World Stem Cell Summit 2012

Transcription

1 World Stem Cell Summit 2012 NYSE MKT: BTX December 4, 2012

2 Forward Looking Statements The matters discussed in this presentation include forward looking statements which are subject to various risks, uncertainties, and other factors that could cause actual results to differ materially from the results anticipated. Such risks and uncertainties include but are not limited to the success of BioTime in developing new stem cell products and technologies; results of clinical trials of BioTime products; the ability of BioTime and its licensees to obtain additional FDA and foreign regulatory approval to market BioTime products; competition from products manufactured and sold or being developed by other companies; the price of and demand for BioTime products; and the ability of BioTime to raise the capital needed to finance its current and planned operations. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. As actual results may differ materially from the results anticipated in these forward-looking statements they should be evaluated together with the many uncertainties that affect the business of BioTime and its subsidiaries, particularly those mentioned in the cautionary statements found in BioTime's Securities and Exchange Commission filings. BioTime disclaims any intent or obligation to update these forward-looking statements. 2

3 Four Criteria for Success in RegenMed Scalable & reproducible product Purity and identity of cells A formulation optimizing viability & immobilization of engraftment Strategies for near-term commercialization 3

4 Solving the Technical Hurdles Old Stem Cell Process 1.0 ES Cells Differentiation Purification of desired cell type Toxicity from impurities: Ectopic cysts tumors New Stem Cell Process 2.0 >200-fold diversity Scalable, monoclonally purified progenitors 4

5 Manufacturing Technology 2.0 Human embryonic progenitor (hep) cell lines: > 200 diverse cell types isolated Diversity Precise identity Purity Scalability Patents pending 5

6 Precise Identity Foxf1 Lhx8 Barx1 Foxf1 Genes & Dev. 18: Lhx8 Science 24:306: Barx1 Development 136:

7 Diversity of Defined Osteochondral Cells 7

8 Choroid Plexus & Alzheimers Disease 8

9 The Challenge: Cell survival demands a habitable environment Many regenerative medicine applications will require ü a temporary 3-D scaffold for cell immobilization and survival ü an injectable product capable of safely crosslinking in vivo. ü a natural and biodegradable product that does not induce inflammation 9

10 Renevia TM Cell Delivery Device Injectable, biocompatible, and biodegradable Cells in Renevia Matrix (Crosslinked Collagen and Hyaluronate) Low COGs, Stable Product, Potential Widespread Applications

11 HyStem Hydrogels Competitive Advantages Injectable Multiple Formulations Stays as liquid for ~ 20 minutes Polymerizes safely in vivo Supports survival of cells and 3-D structure Durable Cast Hydrogel Films 3-D Lattices Heparin-mediated Cells Sponge Slow Release 11

12 Renevia TM Development Indication: As a delivery matrix for autologous adipose derived cells to correct subcutaneous lipoatrophy arising from trauma, oncologic resection, or congenital defect. 3 Phase Clinical Evaluation of Renevia TM Phase 1: Safety - open label, Renevia TM only, subcutaneous injection, 10 subjects, 4 week follow up. Phase 2: Treatment Effect - open label, Renevia TM + ADSCs, 15 patients, 3-6 months follow up. Phase 3: Pivotal - randomized, controlled, evaluator blind, noninferiority, Renevia TM + ADSCs vs. Cell-Assisted Lipotransfer, # of patient per group TBD, 6 months follow up. Primary endpoints: Aesthetic Improvement Score & photo review Secondary endpoint: Change in subcutaneous defect volume Single Site: The Stem Center, Palma de Mallorca, Spain 12

13 Renevia TM A Cell Delivery Device Renevia TM Regulatory Strategy Adipose tissue restoration with autologous ASCs Medical device (CE Mark in the EU then CBER/CDRH) Outsource cgmp manufacture Near-term opportunity, low cost for approval Renevia TM Timeline Expected Renevia TM CE Mark in 2015 Complete cgmp Manufacture Safety Clinical Trial Initiated ISO Certification Treatment Effect Clinical Trial Initiated Pivotal Clinical Trial Initiated CE Mark Final Renevia Dossier Submission Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q

