BIOCRATES Life Sciences AG Short Company Presentation Dr. Alexandra C. Gruber MBA MMFin Director Business Development / Marketing Austria Showcase Life Science Japan, Tokyo, December 2010 BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 1
Personalized Medicine: A Modern Topic Current Practice Personalized Medicine BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 2
Omics Technologies in Biomarker Discovery Metabolomic testing: examines the body s ability to use and excrete chemicals or metabolites (such as lipids and sugars) Proteomic tests: study the structure, chemical modifications and overall functions of proteins; can be used to investigate associations between various protein types and bodily levels and onset or progression of disease Transcriptomic tests: The study of transcriptomics, also referred to as expression profiling, examines the expression level of mrnas in a given cell population, often using high-throughput techniques based on DNA microarray technology Genetic tests: focus on how genes affect health status A systematic approach will involve multiple omic technologies, including whole genome association studies to identify causative mutations or polymorphisms as well as expression profiling, proteomics, and metabolomics to identify expression signatures and protein or small-molecule profiles that are either specific to a disease process or provide mechanistic insights into disease pathology. BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 3
Omics Technologies: Market Projections Biomarker markets according to technologies/applications 2008-2018 (Jain Pharma Biotech Report) Technology / applications Market in 2008 Market in 2013 Market in 2018 Biomarker Total Biomarker Total Biomarker Total Proteomics $ 2.0 billion $ 6.8 billion $ 3.3 billion $ 11.0 billion $ 5.7 billion $ 20.0 billion Metabolomics $ 2.4 billion $ 31.0 billion $ 3.8 billion $ 45.0 billion $ 5.8 billion $ 70.0 billion Mol Diagnostics $ 2.5 billion $ 7.6 billion $ 4.4 billion $ 14.0 billion $ 5.9 billion $ 37.0 billion Drug discovery $ 1.7 billion $ 37.0 billion $ 3.0 billion $ 41.5 billion $ 4.5 billion $ 58.0 billion Clinical trials $ 1.9 billion $ 53.0 billion $ 3.8 billion $ 63.0 billion $ 5.6 billion $ 78.0 billion Bioinformatics $ 0.5 billion $ 2.2 billion $ 0.7 billion $ 3.5 billion $ 1.5 billion $ 6.5 billion Total $ 11.0 billion $ 137.6 billion $ 19.0 billion $ 176.0 billion $ 29.0 billion $ 269.5 billion Metabolomics: the youngest omics discipline number of companies working in metabolomics is still rather small mass spectrometry is the leading technology for biomarker discovery in this field pharma companies integrate this new omic technology for stratifying patients in CTs Metabolomics is THE enabling technology for Personalized Medicine 2018: 5.8 $bn market for metabolomics based biomarker diagnostics BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 4
The Future of Diagnostics Follows a New Plan: BIOCRATES Tailored Biomarkers for Health. Socrates 470-399 BC Intelligence Wisdom Hippocrates 460-377 BC Medicine Health BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 5
Leader in Metabolomics Founded in 2003 in Innsbruck as university spin-off Mission: Discovering tailored biomarkers for health USP: Unique strength in accelerating biomarker discovery with experience in diagnostic product development Business model with attractive risk-return profile Strong IP situation (5 granted, 11 applied) and first mover advantage in multiple diagnostic marker field Established market player with relevant revenues (>3mUS$ in 2009) Solid investor base since the first round of financing Now taking final step of becoming a global leader in metabolomics based biomarker diagnostics BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 6
Proof-of-Concept in Metabolomics: Success Story of Neonatal Screening Newborn screening for inborn metabolic disorders replaced expensive monoparametric assays and broadened diagnostic portfolio simultaneous diagnosis of 20-30 monogenic diseases (AA metabolism, FATMO) with immediate treatment option total incidence > 1:2000 unprecedented sensitivity, specificity, and predictive values co-pioneered in the mid-90s by BIOCRATES founder Prof. B. Roscher > 1,500,000 newborns screened in Munich similar labs worldwide BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 7
Unique Technology Approach Sample cohorts Metabolic profiling (e.g. full scan LC-MS) Targeted metabolomics (ID / quantitation by MS/MS) Differential pattern information Metabolite concentration shifts Identification of relevant metabolites Functional annotation Targeted metabolomics: systematic identification and quantification of endogenous metabolites in bodily fluids and tissues >>> the quickest, most cost-effective way of identifying and quantifying biomarkers BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 8
