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Stem cells usa rm congress cord blood (boston, september 12, 2011)v.1

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Stem cells usa rm congress cord blood (boston, september 12, 2011)v.1

  1. 1. Stem Cells USA RM Congress:Cord Blood Banking and TherapeuticsBoston, MassachusettsGregory A. BonfiglioProteus Venture PartnersSeptember 12, 2011
  2. 2. Proteus: An Investment and AdvisoryFirm Focused on Regenerative Medicine Proteus, Inc. Proteus Proteus Proteus Management, LLC Insights, LLC Advisors, LLC (Fund Management) (Consulting Services) (Investment Banking Services) CONFIDENTIAL 2
  3. 3. AgendaI. The Regenerative Medicine Market  Key Market Metrics  RM Therapeutic ApplicationsII. The Cord Blood Market  Key Market Metrics  Banking Other TissuesIII. Therapeutic Applications for Cord Blood  Key Transplant Metrics  Current Treatments  Therapies in Clinical DevelopmentCONFIDENTIAL 3
  4. 4. Development of Regenerative Medicine Current technologies build on 50 years of research 1960s 1970s 1980s 1990s 2000s 1973 – First 2001 – unrelated 1997 – Dolly the RhBMP-7 1963 – Mouse bone marrow 1981 – Mouse Sheep cloned; – Approved ASCs isolated transplant ESCs Isolated FDA Approves Carticel (19997) & Apligraf (1999) FDA Approves Dermagraft (2001) 1986 – First 1968 – ALL Mouse Cloned patient irradiated, 2005 Use of infused with 1989 – First unrelated cord identical twin Sibling Cord Blood blood in BMT BMT Transplant (Gluckman) 1998 – 2007 – Human iPSCs ESCs from 1993 – ViaCell isolated Humans Begins Banking Cord Blood Source: Company websites, NIH, Pubmed CONFIDENTIAL 4
  5. 5. RM Is Entering A New ERA RM Market is Maturing: Key Metrics Rapidly Expanding Market: Commercial Products • $1.6B in 2010 • 400 on Market (Mostly Skin, Tools Media, & Devices); • $20.0B in 2025 – 900+ in Development • CAGR of 18.34% • 44 Cell Therapies on Market – $1B Revenues Dramatic Revenue Growth – 400 in Development • $130M in 2001 – 28 in PIII/Pivotal Trials • $1.6B+ in 2010 1.2M+ Patients Treated with RM Products. Worldwide funding for research Increasing • 320K+ Cell Therapy Patients • $2.5B Now RM Companies • $14B in 10 Years • 700+ Co’s involved in RM • 50+ Public Co’s; Clinical Programs • Over 3600 Clinical Trials – $8-$10B Total Market Cap • Over 400 ex-Oncology • 250+ Private Co’s CONFIDENTIAL 5
  6. 6. RM Market: Expanding Rapidly Dramatic Cell Therapy Revenue Growth CTI Revenues: $410M (2008) - $5.1B (2014) Cell Therapy Industry: Billion Dollar Global Business With Unlimited Potential; Regenerative Medicine; Chris Mason, David Brindley, Emily J Culme-Seymour & Natasha L Davie CONFIDENTIAL 6
  7. 7. RM Market: Global Company DistributionCanada UK24 firms 133 firms Europe (ex. UK) 3% 19% 14% 93 firms Asia 56% 2% 32 firms 5% Middle East 17 firms USA 386 firms 700+ RM companies worldwide! CONFIDENTIAL 7
  8. 8. Big Pharma is Actively EngagedGSK & HSCI: $25M Deal Merck, Pfizer & Lily Launch Enlight BioSciencePfizer RM Division: $111M Deal withAthersys (IBD); UCL (RPE); ViaCyte(Diabetes) GSK, AZN & Roche Help Launch Stem Cells for Safer Medicine in UKGenzyme & Osiris: $1.25B Deal Johnson & Johnson Invests in Tengion & ViaCyte (NovoCell)Cephalon & Mesoblast: $2B Deal GE & Cytori: StemSourceShire Acquires ABH for $750M CONFIDENTIAL 8
