Regenerative Medicine Industry Outlook 2014
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The document summarizes the regenerative medicine field based on a report from the Alliance for Regenerative Medicine, including an overview of major industry players and subsectors, clinical trials and products in development, financial performance and investments in 2013, and insights from a panel discussion on key areas of focus. It analyzes trends in cell and gene therapies, areas attracting pharmaceutical investment, and challenges facing the commercialization of regenerative medicine technologies and therapies.
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Regenerative Medicine Industry Outlook 2014
- 2. 1. About ARM a. Review of Regenerative Medicine b. RM Stakeholders c. Major Industry Sub-Sectors 2. RM Product and Clinical Trial Overview 3. 2013 RM Financial Performance Analysis 4. Highlights from ARM Pharmaceutical Survey 5. Panel Discussions a. Key Clinical Data Inflection Points for 2014 b. Recent Financings in the Sector
- 3. • Founded in 2009, ARM includes all of the major stakeholders in the field of RM working together to bring transformative healthcare solutions to patients • ARM’s activities focus on reducing uncertainty in the RM field through legislative, advocacy, regulatory, reimbursement, informational and scientific initiatives ARM Membership Demographic 152 Members 9% 3% 5% Big Pharma (5%) Therapeu8c Companies (33%) 9% 33% 17% 24% Tools and Service Companies (24%) Pa8ent Advocates, Founda8ons and Associa8ons (17%) Academic Research Ins8tu8ons (9%) Tissue Engineering, Biomaterials and Devices (9%) Capital Providers and other Financial Ins8tu8ons (3%)
- 4. Regenerative Medicine is a revolutionary field focused on translating fundamental knowledge in biology, chemistry and physics into materials, devices, systems and a variety of therapeutic strategies which augment, repair, replace or regenerate organs and tissues. Regen Med Industry Sectors Tools & Reagent Companies 15% 476 Companies Analyzed Cell & Tissue Banks 33% Therapeu8c and Device Companies 40% Service Companies 12% Company Breakdown: Therapeutics Cell Therapy Companies 27% Gene Therapy Companies 5% 8% 60% Small Molecule and Biologic Companies -‐ Endogenous Ac8va8on Tissue Engineering Companies
- 5. Cell Therapy Product Revenue 2010 Product Revenue 2011 2012 2013 $460 Million $730 Million $900 Million $1.3 Billion There are Currently Over 40 Cell Therapy Products Commercially Available 2% 7% Non-healing wounds 10% 35% Musculoskeletal Skin 11% Cancer Ocular Cardiovascular 35% Sample of Leading Commercial Cell Therapy Products Company Product AlloSource (distributed by NuVasive) Alphatec Spine Anterogen Avita Medical BioD (distributed by Amedica) BioD (distributed by Amedica) Dendreon Fibrocell Genzyme, a Sanofi company Genzyme, a Sanofi company Genzyme, a Sanofi company Medipost NuVasive Organogenesis Organogenesis Orthofix Osiris Therapeutics Pharmacell Shire TiGenix Zimmer and ISTO Technologies Osteocel PureGen Cupistem ReCell BioDfactor BioDfence Provenge LAVIV Carticel Epicel MACI Cartistem Osteocel Apligraf GINTUIT Trinity/Trinity Evolution Grafix Heartcelligram-AMI Dermagraft ChondroCelect DeNovo NT
- 6. By Jurisdiction United States Europe Other Regulated Jurisdictions Dermagraft MySkin Cupistem (S.Korea) Osteocel CryoCell Heartcelligram (S. Korea) PureGen ReCell Cartistem (S. Korea) BioDfactor CartiCel BioDfence Epicel J-TEC Epidermis (Japan) LAVIV MACI Provenge AlloStem J-TEC Corneal Epithelium (Japan) Carticel BioSeed-C CureXcell (Israel) Epicel Co.don Chondrospheres Prochymal (Canada & NZ) Nucel Apligraf GINTUIT Trinity Evolution J-TEC Cartilage (Japan) Epidex EpiGraft ChrondroCelect Grafix DeNovoET Prokera AmnioGraft Source: ARM 2013 Annual Report
- 7. “The number of cell and gene therapies in clinical pipelines has grown from 429 to 575 in the past five years according to McKinsey & Co. The pipeline is also maturing, with more treatments in Phase III trials.” Diller, Wendy "Shedding Earlier Fears, Pharma Takes A Fresh Look At Cell, Gene Therapies." Elsivier Business Intelligence: In Vivo; December 23, 2013.
