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Next Generation Therapies and Related Life Sciences Topics 1 Contents Next Generation Therapies and related Life Sciences topics 1 Contents 1 Overview of Deloitte’s Next Generation Therapy Practice 3 2 Overview of Next Generation Therapies 8 3 Gene Therapy 19 4 Challenges of Commercializing Gene Therapy 31 5 Gene Editing 35 6 Regenerative Medicine 47 7 Supply Chain/Commercialization 51 8 Appendix 1: Next Generation Therapies Deloitte Case Studies 58 9 Appendix 2: Deloitte’s Life Sciences M&A Opportunities 64 © 2020. For information, contact Deloitte Touche Tohmatsu Limited. Global Life Sciences and Healthcare 2 Overview of Next Generation Therapies © 2020 Deloitte Global Services Limited Global Life Sciences and Health Care | Practice overview 3 Evolution to Next Generation Therapies We are moving toward an ability to cure and heal rather than merely ameliorating symptoms Increasing Complexity Regulatory/Reimbursement of Therapeutic Intervention Supply Chain/Commercialization AI/Bioinformatics Modify Gene Gene Expression Correction • Antisense • Gene Therapy • RNAi/siRNA with Viral Vectors • Aptamers • Gene Therapy Polyclonal & • Ribozymes with Other Vectors Monoclonal • Exon Skipping • Gene Editing Antibodies (Meganucleases, ZFN, TALENs, CRISPR) Plasma & Recombinant Regenerative Proteins Medicine • Autologous & Allogenic Cells • Stem Cell Therapy Small Molecules • Mitochondrial Transfer & Infectious • Tissue Engineering Disease Vaccines © 2020. For information, contact Deloitte Touche Tohmatsu Limited. Global Life Sciences and Healthcare 4 What Makes Next Generation Therapies Different? Next gen therapies alter the healthcare ecosystem Next Generation Therapies Translational Medicine at AMC Biotech Discovery & Outlicensing Internal R&D Ultra Orphan & Accelerated Discovery Pathway Programs BLA/Orphan Indications New Drug Application (NDA) Value Based Billing & Regulatory Approval Pathway Individual Approvals Prior Authorization & Clinical Protocols Formulary & Tenders Individual Product Production Reimbursement Mechanisms Biological Batch Systems Chemical Synthesis Direct Shipment w/ Manufacturing Process Product COI & COC Specialty & Hospital Pharmacy w/ Batch COI & COC Retail Pharmacy w/Batch Certified & Trained Institutions Distribution Channels COI & COC Clinics & Hospitals Outpatient Therapy Delivery Medical Affairs Led Team Engaging Institution Specialty Sales Forces and P&T Committees Field Sales Force to High Prescribers Multiple Stakeholders through Commercial Focus on-line portals Conferences & Peer Reviewed Articles DTC/DTP Advertising, Long-Term Patient Registry Market Engagement Samples & Coupons EHR & REM/RMPs Rx Data & Adverse Event Reports Real World Evidence Capture © 2020. For information, contact Deloitte Touche Tohmatsu Limited. Global Life Sciences and Healthcare 5 Revenues of Currently Approved Next Generation Therapies Analysts anticipate a ~$9 Billion market for 48 approved products in the next 6 years WW Sales of Next Generation Therapies Number of Approved and Total = 48 (USD $MM) Marketed Next Generation Therapies $8,955 9000 $8,335 1,009 8000 889 $7,549 6% $6,742 7000 780 679 $5,676 17% 6000 4,235 3,847 585 5000 3,363 $4,472 2,790 Total = 487 4000 2,152 $3,263 1,509 48 3000 386 867 $1,739 2000 3,599 3,711 3,406 77% 300 3,273 2,939 400 2,476 1000 2,010 1,039 0 2017 2018E 2019E 2020E 2021E 2022E 2023E 2024E Gene Expression Gene correction Regenerative Medicine Source: Evaluate Pharma Analyst Consensus Projections; Annual Reports, Product websites and Deloitte Analysis Note: Gendicine and Oncorine are only sold in China with combined sales of USD ~$8 Million in 2017 are not included in revenue projections; Glybera was pulled from the market in 2017 and is not included. © 2020. For information, contact Deloitte Touche Tohmatsu Limited. Global Life Sciences and Healthcare 6 In the last three years, acquisitions worth over USD $100 Billion have demonstrated the market value of these new technologies M&A in Next Generation Therapies* Roche** (USD $ Billion) BMS Spark Celgene 5.0 1.0 113.2 74.0 10 Deals These 3 Acquisitions Accounted for more than USD $29.6 Billion Boehringer ViraTherapeutics PTC Celgene Agilis Takeda Merck Juno Viralytics Novartis Tigenix AveXis Gilead 9.0 .2 .2 Gilead Cell Design Allergan Kite LifeCell 8.7 .6 .4 (Tissue Matrices) 11.0 .2 2.9 Dec-16 Oct-17 Dec-17 Jan-18 Jan-18 Feb-18 Apr-18 Jul-18 Sep-18 Jan-19 Feb-19 Other Total Source: Company Websites & Press Releases Note:*Only upfront payments represented due to uncertainties about future milestone achievement and sales to generate royalties **Announced February 24, 2019 © 2020. For information, contact Deloitte Touche Tohmatsu Limited. Global Life Sciences and Healthcare 7 In Licensing of Next Generation Therapies The majority of value in licensing deals