  9. 9. RM Business Models: Autologous v. Allogeneic Autologous Model Allogeneic Model Patients Own Cells/Tissue Universal Cells in a Bottle • Personalized Medicine • Big Pharma “Drug Model” Advantages: • Autologous Treatment for Prostate Advantages: Cancer Using Dendritic Cells • Easier Regulatory Path • Scalable (GTP) -Centralized Processing • Low COGS • No Immune Response • $93K per Treatment Challenges: Challenges: • More Difficult -$1B+ Projected Revenues Regulatory • Difficult to Scale Path • High COGS• $5.0B Market Cap (Until Aug 3,Response • Immune 2011) Service vs. Product CONFIDENTIAL 9
  10. 10. AgendaI. The Regenerative Medicine Market  Broad RM Market: Key Metrics  RM Therapeutic ApplicationsII. The Cord Blood Market  Cord Blood Market: Key Metrics  Banking Other TissuesIII. Therapeutic Applications for Cord Blood  Key Transplant Metrics  Current Treatments  Therapies in Clinical DevelopmentCONFIDENTIAL 10
  11. 11. Cord Blood Market:Key Metrics Cord Blood Market Metrics Market Size: Market Penetration • $3.4B (2010) • 1-6% of All Births (varies by • $14.9B (2015) country/region) • CAGR: 27.9% Average Fees: Private Banks Cord Blood Banks: • $1,750 Initial Collection & Storage • 150+ Private Banks – Range: $890 - $2300 • 44 Public Banks • $125 Annual Fee • 26 Countries – Range: $85-$150 Total Cord Blood Units Stored Average Fee: Public Banks • 500,000 Units in Public Banks • $35,000 per Unit (US/EU) • 1M+ Units in Private Banks Public Bank Utilization Rate: • 1-3% of Units per year CONFIDENTIAL 11
  12. 12. Cord Blood Market:Key Metrics Cord Blood Market Metrics Total Cord Blood Transplants: Regulatory Framework 25,000 in 43 Countries • Regulated as a “Biologic” by FDA/EMA • 1,500 per year (2005) • By October 2011, All Cord Blood • 3,000 per year (2010) Banks Must be Licensed (BLA) • 10,000 per year (2015) Therapeutic Applications 50% of All Patients Seeking a • 60+ in Clinical Practice BM/PBSC Transplant Cannot • Leukemia; Lymphoma; Blood Find a Match Disorders; Hematopoietic Restoration Fastest Growing Segment of Clinical Trials Cell Transplant Market • Over 600 FDA Clinical Trials • 22% of All Cell Transplants in – 425+ New Therapies 2010 – 57 Pivotal/PIII Trials • 40% by 2015 CONFIDENTIAL 12
  13. 13. Cord Blood Therapies in Clinical Development:600+ Ongoing FDA Trials Source: ClinicalTrials.gov (www.clinicaltrials.gov) CONFIDENTIAL 13
  14. 14. Banking Other Tissues• Wharton’s Jelly/Entire Cord • CHORI Technology (www.chori.org)• Placental Tissue (MSCs) • Pluristem Therapeutics, Inc. (www.pluristem.com) ; LifebankUSA (www.lifebankusa.com)• Amniotic Fluid (MSCs & HSCs) • Biocell Center (www.biocellcenter.com)• Autologous Marrowized Bone • Arnie Caplan (Cell Bank Technologies)• iPS Cell Lines • Shinya Yamanaka (Japan)/Janet Rossant (Toronto); iPerian (www.ipierian.com)CONFIDENTIAL 14
