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25 th Annual ROTH Conference NYSE MKT: BTX March, 2013.

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Presentation on theme: "25 th Annual ROTH Conference NYSE MKT: BTX March, 2013."— Presentation transcript:

1 25 th Annual ROTH Conference NYSE MKT: BTX March, 2013

2 Forward Looking Statements 2 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.

3 The Health Care Challenge & Opportunity A global tsunami of health care costs from ineffectiveness of drugs to treat chronic degenerative disease (CDD) CDD due to lack of cellular regeneration 80% Americans have CDD of aging 50% of Americans have two CDDs 95% costs in aging is CDD

4 The Health Care Challenge & Opportunity – Gene cloning technology developed Moratorium on rDNA research initiated led to established guidelines on rDNA research 1989 – First $B product EPO Today, products from the use of rDNA technology are ubiquitous >140 clinical trials Current Global Market $75 B Recombinant DNA Technology Hybridoma technology developed First $B Product Rituximab Advances in Mab Engineering Today, eight of the 20 best- selling biotechnology drugs in therapeutic monoclonal antibodies > 200 clinical trials Current Global Market $44 B Monoclonal Antibodies 1998 – Embryonic Stem Cells allowing for the first time in the history of medicine the manufacture of all human cellular components 2001 – U.S. Federal funding restriction (reversed in 2009) 2010 – 1 st hES Clinical trial Future – 1 st $B product Regenerative Medicine Progressive evolution of new & more interventional products over past four decades

5 The BioTime Opportunity 5 Consolidating key stem cell assets - ES Cell International - Geron stem cell assets - HyStem hydrogels Integration with PureStem technology Focus on near-term products Significant clinical milestones for 2013

6 PureStem Manufacturing Technology 6 Human embryonic progenitor (hEP) cell lines: > 200 diverse cell types isolated Diversity Precise identity Purity Scalability Patents pending

7 Injectable, biocompatible, and biodegradable Renevia TM – Cell Delivery Device Cells in Renevia Matrix (Cross- linked Collagen and Hyaluronate) Low COGs, Stable Product, Potential Widespread Applications

8 HyStem Hydrogels – Competitive Advantages 8 Polymerizes safely in vivo Stays as liquid for ~ 20 minutes Supports survival of cells and 3-D structure Cast HydrogelCells in Sponge Injectable Multiple Formulations Durable Films 3-D Lattices Heparin-mediated Slow Release

9 9 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, non- inferiority, 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 Renevia TM – Development

10 Renevia TM – A Cell Delivery Device 10 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 Regulatory Strategy Renevia TM Timeline 2012 Q4Q1Q2Q3Q4Q1Q2Q3Q ISO Certification Final Dossier Submission CE Mark Renevia™ Complete cGMP Manufacture Q Safety Clinical Trial Initiated Treatment Effect Clinical Trial Initiated Pivotal Clinical Trial Initiated Expected Renevia TM CE Mark in 2015

11 Potential Renevia TM Applications 11 Heart Disease ✔ 165 B Alzheimer’s ✔ 69 B Diabetes ✔ 92 B Stroke ✔ 28 B Renal Failure ✔ 33 B Potential Utility Potential Applicable U.S. Market Regen. Med. (2008) 3(3), 351–363

12 Subsidiaries 12 OncoCyte Corporation Cancer diagnostics and anti-cancer therapies OrthoCyte Corporation Cartilage repair ES Cell International PTE Ltd. Clinical-grade master stem cell banks LifeMap Sciences, Inc. Stem cell data base and research products Cell Cure Neurosciences Ltd. Age-related macular degeneration (AMD) ReCyte Therapeutics, Inc. Age-related vascular disorders BioTime Acquisition Corp.. Expected Contribution of Geron and BioTime Assets

13 OncoCyte Corporation- PanC-Dx TM 13 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 antibody- based 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 2014

14 PanC-Dx TM Cancer Diagnostic Q4 Q1Q2 Q3 Q4 Q1Q2 Q3 Q4 Q Marker Validation Antibody Production Final Kit Developed CE Mark Regulatory Strategy CE Mark (EU) then CDRH/OIVD (U.S.) Near-term opportunity, low cost for approval Timeline leading to 2014 launch Patient Study Completion Antibody Screening 2013 Q4 Q3 Q2Q1

