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8-K - BIOTIME, INC 8-K 1-12-2015 - Lineage Cell Therapeutics, Inc.form8k.htm

EXHIBIT 99.1
 
 Regenerative Medicine Through PluripotencyNYSE MKT: BTX  January, 2015 

 Safe Harbor Statement  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  Pluripotent stem cell platform provides competitive barriers:Potential to manufacture all human cell types on an industrial scaleRegenerative vs. palliative therapyTargeting large degenerative disease markets 600 patents/patent applications worldwideAttractive manufacturing costsLong asset life - no FDA pathway for generics or biosimilars Anticipating multiple near-termclinical milestonesBuilding board and management for initial commercialization        Pluripotent Stem Cells  OrthopedicsArthritisIVD diseaseTendon repair  EndocrinologyDiabetesObesity  NeurologySpinal cord injuryStrokeParkinsons  CardiologyHeart failureIschemiaHypertension  OphthalmologyMacular degeneration  HematologyImmunotherapy  OncolologyLung Cancer  Leading Regenerative Medicine 
 

 4  Over the next 12 months, BioTime and its subsidiaries arepositioned to increase shareholder value by…  Announcing results from six clinical studiesCommercializing cancer screening diagnosticsCompleting Renevia pivotal clinical trial Implementing clinical and financial de-risking strategiesUnlocking subsidiary company value for BioTime shareholdersBuilding management & Board for commercial phase  Near-Term Value Creation 
 

 Clinical Milestones in 1H 2015  5  Publication and presentation at major scientific meetings on results of clinical studies of the PanC-Dx family of diagnostics addressing lung, bladder and breast cancerInterim data from the pivotal trial of Renevia, a therapeutic product addressing a large market opportunity in lipoatrophyCompletion of enrollment of first cohorts in two Phase 1/2a clinical trials (OpRegen and OPC1) each addressing multi-billion dollar market opportunities 
 

 6  Multiple products, each addressing large market opportunities  Product  Medical Need  Market Opportunity  OPC1  Spinal cord injury  > $20B economic impact in U.S.  VAC2  Non-small-celllung cancer  > $38B economic impact in U.S.  OpRegen  Dry AMD  > $5B annual spend on wet AMD maintenance therapies  Renevia  Lipoatrophy  > 3M HIV patients  PanC-Dx  Cancer diagnostics  $7B annual cost of mammography in U.S.  Product Pipeline 
 

 7  Maximizing shareholder value in a broad platform company  Increases financial flexibility at both the corporate and subsidiary levelLimits direct dilution to existing shareholders of BTXAttracts talented executives with focus on diverse fields of medicineAllows investors to focus on opportunities that most interest themEnables development of more products using our broad technology platformsLimits downside risk by spreading investments among multiple opportunitiesMaintains upside potential as subsidiaries address large market opportunities  Subsidiary Strategy 
 

 Major Therapeutics  8  OPC1  Potential patients in U.S.  Research  Preclinical  Phase 1  Phase 2  Spinal cord injury  12,000/year          Multiple sclerosis  180,000 prevalence          Stroke  800,000/year          VAC1            Prostate cancer  240,000/year          AML  12,000/year          VAC2            Lung cancer                        OpRegen            Dry AMD  7.3 million prevalence           
 

 OPC1 & VAC2  9    Developing Breakthrough Technologies for Regenerative Medicine(NYSE MKT: AST)Pedro Lichtinger, CEOFormer CEO Optimer Pharmaceuticals, and President of Pfizer’s Global Primary Care Unit  9 
 

 OPC1 – Phase 1 Results  10  Feasible and Safe            Five subjects received 2 mil AST-OPC1 cells, followed for 2-3 years Clean safety profile observed to date:No serious adverse events related to surgery, AST-OPC1, or immunosuppressionMonitoring through one year shows no evidence of immune responses to AST-OPC1Potential evidence of biological activity:MRI results in 4 of 5 subjects are consistent with prevention of lesion cavity formationCleared to enter Phase 1/2a trial in target population for initial indication: complete cervical SCI 
 