  15. 15. Banking Other Tissues• Peripheral Blood/Bone Marrow • Stem Cell Assurance, Inc. (www.stemcellassurance.com) • StemCor - Marrow-Miner• ALDH Cells (Hematopoietic Stem And Progenitor Cells) • Aldagen (www.aldagen.com)• Adipose Derived Mesenchymal Cells (MSCs) • Cytori (www.cytori.com); GE Healthcare • Stem Cell Assurance, Inc. (www.stemcellassurance.com)• Menstrual Blood • CryoCell (www.cryo-cell.com)CONFIDENTIAL 15
  16. 16. AgendaI. The Regenerative Medicine Market  Key Market Metrics  RM Therapeutic ApplicationsII. The Cord Blood Market  Key Market Metrics  Banking Other TissuesIII. Therapeutic Applications for Cord Blood  Key Transplant Metrics  Current Treatments  Therapies in Clinical DevelopmentCONFIDENTIAL 16
  17. 17. Current Therapeutic Applicationsfor Cord Blood Market Key Metrics • 25,000 + Cord Blood Transplant To Date • 3000+ Transplants in 2010 o 9% Increase over 2009 o 100% Increase (2X) over 2005 • 10,000 Transplants per year projected by 2015 o CAGR: 49.3% • Multi-Cord Transplants Increased 25% in 2010 • 60+ Diseases Treated CONFIDENTIAL 17
  18. 18. Cord Blood Therapeutics:Comparison with Bone Marrow Cord Blood v. Bone Marrow HLA 2nd or Donor Units Search Engraftment GvHD Ethnic Match Double Morbidity Available Time Time Risk Match Required GraftCord 550,000 None 4/6 1 Day Yes 26 Days Minimal EasierBlood Units PossibleBone 8/8 14.6M 3-4 if donor 50% 18 Days 50% DifficultMarrow 10/10 Donors Months is available Confidential 18
  19. 19. Cord Blood Transplants Have Doubled InLast 5 Years (NMDP Data)CONFIDENTIAL 19
  20. 20. Cord Blood Is the Preferred Cell Sourcefor Pediatric Patients (NMDP Data)CONFIDENTIAL 20
  21. 21. Adult Patients Are the Fastest GrowingPopulation: 58% 0f 2010 Cord Transplants Cord Blood Transplants By Patient Age (NMDP Data) 0-17 18+ 1,200 1,000 800 600 400 200 0 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 CONFIDENTIAL 21
  22. 22. Transplant Outcomes Have ImprovedDramatically in Last 5 Years Report Year Period One-Year Survival 2008 2002-2006 54.0% 2007 2001-2005 51.5% 2006 2000-2004 48.8% 2003 1996-2001 42.2% • Improved HLA Matching; • Advances in Conditioning Regimes; KEY FACTORS • Advances in Post-Transplant Supportive Care CONFIDENTIAL 22
  23. 23. Clinical Outcomes Related toQuality of Tissue & Match Key Metrics Quality of Tissue • TNC Count (150+) • CD34+ Count • Size of Unit Matching Criteria • HLA Match (4/6 Min) • Race & Ethnicity Match • Genetic Match (DNA tissue typing) CONFIDENTIAL 23
  24. 24. Cord Shipments By TNC Count for Adults1,2001,000 800 150+ 600 125-149 90-124 <90 400 200 0 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 Most Units (77%) for Adults Had TNC >150 (NMDP Data) CONFIDENTIAL 24
  25. 25. Public Bank Utilization Rates: 2010 Inventory and Shipments TNC of Shipments - TNC of Inventory as of CY 2010 June 30, 2010 Less than 125 Greater 18% than or equal to 125 31% Less than Greater 125 than or 69% equal to 125 82%82% of all 2010 shipments came from 31% of the available inventory.(NMDP Data) CONFIDENTIAL 25
  26. 26. Current Therapeutic Applicationsfor Cord Blood 60+ Diseases Treated • Leukemias And Lymphomas, Including: – Acute Myelogenous Leukemia – Acute Lymphoblastic Leukemia • Multiple Myeloma And Other Plasma Cell Disorders • Severe Aplastic Anemia And Other Marrow Failure States, Including: – Severe Aplastic Anemia – Fanconi Anemia • SCID And Other Inherited Immune System Disorders, Including: – Severe Combined Immunodeficiency (SCID, All Sub-types) – Wiskott-aldrich Syndrome • Hemoglobinopathies, Including: – Beta Thalassemia Major – Sickle Cell Disease CONFIDENTIAL 26