- 8. Ongoing Industry-Sponsored RM Trials by Stage ARM Database Research Pre-Clinical Early-Stage (Ph. I) Mid-Stage (Ph. II) Late-Stage (Ph. III) 58 Trials 245 Trials 70 Trials 174 Trials 24 Trials Late-‐Stage Industry-‐Sponsored RM Trials Stroke, 2% AutoImmune, 4% Skin, 2% Ø 185 Therapeutic Companies Ø 320 Regen Med Products Early-‐to-‐Mid Stage Industry-‐Sponsored RM Trials Other, 2% Ocular, 4% Other, 18% Skin, 2% Cancer, 32% Cardiovascular, 11% Cancer, 28% Spinal Cord Injury , 3% Diabetes, 4% Stroke, 5% Non-healing wounds, 15% Ocular, 5% Musculoskeletal , 28% AutoImmune, 7% Musculoskeletal, 8% Cardiovascular, 12% Non-‐ healing wounds, 8%
- 9. Date Company Product Indication Milestone 1Q14 Pluristem PLX-PAD Trauma Phase I/II results 1H14 Cytori ADSRCs Myocardial Ischemia Phase II results 1H14 Athersys MultiStem Ischemic Stroke Preliminary Phase II results 2Q14 Athersys MultiStem Ulcerative Colitis Phase II results 3Q14 NeoStem AMR001 Myocardial Infarction Phase II results 4Q14 TiGenix Cx601 Perianal Fistula Phase III results 2H14 ReNeuron ReN009 Critical Limb Ischemia Phase I results 2H14 Fibrocell Laviv Burn Scar Phase II results 2014 Juventas JVS100 Cardiovascular Failure Phase II results 4Q15 Cardio3 C-Cure Congestive Heart Failure Phase III results 2015 Mesoblast Revascor Congestive Heart Failure Market launch 2015 bluebird LentiGlobin Sickle Cell/Beta Thalassemia Market launch Mid-2015 ReNeuron ReN001 Cerebral Ischemia Phase II End Source: Medtrack
- 10. Industry-Sponsored Cell-Based Immunotherapy Trials Late-Stage Number of Trials Mid-Stage Early-Stage 13 54 10 Source: ARM 2013 Annual Report 2014 Cell-Based Immunotherapy Trials to Watch For Date Company Product Indication Milestone 1Q2014 Northwest Biotherapeutics DCVax Direct Solid Tumors Phase I/II results 2H2014 Opexa Tcelna MS Phase IIb End 3Q2014 Prima BioMed Cvac. Ovarian Cancer Interim Phase III results 4Q2014 Northwest Biotherapeutics DCVax L Glioblastoma Phase III results Source: Medtrack
- 11. Deal Type Company(s) Deal Value/Upfront Date Collaboration Sangamo / Biogen Idec 20M 1/9/14 Collaboration/Licensing Deal Capricor / J&J 12.5M 1/6/14 Acquisition Intrexon / Medistem 26M 12/20/13 Licensing Deal Pluristem / CHA Biotech 10.4M 12/17/13 Licensing Deal Cytori / Lorem Vascular 24M 11/4/13 Acquisition Mesoblast / Osiris 100M 10/11/13 Collaboration Stratatech / BARDA Contract 47.2M 7/31/13 Collaboration Tengion / Celgene 33.6M 7/1/13 Merger Capricor / Nile Therapeutics NA 7/8/13 Acquisition Smith & Nephew / Healthpoint 782M 11/28/12 Acquisition Royal DSM / Kensey Nash 360M 5/3/12 Acquisition Cytomedix / Aldagen 16M 2/8/12 Licensing Deal Sangamo / Shire 13M 2/1/12
- 12. Initial Public Offerings by Regenerative Medicine Companies in 2013 Company Amount Raised Date Exchange Performance since IPO Cardio 3 Biosciences $31.3M 6/4/13 EBR 53.45% bluebird bio $116M 6/18/13 NASDAQ (22.04%) Capricor Therapeutics N/A 7/8/13 OTCMKTS 853.95% Cellular Dynamics International $46M 7/24/13 NASDAQ 82.84% Organovo $46.6M 8/7/13 Up-listed to NYSE 125.6% Intrexon $184M 8/13/13 NYSE (3.76%) Regeneus Ltd $10.5M 9/12/13 ASX 88.89% Fate Therapeutics $40.4M 10/4/13 NASDAQ (3.78%) UniQure $75M (expected) 1/2/14 (file date) NASDAQ N/A
- 13. 2012 2013 PIPES, VC & Private Equity $765M $644.1M Public Offerings $170M $736.6M TOTAL $935M $1.381B $900 $800 $700 $600 PIPES, VC & Private Equity $500 $400 Public Offerings $300 $200 $100 $0 2012 2013