 OPC1 – Phase 1/2a Trial  11     
 

 12  OPC1 Opportunity: SC Injury 
 

 VAC2 - Proof of Concept  Telomerase is expressed in ~95% of cancer typesVAC1 was safe and stimulated anti-telomerase immune responses in two clinical trials with improved or stabilized biomarkers  13    Phase 1: prostate cancerDukeJ. Immunol 2005, 174:3798  Phase 2: acute myelogenous leukemia Multi-centerKhoury ASH 2010  Patients treated  20  21  Tolerability  Excellent  Excellent  Patients immunized against telomerase  95%  55%  Laboratory & clinical impact  Highly significant increase in PSA doubling timesClearance of circulating immune complexes  Significant increase in 12-month DFS in high-risk group (N=11) compared to published historical controls 
 

 VAC2 – Unmet Medical Needs  14  Advantages of VAC2 Platform         
 

 15  AST-VAC2 – Trial Design 
 

 16  1 Source: Cancer Research Technologies2 Source: National Institutes of Health3 Source: Decision Resources  VAC2 Opportunity: NSC lung cancer 
 

 OpRegen  17    Advancing Medicine Through Stem Cell TherapiesCharles Irving, Ph.D., CEO  17   
 

     AMD and Loss of RPE Cells      OpRegen  Photoreceptors depend on RPE cells for nourishment, recycling of visual pigment and waste disposal When RPE cells die, the photoreceptors also die and central vision is lost Geographic Atrophy  (RPE)   
 

     OpRegen®: Cell replacement therapy for the dry form of age–related macular degeneration (dry-AMD) utilizing a suspension of animal product-free RPE cells produced from human embryonic stem cells. Indication: Geographic atrophy (the severe dry-form AMD)Formulation: Cell suspension in BSS Plus; ready for injection Delivery Route: Subretinal administrationMechanism of Action: Integration into subretinal spaceand replacement of missing or diseased RPE cellsPlanned Clinical Trial: Phase I/IIa trial inprogressive dry-form AMD with GAStudy Site: Hadassah Univ Medical Center, JerusalemStage of Development: FDA & Israel Ministry of Health of Health authorization received  Therapy for Dry-AMD      OpRegen 
 

     Trial Design      OpRegen  Phase I/IIa Dose Escalation Safety and Efficacy Study of OpRegen® Transplanted Subretinally in Patients with Advanced Dry-Form AMD (Geographic Atrophy)Open label, non-randomized, sequential, single center trialStudy Site: Hadassah University Medical Center, Jerusalem, IsraelDose and Administration: Single injection of 50,000-500,000 viable cells in saline delivered via a cannula through a small retinotomy into the subretinal space. Part 1Cohort 1: 3 Patients, BCVA 20/200 or less, 50,000 OpRegen® cellsCohort 2: 3 Patients, BCVA 20/200 or less, 200,000 OpRegen® cellsCohort 3: 3 Patients, BCVA 20/200 or less, 500,000 OpRegen® cellsPart 2Cohort 4: 6 Patients, BCVA 20/100, 500,000 OpRegen® cells 
 

 Device & Diagnostic Pipeline  21  Partner    Platform  Pre-Clinical  Design control /IND enabling  Clinical development      Regulatory Pathway        Safety  Efficacy  Pivotal    HyStem®Renevia            CE Mark as a medical device  HyStem®ReGlyde      N/A  N/A  N/A  510 (k) device  HyStem®Premvia            Cleared Class II device August 2014                Indication  Research  Development  Clinical Validation      Commercialization  Breast      Clinical Studies in Progress      2015  Bladder      Clinical Studies in Progress      2015  Lung      Clinical Studies in Progress      2015  Injectable for the treatment of lipoatrophies  Wound Management  Protection following surgery  Screening/Recurrence Setting  Screening/Recurrence Setting  High Risk Screening  Pivotal 
 

   HyStem - Unmet Medical Needs  22              Polymerizes safely in vivo   Stays as liquid for ~ 20 minutes  Supports cells including adipocytes in 3-D            Cast Hydrogel  Cells in Sponge                                    Injectable  Multiple Formulations                    Durable Films  3-D Lattices  Heparin-mediated Slow Release   
 

