Slide 1: Shifting the drug discovery paradigm
Shifting the drug discovery paradigm Evotec and Celgene in exclusive drug discovery collaboration for neurodegenerative diseases Evotec AG, Investor and Analyst call, 16 December 2016
Slide 2: Forward-looking statement
Forward-looking statement Information set forth in this presentation contains forward-looking statements, which involve a number of risks and uncertainties. The forward-looking statements contained herein represent the judgement of Evotec as of the date of this presentation. Such forward-looking sta
Slide 3: iPS cells – A paradigm shift in drug discovery
iPS cells – A paradigm shift in drug discovery Evotec‘s industrialised iPSC infrastructure represents one of the largest and most sophisticated iPSC platforms in the industry
Slide 4: Agenda
Agenda Rationale for next generation drug discovery Paradigm shift with iPS cells (“iPSC”) Evotec and Celgene: Alliance in neurodegeneration Next Steps PAGE 3
Slide 5: Improving R&D productivity in the pharmaceutical industry is imperative
Improving R&D productivity in the pharmaceutical industry is imperative Development costs increase while average peak sales decrease Cost, USD billion Sales, USD million 1,576 816 1,188 +33% 416 -49% 2010 2015 2010 2015 Cost to develop an asset has increased by Average peak sales per asset have halv
Slide 6: Patient centric approaches improve success rates
Patient centric approaches improve success rates Human genetics and biomarkers are improving the odds of success Human genetics supported targets 1) Biomarker based patient stratification 2) % success % success 30 30 ~3x 25 ~2x 25 20 20 15 15 10 10 5 5 0 0 All programs Genetically supported No bioma
Slide 7: Fully integrated iPSC-based drug discovery platform
Fully integrated iPSC-based drug discovery platform The Evotec iPSC accelerator platform iPSC Patient-derived Hit H2L/LO In vivo culture disease models identification pharmacology Access to SC-derived High-content Cellular assays PK/PD iPSC lines disease-relevant screening Medicinal Bi
Slide 8: Latest stage technologies open new horizons
Latest stage technologies open new horizons Combining human genetics with disease relevant read-outs Combination of break- through technologies enable a new era in drug discovery Patient derived cell- based assay systems (e.g. iPSC) Gene editing (e.g. CRISPR) Cellular molecular analysis (e.g.
Slide 9: Agenda
Agenda Rationale for next generation drug discovery Paradigm shift with iPS cells (“iPSC”) Evotec and Celgene: Alliance in neurodegeneration Next Steps PAGE 8
Slide 10: Only few moments in science are undisputed as genuine and elegant
Only few moments in science are undisputed as genuine and elegant in creativity and simplicity Shinya Yamanaka – Discovered iPSC in 2007 – Nobel prize in Medicine 2012 “The induced pluripotent stem cells created by Shinya Yamanaka will allow us to interrogate and understand the full extent and varia
Slide 11: iPSC technology shifts the drug discovery paradigm
iPSC technology shifts the drug discovery paradigm in drug discovery as well as cell-based therapy Advantages & hurdles with iPSC Drug discovery Cell therapy Focus on human disease relevance Autologous cell therapy can circumvent already at screening stage immune rejection Replacement of roden
Slide 12: iPSC-based drug discovery platform has been established over the last 5 years
iPSC-based drug discovery platform has been established over the last 5 years Key elements of drug discovery process with iPSC Disease-specific drugs High quality iPSC cultures at large scale (QC) Industrial processes for Screening Patient generation of patient-derived target cell types Robust
Slide 13: Focus on human disease relevance from the start
Focus on human disease relevance from the start iPSC will shift the drug discovery paradigm Disease relevance Key Advantage OLD NEW with iPSC Patient-derived disease models for In vitro models for drug Low High drug screening and profiling confers screening / profiling disease relevance early on Eff
Slide 14: Initial focus on key neuronal cell types