15 Subsidiaries 15 OncoCyte Corporation Cancer diagnostics and anti-cancer therapies OrthoCyte Corporation Cartilage repair ES Cell International PTE Ltd. Clinical-grade master stem cell banks LifeMap Sciences, Inc. Stem cell data base and research products Cell Cure Neurosciences Ltd. Age-related macular degeneration (AMD) ReCyte Therapeutics, Inc. Age-related vascular disorders BioTime Acquisition Corp.. Expected Contribution of Geron and BioTime Assets

16 Cell Cure Neurosciences Ltd. 16 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

17 Cell Cure Neurosciences Ltd. 17 Planned IND in 2013/2014 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. non- grafted sites (in the same eye) Perimetry- extent and depth of central scotomata Single site: Hadassah, Israel Duration (enrollment and follow up): 18 months

18 Subsidiaries 18 OncoCyte Corporation Cancer diagnostics and anti-cancer therapies OrthoCyte Corporation Cartilage repair ES Cell International PTE Ltd. Clinical-grade master stem cell banks LifeMap Sciences, Inc. Stem cell data base and research products Cell Cure Neurosciences Ltd. Age-related macular degeneration (AMD) ReCyte Therapeutics, Inc. Age-related vascular disorders BioTime Acquisition Corp.. Expected Contribution of Geron and BioTime Assets

19 OrthoCyte Corporation 19 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

20 OrthoCyte- Cartilage Progenitors 20 E15

21 Subsidiaries 21 OncoCyte Corporation Cancer diagnostics and anti-cancer therapies OrthoCyte Corporation Cartilage repair ES Cell International PTE Ltd. Clinical-grade master stem cell banks LifeMap Sciences, Inc. Stem cell data base and research products Cell Cure Neurosciences Ltd. Age-related macular degeneration (AMD) ReCyte Therapeutics, Inc. Age-related vascular disorders BioTime Acquisition Corp.. Expected Contribution of Geron and BioTime Assets

22 Regenerative Medicine 22 Human embryonic stem (hES) for limited allogeneic applications Twin strategies for cell transplantation Sites behind the blood brain barrier Sites behind synovial membranes Cells targeting tumors for destruction Human induced pluripotent stem (iPS) cells for auto- logous transplantation Vascular disease

23 ReCyte Therapeutics, Inc. 23 RCX-VP01 for Age-Related Vascular Insufficiency American Heart Association Report on Heart Disease and Stroke Statistics: 2011 Update Cardiovascular Disease (CVD) Prevalence:  82,600,000 American adults (>1 in 3) with 1 or more types of CVD  2200 deaths from CVD per day; average life expectancy shortened  7 years Stroke (Cerebrovascular Disease) Incidence:  800,000 new (  77%) or recurrent (  23%) strokes per year  1 of every 18 deaths in U.S. in 2007 Healthcare Impact and Cost:  $286 billion total direct and indirect costs of CVD and stroke in U.S. in 2007 nearly 7 million inpatient cardiovascular operations and procedures in 2007

24 Subsidiaries 24 OncoCyte Corporation Cancer diagnostics and anti-cancer therapies OrthoCyte Corporation Cartilage repair ES Cell International PTE Ltd. Clinical-grade master stem cell banks LifeMap Sciences, Inc. Stem cell data base and research products Cell Cure Neurosciences Ltd. Age-related macular degeneration (AMD) ReCyte Therapeutics, Inc. Age-related vascular disorders BioTime Acquisition Corp.. Expected Contribution of Geron and BioTime Assets

25 LifeMap Sciences, Inc. 25 Used in more than 1,000 institutions world-wide including academia, research hospitals, patent offices, and the leading biotech and pharma More than 12 Million page visits/year by hundreds of thousands of unique users 30% usage growth in the past 12 months Consistently leads in top positions for gene search results in Google The leading human gene database