has been allocated to the back-end Total Value of Licensing* Major Licensing Deals in the Last 3+ Years (USD $ Millions) (USD $ Millions) Date Upfront Total Buyer Licensee Therapeutic Area Announced Payments Value Oct-18 J&J Arrowhead RNAi for Hepatitis B $235 $3,700 2015 2,216 Aug-18 Pfizer BioNTech mRNA flu vaccine $120 $425 Cell Therapy for Type 1 Apr-18 Eli Lilly Sigilon $63 $473 diabetes AAV Vectors for GT for Feb-18 Abbvie Voyager $69 $1,119 2016 647 Alzheimer's ZFN for Cancer Drug Feb-18 Gilead Sangamo $150 $3,000 Development Marketing Luxturna in Jan-18 Novartis Spark $105 $170 Europe 2017 1,086 BCMA-targeting CAR-T for Dec-17 J&J Legend $350 -- Multiple Myeloma May-17 Pfizer Sangamo ZFN for Hemophilia A $70 $545 Baxalta/ Precision Allogenic CAR-T using Arcus Feb-16 $105 $1,700 Shire Bioscience Platform 2018H1 914 Form JV (Casebia) to Dec-15 Bayer CRISPR $ 300 $335 develop CRISPR therapies Merck Mar-15 Intrexon CAR-T with non-viral vector $115 $941 Serono Total $1,682 $12,408 Source: Alliance for Regenerative Medicine; Company Websites & Press Releases Note: *Only upfront payments represented due to uncertainties about future milestone achievement and product sales to generate royalties © 2020. For information, contact Deloitte Touche Tohmatsu Limited. Global Life Sciences and Healthcare 8 Worldwide Growth in Next Generation Therapy Companies On a global basis there has been 15% annual growth over the last 3 years with North America and Europe hosting ~80% of Next Generation companies 2015Q1 580 2018H1 875 Europe & Israel 235 (2018H1) 166 (2015Q1) North America 466 (2018H1) 302 (2015Q1) Asia 135 (2018H1) 92 (2015Q1) South America 15 (2018H1) Africa Oceania 8 (2015Q1) 1 (2018H1) 23 (2018H1) (2015Q1) 12 (2015Q1) Source: Alliance for Regnerative Medicine Annual Report © 2020. For information, contact Deloitte Touche Tohmatsu Limited. Global Life Sciences and Healthcare 9 There is a significant opportunity in the China Market in the Next Generation Therapies Space USA and China are the leaders in the development of cell therapies A representation of clinical trials investigating cell therapies across major markets in the world Canada Europe Russia 18% 17% 16% 29% USA and China 5% 3% 18% are the leaders in the 40% 1% 36% 17 30 246 development of cell therapies 23% 13% 7% 35% 3% 14% 22% Stem cell & CAR-T therapies represent the largest share of the global cellular therapy market Japan USA 21% 33% USA has the largest number 6% 7% of Stem cell therapies under 1% 24 25% investigation (300+) followed 11% 734 4% 17% by EU (80+) 11% 41% 23% Southeast Asia Australia Middle East 9% 27% 10% 27% 12% 46% 11 15 10% 50 9% 7% 46% 68% 9% 20% • Stem cells • CAR-T cells • TCR-T cells • NK-T cells • TAA/TSA • TIL • Gamma Delta T Cells Data as of December 10, 2018 Note: The percentages indicate the proportion of studies for a certain intervention/therapy and the number inside the doughnut hole indicates the grand total of on-going studies registered on Clinicaltrials.gov © 2020. For information, contact Deloitte Touche Tohmatsu Limited. Global Life Sciences and Healthcare 10 Deloitte’s Experience with Next Generation Therapies Over the last 3 years Deloitte has completed over 20 engagement with clients which span the entire patient journey and industry value chain Next Generation Therapies Patient Journey 2 Enrollment 4 Treatment 6 Long-term • Confirm • Supportive care Follow Up patient • Community Post-Treatment 1 Referral eligibility 3 Evaluation 5 Physician Evaluation Physician initial • Patient • Patient education Monitoring assessment consent • Genetic eligibility • Observation • Patient registries and referral to • Outpatient/ treatment center inpatient care Value Chain Element Research & Development Supply Chain Commercial Representative Projects • Identification of new • Order to Cash • Pricing & Market Access viral vectors acquisition optimization for CAR-T targets for gene therapy • Chain of Identity (COI) • Development of • Risk analysis & mitigation analysis for European stakeholder portal strategy for Phase I launch for launch of CAR-T clinical trials • Supply chain assessment • Customer experience and playbook definition workshops for product launch © 2020. For information, contact Deloitte Touche Tohmatsu Limited. Global Life Sciences and Healthcare 11 Regulatory/Reimbursement Supply Chain/Commercialization Gene AI/Bioinformatics Modify Gene Gene Expression Correction Therapy • Antisense • Gene Therapy • RNAi/siRNA with Viral Vectors • Aptamers • Gene Therapy • Ribozymes with Other Vectors • Exon Skipping • Gene Editing (Meganucleases, ZFN, TALENs, CRISPR) Regenerative Medicine • Autologous & Allogenic Cells • Stem Cell Therapy • Mitochondrial Transfer • Tissue Engineering © 2020 Deloitte Global Services Limited Global Life Sciences and Health Care
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