Initial focus on key neuronal cell types From prototype protocol to industrial process Motor neurons Amyotrophic lateral sclerosis iPSCs Cortical neurons Huntington‘s disease Frontotemporal dementia Alzheimer’s disease Ectoderm Mesoderm Endoderm Germline Brain Muscle Pancreas Sperm D
Slide 15: Patient derived functional neurons
Patient derived functional neurons Manufacturing process adapted to 384-well format suitable for HCS PAGE 14
Slide 16: Industrialised process to generate patient neurons in 384 well format
Industrialised process to generate patient neurons in 384 well format suitable for HCS Highly robust, semi-automated human neuron manufacturing process Low inter/intra inter / intraplate platevariability variability Low Low day-to-day day-to-day variability variability 64% 384-well format PAGE
Slide 17: Unprecedented performance indicators in iPSC-derived human neuron assays
Unprecedented performance indicators in iPSC- derived human neuron assays Excellent screening performance indicators throughout screening campaign Pilot screening 0.84 Z’ Ø 0.7 Motor neuron yield ~55% Primary screening Z’ Ø 0.8 Z’ Primary screening plates Motor neuron yield ~60% in chronological
Slide 18: Highly robust manufacturing process works for all lines tested so far
Highly robust manufacturing process works for all lines tested so far General applicability of human neuron manufacturing process Patient A Patient B Patient C Patient D Patient E Healthy subject PAGE 17 Images obtained with Phenix at 20x magnification
Slide 19: Early patient stratification via 'clinical trial in a dish'
Early patient stratification via ‘clinical trial in a dish’ Patient-derived iPSC-lines available for multiple genotypes (e.g. ALS) PAGE 18
Slide 20: Focus on genetically defined subpopulations of widespread diseases
Focus on genetically defined subpopulations of widespread diseases Many opportunities beyond neurodegeneration focus Neurodegenerative diseases Lysosomal storage diseases Alzheimer’s disease Gaucher, Fabry Parkinson’s disease Batten, MLIV1) Amyotrophic lateral sclerosis Huntington diseas
Slide 21: Agenda
Agenda Rationale for next generation drug discovery Paradigm shift with iPS cells (“iPSC”) Evotec and Celgene: Alliance in neurodegeneration Next Steps PAGE 20
Slide 22: Clear strategy towards global leadership in iPSC-based drug discovery
Clear strategy towards global leadership in iPSC- based drug discovery From scientific discovery to strategy 2025 2016 2020 2012 Initiation of Broadening of pipeline Outlook iPSC platform and first strategic deals Strategic platform development into CureMN (ALS / Build > 50 scientists and othe
Slide 23: Neurological diseases will remain a serious threat to public health
Neurological diseases will remain a serious threat to public health Societal burden of CNS related diseases is daunting1) Why CNS diseases? Why Why patient derived ? Constitute the biggest neurodegenerative iPSC technology is health care challenge diseases (ND)? uniquely suited to Dementia account
Slide 24: Global neurodegenerative disease market
Global neurodegenerative disease market Market overview in mio USD 35,000 6.5% 33,042.95 31,143.58 6.10 6.0% 30,000 29,553.94 28,225.42 27,118.93 5.38 5.5% 26,177.20 25,000 5.0% 4.71 4.5% 20,000 4.08 4.0% 15,000 3.5% 10,000 3.0% 2.5% 2.56 5,000 1.93 2.0% 0 1.5% 2014 2015 2016 2017 2018 2019 Revenue
Slide 25: Major exclusive alliance in Neurodegeneration
Major exclusive alliance in Neurodegeneration Celgene & Evotec Mission Develop novel therapies for a broad range of neurodegenerative diseases, based on Evotec’s unique patient-derived drug screening platform (“iPSCs”) Strategic alliance Celgene & Evotec Upfront payment of $ 45 m Potential mil
Slide 26: Agenda
Agenda Rationale for next generation drug discovery Paradigm shift with iPS cells (“iPSC”) Evotec and Celgene: Alliance in neurodegeneration Next Steps PAGE 25
Slide 27: Last tick for 2016 done – A strong 2017 ahead of us
Last tick for 2016 done – A strong 2017 ahead of us EVT Execute & EVT Innovate – Expected key milestones 2016 New clinical initiations and good New long-term deals with large progress of clinical pipeline within and mid-sized Pharma partnerships Expansion of foundations and biotech Expansion
Slide 28: Research never stops
"RESEARCH NEVER STOPS’