  27. 27. Diseases Treated:Hematologic Malignancies (NMDP Data)CONFIDENTIAL 27
  28. 28. Diseases Treated:Non-Malignant Disorders (NMDP Data)CONFIDENTIAL 28
  29. 29. FDA Trials Involving Cord Blood AddressNumerous Conditions & Diseases • Bacterial and Fungal Diseases • Mouth and Tooth Diseases • Behaviors and Mental • Muscle, Bone, and Cartilage Disorders Diseases • Blood and Lymph Conditions • Nervous System Diseases • Cancers and Other Neoplasms • Nutritional and Metabolic Diseases • Digestive System Diseases • Parasitic Diseases • Diseases and Abnormalities at • Respiratory Tract (Lung and or before Birth Bronchial) Diseases • Ear, Nose, and Throat Diseases • Skin and Connective Tissue • Eye Diseases Diseases • Gland and Hormone Related • Substance Related Disorders Diseases • Symptoms and General Pathology • Heart and Blood Diseases • Urinary Tract, Sexual Organs, and • Immune System Diseases Pregnancy Conditions • Wounds and Injuries • Viral Diseases Source: ClinicalTrials.gov (www.clinicaltrials.gov) CONFIDENTIAL 29
  30. 30. APPENIDXCONFIDENTIAL 30
  31. 31. NMDP DataCONFIDENTIAL 31
  32. 32. NMDP DataCONFIDENTIAL 32
  33. 33. NMDP DataCONFIDENTIAL 33
  34. 34. Adult Patients Are the Fastest GrowingPopulation: 58% 0f 2010 Cord Transplants CONFIDENTIAL 34
  35. 35. The Promise of Regenerative Medicine To Cure Diabetes To Cure Vision Disorders To Heal Acute Spinal Cord Injury Paralyzed Rat Paralyzed Rat Walks (double click to see video) (double click to see video)To Replace Entire Organs Tools for Drug Discovery To Regenerate Heart MuscleCONFIDENTIAL 35
  36. 36. Gartner’s Hype Cycle of Emerging TechnologiesVisibility Technology Peak of Inflated Trough of Slope of Plateau of Productivity Trigger Expectations Disillusionment Enlightment Time CONFIDENTIAL 36
  37. 37. RM Market: On 2nd Half of the Gartner CurveVisibility Peak of Inflated Trough of Slope of Plateau of Expectations Disillusionment Enlightment Productivity 2001: 3300 jobs, 73 firms, mkt cap > $2.5B 2011 Shire Acquires ABH -$750M 2001 Ortec FDA approved 2000 Time Magazine: 2010 Cephalon & Mesoblast $2B Deal TE No. 1 job 2001 Dermagraft FDA approved 2010 Dendreon’s Provenge Approved 1999 TE bladders in clinic 2001 Bush “partial ban” on HESCs 1999 First FDA approved 2009 Obama Ends Ban on hESCs TE product (Apligraf) 1998 Human ESCs first derived Nov 2008 Genzyme-Osiris $1.25B Deal 1997 Dolly the sheep 2007 iPS Technology Developed 1997 First FDA approved cell therapy (Carticel) 2007 Apligraf - 200,000 Patients Treated 2006 Carticel - 10,000 Patients Treated 2002 ISSCR Founded 1992 Geron founded 2005 CIRM Founded 1988 SyStemix founded 1986 ATS and Organogenesis founded 2003 UK Stem Cell Bank Formed 1980 Early TE research (MIT) 2002 ATS + Organogenesis file Chapter 11 Technology Trigger Stage of Development Time CONFIDENTIAL 37

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