14 Subsidiaries OncoCyte Corporation Cancer diagnostics and anti-cancer therapies Cell Cure Neurosciences Ltd. Age-related macular degeneration (AMD) OrthoCyte Corporation Cartilage repair ReCyte Therapeutics, Inc. Age-related vascular disorders ES Cell International PTE Ltd. Clinical-grade master stem cell banks LifeMap Sciences, Inc. Stem cell data base BioTime Acquisition Corp.. Acquisitions 14

15 OncoCyte Corporation- PanC-Dx TM Screening in asymptomatic patients is the largest market opportunity PanC-Dx TM Pan diagnostic to detect the presence of various human cancers, including cancers of the breast, lung, bladder, uterus, stomach, and colon, during routine check-ups using a low-cost antibodybased blood test. mab production for seven antigens in process Specificity of >50 mabs characterized Dynamic testing for use in ELISA and point of care formats currently underway. Initially develop and seek regulatory approval of PanC-Dx TM in Europe Expected launch in

16 PanC-Dx TM Cancer Diagnostic Regulatory Strategy CE Mark (EU) then CDRH/OIVD (U.S.) Near-term opportunity, low cost for approval Timeline leading to 2014 launch Marker Validation Antibody Screening Antibody Production Final Kit Developed Patient Study Completion CE Mark Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q

17 Subsidiaries OncoCyte Corporation Cancer diagnostics and anti-cancer therapies Cell Cure Neurosciences Ltd. Age-related macular degeneration (AMD) OrthoCyte Corporation Cartilage repair ReCyte Therapeutics, Inc. Age-related vascular disorders ES Cell International PTE Ltd. Clinical-grade master stem cell banks LifeMap Sciences, Inc. Stem cell data base BioTime Acquisition Corp.. Acquisitions 17

18 Cell Cure Neurosciences Ltd. OpRegen RPE Cells for the Treatment of AMD 7.3 million have early stage dry AMD in US Any effective treatment expected to achieve blockbuster sales OpRegen Suspension of retinal pigment epithelial (RPE) cells for dry age-related macular degeneration (AMD) OpRegen Plus Matrix bound RPE cells for dry AMD Partnered with TEVA 18

19 Cell Cure Neurosciences Ltd. Planned IND in 2013 Phase I/IIa Study of hesc-rpe in Progressive Dry-Form Age- Related Macular Degeneration (12 patients, 1 year follow up time, single doses) Primary endpoint: Safety Secondary endpoint: Efficacy Visual Acuity Maintenance of graft size over serial measurements (graft survival) Reduced expansion of geographic atrophy in grafted sites vs. nongrafted sites (in the same eye) Perimetry- extent and depth of central scotomata Single site: Hadassah, Israel Duration (enrollment and follow up): 18 months 19

20 Subsidiaries OncoCyte Corporation Cancer diagnostics and anti-cancer therapies Cell Cure Neurosciences Ltd. Age-related macular degeneration (AMD) OrthoCyte Corporation Cartilage repair ReCyte Therapeutics, Inc. Age-related vascular disorders ES Cell International PTE Ltd. Clinical-grade master stem cell banks LifeMap Sciences, Inc. Stem cell data base BioTime Acquisition Corp.. Acquisitions 20

21 OrthoCyte Corporation Developing regenerative therapeutics for orthopedic applications Cartilage cannot regenerate on its own Osteoarthritis and spinal disc degeneration have a significant impact on mobility and health - > 26 million people in U.S. and growing Drug treatments target the reduction of pain and inflammation as opposed to repairing tissue Studies in animal models of IVD disease underway - Non-hypertrophic progenitors - Off the shelf approach 21

22 4D20.8 Scalability Regen Med 2012 Jul;7(4):

23 COL2A1 (Cartilage) Induction Regen Med 2012 Jul;7(4):

24 COL2A1 (Cartilage) Induction 24 Regen Med 2012 Jul;7(4):

25 OrthoCyte- Cartilage Progenitors Experimentally-induced trauma Implant OTX-CP03 OTX-CP07 4 weeks 25

26 Thank You Michael West, Ph.D. CEO, BioTime