An Investment in the most comprehensive regenerative medicine platform for orthopedics

Transcription

1 An Investment in the most comprehensive regenerative medicine platform for orthopedics Francois Binette PhD Vice President Research and Business Development

2 The OrthoCyte Vision Our initial focus: Spine and chronic back pain Mission statement: OrthoCyte will produce optimized regenerative medicine solutions for large orthopedic unmet needs with selected highly potent stem cell therapies delivered accurately for targeted sustained benefit while minimizing unwanted side effects. Back Pain 2

3 Rationale Spine: Largest Opportunity: Back pain is a big market: ~10-15 MM US addressable patients per year Speed to market: Faster/cheaper clinical development time: shorter follow up time (Pain and function vs structural repair), faster enrolment Our technology is best positioned to address the problem: Early-stage disease: Chondrogenic cells for disc repair End-stage disease: Osteogenic cells for bone fusion Cohesive SPINE customer-driven total solution: Early intervention: Pain specialists/interventionalists End-stage surgery: Spine surgeons 3

4 OrthoCyte initial focus: the aging spine Today s presentation Opportunity/Unmet need The Challenge Our Solution diverse osteochondral progenitors, biomaterials delivery Execution plan Product development plan 4

5 Chronic Back Pain is largest healtheconomic burden Back pain: 85% lifetime prevalence affects the working productive population < over 3% 12% 11% 39% 35% Direct Medical Cost billion Earning loss, indirect 22.4 billion y.o. 74% 2005 National Health Interview Surveys 5

6 Large therapeutic Gap in CBP care continuum No treatment options between conservative care and invasive surgery Irreversible disease progression Disc repair Bone repair for fusion LARGE THERAPEUTIC GAP: No treatment option 6

7 Tailoring effective stem cell therapies for the aging spine Content Opportunity/Unmet need The Challenge Our Technologies diverse osteochondral progenitors, biomaterials delivery Execution plan Product development plan 7

8 PureStem : The most diversified library of cloned stem cell for orthopedics Combines broad applicability of ESCs with defined targeted utility of adult MSCs Enhanced attributes Diversity >40 lines Purity Identity Potency Scalability 8

9 Selectable Attributes and Diversity of Options > 40 osteochondral progenitor PureStem optimal solutions Restricted attributes Optimized art. cartilage Optimized disc cartilage Optimized bone PureStem Selectable Attributes Distinct molecular signatures 9

10 PureStem cells effect on disc degeneration fibroblasts Degenerated disc Cell suspension OTX-01 OTX-03 OTX-05 AF/NP Gene expression at 7 days Repair: synthesis Pain: Inflammation Matrix degradation 10

11 In vivo degenerative disc disease POC DDD Histology, biochemistry 4 weeks 12 weeks Disc Puncture Cell Injection %DHI MRI treated normal injured FUSION Cell loaded bone graft 2-8 weeks uct, X-Ray

12 OrthoCyte is early but better positioned Competitive Landscape Company Technology platform Product Development stage Remarks Partner Disc degeneration Fusion Mesoblast Allogenic marrow derived mesenchymal stem cells MPCs PII PII Select MSCs Isto Allogenic juvenile chondrocytes NuQu PhI/II N/A Articular chondrocytes TETEC Autologous disc cell transplantation in carrier Novocart Disc N/A Adult disc cells DiscGenics Autologous disc cell transplantation in carrier Discospheres PreClinical Adult disc cells Co.Don Autologous disc cell transplantation ADCT EU marketed for herniated discs Adult disc cells OrthoCyte Progenitors Stem Cells PureStem PreClinical PreClinical >40 different lines targeting all disease states 12

13 Tailoring effective stem cell therapies for the aging spine Content Opportunity/Unmet need The Challenge Our Technologies diverse osteochondral progenitors, biomaterials delivery Execution plan Product development plan 13

14 HyStem Cell Delivery System Dynamic hydrogel enabling biomaterials - Derived from naturally occurring biomolecules: Hyaluronic acid and collagen Ubiquitous Broadly used in medical applications - In situ gelling cell delivery system Targeted delivery Minimally invasive approach 14

15 Near and long-term product development plan Near term: months to market DEVICES Long term: 3y to IND HyStem + Ceramics 510 K Bone Graft Bone repair BIOLOGICS + PureStem Bone BLA Bioactive Spinal Fusion Bone Induction Long term: 3y to IND BIOLOGICS + PureStem Disc BLA Bioative disc repair Disc Repair 15

16 Deliverables: Milestones to FIH Program initiative Tasks US Market opportunity (USD) Bone Graft Substitute Internal Development 300 MM commercialization POC in vivo PureStem Disc Optimization IND enabling >20 B clinicals POC in vivo PureStem Bone Optimization IND enabling 1.5 B clinicals This is what we plan to deliver in the next 2-3 Year: - Bone Graft Pipeline Development (Commercialization): Series of product launches in $300MM market - POC Bone and Disc Pure Stem (pre-clinical), cgmp compliant lines 16

17 Impact on OrthoCyte Valuation Valuation $MM Mesoblast $1.66B: Regen Med bench mark (NO approved Products, No BGS) Orthovita sold to Stryker 2011 for $318MM (3.3 x sales) Osteotech sold for Medtronic 2010 for $123MM (1.3 x sales) Apatech sold to Baxter 2010 for $330MM (5.5 x sales) Biomimetics sold to Wright in 2012 for $380MM (no sales) 17

18 The OrthoCyte Team - Francois Binette: VP R&BD - BioTime team: R&D, RA/QA, Legal, Finance, HR, etc - Seasoned consultants: Product development (former Allosource VP product development), Regulatory (FDC services) OrthoCyte Board: Michael West PhD: BioTime CEO Al Kingsley: BioTime Chairman Robert Peabody: BioTime CFO KOL Scientific Advisors/collaborators: Koichi Masuda MD: UCSD School of Medicine Dept Orthopedic Surgery Arnold Caplan PhD: Case Western Reserve University (Osiris and Cell Targeting founder) Daisuke Sakai MD PhD: Tokai University School of Medicine Dept Orthopedic Surgery KOL Clinical Advisors: TBA 18

19 The OrthoCyte is the only opportunity to address the entire CBP care continuum Multiple regenerative medicine therapeutic options Early intervention $20B OTX-01 disc induction End Stage intervention $300M to $1.6B OTX-11 bone induction Augmentation bone grafting

20 Thank You! Francois Binette PhD Vice President Research and Business Development