Cliniques Universitaires St Luc by Professor Sokal and his team. Proof of concept for the therapeutic use of the cells has been demonstrated in three different animal models. Successful technology transfer of manufacturing process from the University into Promethera GMP facilities and manufacturing of 46 HepaStem batches. The patent protecting the HHALPC 1 cell and all of its applications officially granted in Europe in February ) hhalpc = Heterologous Human Adult Liver-derived Progenitor Cells

8 Promethera Biosciences closed in 2012 a 2 nd round of financing of 23,6M : 17M in equity & 6.6M in loan (Walloon Region). New investors: international pharmaceutical groups and investment funds Mitsui Global Investment, a subsidiary of Mitsui & Co. Ltd. which invests in opportunities that have potential for growth. Shire Strategic Investment Group, Shire s corporate venture vehicle. Boehringer Ingelheim Venture Fund, BI Corporate fund created in March ATMI LifeSciences, a leader in single-use technologies, products and systems for mixing, storage, bioreactors, cell culture technology. Sambrinvest, an actor in risk capital in the region of Charleroi in Belgium Existing investors: national and private investment funds Vesalius Biocapital, Société Régional d Investissement Wallonne (SRIW), NivelInvest / Start-up SA Capital & Croissance/Financière Spin-off Luxembourgeoise (Invest Sud), Life Science Research Partner 8

9 Conclusion Successes Hurdles 9

10 10 Promethera Biosciences : staff has grown from 2 in 2009 to 46 in 2012 Thank you