Integrated Technology Platform Sample preparation Automated separation and derivatization SPE/HPLC Analytics Identification (MRM) Quantitation (SID) QA/QC Bioinformatics Technical validation Statistical analysis Data visualization Biochemical interpretation disease 2 disease 1 LIMS/Database BioBank Clinical & experimental samples Diagnoses & lab data BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 9
Business Model Contract Biomarkers IVD Products Kit Ted Metabolomics Platform BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 10
First Ever Metabolomics Kit Launched in 2008 1. Register the Assay in MetLIMS Import and register samples, generate 96-well plate overview and.csv file for Analyst Software 2. Assay Preparation Perform sample preparation in the laboratory 3. Process Assay in the Mass Spectrometer Analyze extracts in the MS instrument 4. Convert Mass Spectrometer Data Analyst.wiff files are converted and the concentrations are automatically calculated 5. Validate the Kit Plate Automated quality assessment of Standards, Quality Controls and Internal Standards 6. Evaluate and Export Data The results are evaluated and can be exported into other programs 7. Data Analysis Box plots, Scatter plots Univariate tests (pvalues) ROC curves Standardized quantitation of 163 metabolites, e.g. amino acids, acylcarnitines, (lyso-) phosphatidylcholines, sphingomyelins, hexoses First quantitative tool for basic and clinical research in metabolic diseases, phenotyping, association studies, pharmacodynamics, etc. Fully automated and supported by software to ensure data quality and reproducibility (validated according to FDA guidelines) First customers in research centers and industry: Helmholtz Center Munich, DSM, Nestlé, BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 11
Extension of the Kit Product Pipeline BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 12
Biomarker Pipeline: Products Focus and Potential Indication Current Diagnostics Significant Improvements Market Potential Chronic kidney disease (CKD) / Diabetic nephropathy (DN) Creatinine BUN estimated / calculated GFR Albuminuria Early diagnosis of kidney disease Individual prediction of progression 75 450 $m Metabolic Syndrome / Type II Diabetes (T2D) BMI, HWR Blood pressure HDL/LDL cholesterol Triglycerides HbA1c Individual risk assessment Differentiation of insulin resistance for individualized therapy 750-5,550 $m Sepsis Inflammation markers Coagulation markers Early detection host response Efficacy of antibiotic treatment 150 450 $m Stroke Imaging Functional examination On-site confirmation Differentiation hemorrhagic/ ischemic stroke 450 1,500 $m Metabolomic biomarkers have potential to revolutionize early diagnosis of diseases. Next set of biomarkers already in the pipeline: prostate cancer, breast cancer, steatohepatitis, COPD BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 13
Biomarker Discovery / Development Process Biomarker screening and prioritization of candidates completed Two candidates in internal validation Additional patents filed >>> Clear path towards significant value creation BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 14
Biocrates Partnership Opportunities in Molecular Dx Indication Setting Target Customer Products offered Commercialization Model Chronic Kidney Disease / Diabetic Nephropathy Metabolic Syndrome / T2D Sepsis Physician (GP / Specialist) & Hospital Physician (PG) ICU Big labs (hosp./commercial) All labs (hosp./commercial) & physicians Only larger labs (hosp./commercial) Mass Spectrometric Method Biocrates develops a method using Mass Spectrometry Immunoassays & ELISAs Service Supplier Biocrates runs assay In-house and others send their samples Product Supplier Biocrates develops kit product and sells to labs Out License Stroke Point of Care All labs (hosp./commercial) Point of Care Tests Tests are better suited for existing players to explot Biocrates collects fees &Royalties Biocrates Partner / licensee Nephrology & Diabetes: realize early revenues through mass spectrometry based assays (Dx and/or service) Sepsis & Stroke: partner with diagnostics companies for point-of-care tests BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 15
Summary - Biocrates` USP Technology of Biocrates Differentiation from Competition Automated mass spectrometry Standardized, quality-controlled, fully-integrated technology platform for targeted metabolomics Software to calculate metabolite concentrations from mass spectrometric data Targeted metabolomics know-how and experience First company offering a kit product for quantitative analysis of metabolites by mass spectrometry Quantitative analytical assays covering all main areas of metabolism Clinical expertise Ability to discover and validate new metabolite biomarkers fast and precisely UNIQUE POSITION through the combination of technologies and know-how at Biocrates BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 16
Thank you very much for your attention! Dr. Alexandra C. Gruber MBA MMFin Director Business Development / Marketing Tel. +43/512 57 98 23 4222 Mobile +43/676 84 843-4221 alexandra.gruber@biocrates.com www.biocrates.com BIOCRATES Life Sciences AG, Innrain 66/2, 6020 Innsbruck, Austria 17