26 LifeMap BioReagents 26 Cells Powerful Marketing Platform >12 Million page hits/yr Genes Diseases Near-term Revenue Seed for Future Royalties BioTime Products All online % of Sales Third Party Products > 1,000 USCN Antibody kits >1,000 Prospec- Tany Technogene Proteins

27 Subsidiaries 27 OncoCyte Corporation Cancer diagnostics and anti-cancer therapies OrthoCyte Corporation Cartilage repair ES Cell International PTE Ltd. Clinical-grade master stem cell banks LifeMap Sciences, Inc. Stem cell data base and research products Cell Cure Neurosciences Ltd. Age-related macular degeneration (AMD) ReCyte Therapeutics, Inc. Age-related vascular disorders BioTime Acquisition Corp.. Contribution of Geron and BioTime Assets

28 BioTime Acquisition Corp. 28 Geron will contribute: IP and tangible assets related to hES programs Phase 1 trials in acute spinal cord injury & T’ase vaccine IP and sublicense to autologous cellular immunotherapy program Interests in non-therapeutic applications including products developed and sold by GE Heathcare Geron receives: Shares in BAC (approx 21.4% interest) Royalties on sale of products using Geron patents Shares will be distributed by Geron to their shareholders (following closing) Terms subject to closing What Geron Contributes and Receives…

29 BioTime Acquisition Corp. 29 BioTime will contribute to BAC: $5 million in cash (committed through a private investor) 8.9 MM in BioTime Common Stock to be held by BAC 8 million BioTime warrants At closing, to be distributed by BAC to Geron stockholders Minority equity interest in OrthoCyte and Cell Cure Rights to use certain BioTime cell lines BioTime receives: Shares in BAC (Approx. 71.6% interest in BAC) 3.15 MM Warrants in BAC Terms subject to closing What BioTime Contributes and Receives…

30 BioTime Acquisition Corp. 30 Stem Cell Assets Being Contributed from Geron

31 BioTime Acquisition Corp. 31 Feeder-free culture of hPS cells hES-derived dendritic cells for treating cancer TGFbeta inhibitors & Neural RNA isolation from hPS cells or derivatives Feeder free hPS cells in drug screening Medium for hPS cell culture hPS-derived hepatocytes Dopaminergic neurons from hPS cells hPS cell-specific genetic toxicity Selective antigen expression on hPS cells >400 issued & pending patent applications worldwide including: Stem Cell Assets Being Contributed from Geron

32 Current Product Pipeline 32 Phase IPhase IIPhase IIIMarketPartnerPreclinical Hextend Research Products/Database Teva Renevia PanC-Dx TM OpRegen OTX-CP07 RCX-VP01 HyStem ® Hydrogel Products

33 BioTime (NYSE MKT: BTX) 33 $4.4MM cash of 12/31/12 Raised additional $16.3 million to date in 2013 Approx. $10MM in investments held by our subsidiaries Major Shareholders: Greenbelt/Greenway/ Al Kingsley (Chairman) Revenue of $3.9MM in 2012 Avg. qtrly burn rate of $4.9MM per quarter No debt Market Cap approx. $240MM as of 3/15/ % Broadwood Capital (Neal Bradsher, Director) 16.1% Individual Investor 9.2% Insiders & LT Investors 46.7% BioTime shareholder base with significant long term investors A Technological Leader with Revenue, Low Burn Rate and Near-term Product Opportunities Key Statistics:

34 BioTime’s Key Milestones 34 Next 12 months… Initiation of HyStem ® -Rx clinical trial IND filing of OpRegen  for AMD PanC-Dx  patient study Corporate partnering and collaborations Increased revenue from LifeMap BioReagents

35 35 >200 purified human cell types manufactured for the first time Leading patent position Emphasis on near-term products: - Renevia TM - PanC-Dx TM - LifeMap BioReagents Longer–term first-in-class stem cell- based therapeutics for currently incurable degenerative diseases Subsidiaries focused on specific disease areas Positioned to Lead the Commercialization of Pluripotency in Regenerative Medicine Alameda, California Headquarters Summary

36 Thank You Michael West, Ph.D. CEO Pete Garcia CFO


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