- 14. Where Pharma is Investing in Regenerative Medicine 80% 70% 60% 50% 40% 30% 20% 10% 0% 70% 60% 50% 40% 30% 20% 10% 0% Survey Participants: Allergan, Amgen, Baxter, Biogen Idec, Boehringer Ingelheim, Celgene, Eli Lilly, GSK, Johnson & Johnson, Merck Serono, Novartis, Novo Nordisk, Pfizer, Roche, Sanofi-Genzyme, Shire
- 15. Pharma’s Interest in the Pillar Technologies • Areas showing moderate to significant interest 80% 70% 60% 50% 40% 30% 20% 10% 0% Survey Participants: Allergan, Amgen, Baxter, Biogen Idec, Boehringer Ingelheim, Celgene, Eli Lilly, GSK, Johnson & Johnson, Merck Serono, Novartis, Novo Nordisk, Pfizer, Roche, Sanofi-Genzyme, Shire
- 16. Where Pharma Sees Therapeutic Opportunities 70% 60% 50% 40% 30% 20% 10% 0% Survey Participants: Allergan, Amgen, Baxter, Biogen Idec, Boehringer Ingelheim, Celgene, Eli Lilly, GSK, Johnson & Johnson, Merck Serono, Novartis, Novo Nordisk, Pfizer, Roche, Sanofi-Genzyme, Shire Over Next 10 yrs
- 17. What Pharma Sees as the Key Challenges • Areas posing significant and moderate concerns 50% 45% 40% 35% 30% 25% 20% 15% 10% 5% 0% Survey Participants: Allergan, Amgen, Baxter, Biogen Idec, Boehringer Ingelheim, Celgene, Eli Lilly, GSK, Johnson & Johnson, Merck Serono, Novartis, Novo Nordisk, Pfizer, Roche, Sanofi-Genzyme, Shire significant concern/ challenge moderate concern/ challenge
- 18. • We realize it’s a frontier technology beyond a five year time horizon and we don’t want to miss the boat. Our company is engaged in various levels and resources are internally devoted. • Focusing on rare diseases allows us to test transformative platforms on small patient groups with lower regulatory boundaries. • We would like to invest early, close to proof-of-concept. We will continue to invest in areas such as stem cells, gene therapy and other RM venture investments. • In 10 years we will crack Diabetes. This is a major area of interest. • Most promising areas of RM include the ischemic space/cardiovascular, autoimmune/UC/IB/ GVHD, skin and musculoskeletal related injury and disease. • We believe that monogenic disease is where you can focus and be successful because there's no other therapeutic option. We can also be successful in this area because the MOA is 100% clear. Large indications are tough because we don't really understand the disease. • iPSCs for drug discovery, toxicology and modeling is our core focus in regenerative medicine. This technology not only enhances drug discovery, it is a paradigm shift in drug discovery. • Most of our efforts in RM would be through partnerships…we want to be working with the experts.
- 19. Moderator: • Jason Kolbert, Managing Director, Maxim Group Panelists: • Robert Brenner, President & CEO, AlloCure • Andrew Pecora, Chief Visionary Officer, NeoStem • Marc Penn, Chief Medical Officer, Juventas Therapeutics; Co-PI of MultiStem Cardio Trial, Athersys • Eliseo Salinas, EVP & Head of R&D, StemCells, Inc.
- 20. Moderator: • Dave Stadinski, Head, Equity Capital Markets, Piper Jaffray Panelists: • Christopher Calhoun, CEO, Cytori Therapeutics • Keith Murphy, Chairman & CEO, Organovo • Michael Schuster, Founding Executive & Head of Global Therapeutic Products, Mesoblast • Jeffrey Walsh, Chief Operating Officer, bluebird bio