 ReneviaTM Pivotal Trial   23  Renevia™ is an injectable matrix designed to safely produce 3-D tissue in vivo, keeping cells where the surgeon places them. It is expected to have numerous applications in multiple tissue typesBioTime initially is seeking a CE mark for use in HIV-associated lipoatrophy in combination with autologous fat cells.An estimated 33M people worldwide have HIV. Number on HIV treatment has tripled in five years – now ~10M, target of 15M by the end of 2015. An estimated 35-50% of patients on ARV therapy have lipoatrophy.A greater number of people in the US have lipoatrophy due to wounds and age.  Age-Related Lipoatrophy 
 

 24    ReneviaTM Pivotal Trial  Multicenter, randomized, controlled, single blind trial     Treated vs. delayed treatment control  25 - 92 subjects in each group with treatment effect measured at 1, 3, and 6 months  Primary Endpoint   Increase in skin thickness as measured by ultrasound at 6 months  Secondary Endpoint  Mid-face volume deficit score Global aesthetic improvement score  Sites  2 sites in Palma de Mallorca, Spain  Trial Design 
 

 25    Advanced Noninvasive Cancer DiagnosticsJoseph Wagner, Ph.D., CEO  25    PanC-Dx 
 

 PanC-Dx – Cancer Diagnostics  26  PanC-DX™  Potential market size  Status  Results Announced  Breast cancer  $2.5 billion  Clinical study underway  1H 2015  Bladder cancer  $500 million  Clinical study underway  1H 2015  Lung cancer  $525 million  Clinical study underway  1H 2015  Novel products for the diagnosis and treatment of cancer in order to improve both the quality and length of cancer patients’ livesInternally developed cancer gene discovery platformPlatform based on extensive microarray datasetMarket discovery principle based on similarity between embryonic development and cancerScores of potential targets identifiedMultiple product opportunitiesDevelop and market low-cost molecular diagnostic tests for major types of cancer     
 

 Balance of Near-Term Products  PanC-Dx™ Breast Cancer Diagnostic: Blood-based screening diagnostic Panel of protein markers$25M initial market opportunity, $2.5B expanded opportunityPanC-Dx™ Bladder Cancer Diagnostic:Urine-based screening diagnostic for recurrence Panel of RNA markers$15M initial market opportunity, $500M expanded opportunityPanC-Dx™ Lung Cancer Diagnostic:Blood-based screening diagnostic Panel of RNA markers$25M initial market opportunity, $525M expanded opportunity  27  Three Diagnostic Products to be Commercialized within 12 Months 
 

 Clinical De-Risking Strategies  OPC1: Initiative to accelerate current timelines for clinical program by about six months to obtain safety and efficacy readouts more rapidlyOPC1: Accelerated trial by six months and announced intent to strengthen robustness of proof of concept by seeking FDA permission to expand the number of patients from 13 to 40PanC-Dx™ and Renevia are judged by the company to be relatively low-risk products  28 
 

     AST-OPC1  AST-VAC2  $14.3 Million Grant  Includes funding for:Execution of Phase 1/2a studyProcess and assay development activities to prepare for pivotal trials and commercializationFacilities and indirect costsPotential follow-on grants to expand and accelerate trial  Clinical Development Partnership  Asterias performs scale-up and tech-transfer of AST-VAC2 manufacturing processCRUK provides personnel and funding for cGMP manufacturing, regulatory filing, Phase 1/2a trialAsterias has first option to reacquire program on preset, reasonable terms; majority revenue share on partner’s development if does not choose to reacquire  Estimated ~$40 Million of Total Non-dilutive Funding  29  Financial De-Risking 
 

 Unlocking Subsidiary Value  30      Asterias Biotherapeutics is the first of BioTime’s subsidiaries to be publicly traded    Timeline  1  Created subsidiary to acquire Geron stem cell assets  2  Closed acquisition of assets  3  Hired a seasoned management team  4  Appointed Pedro Lichtinger as CEO, a leader with significant commercialization expertise  5  Executed clinical and financial de-risking strategies  6  Executed spin-off to public company  7  Listed on NYSE MKT under symbol “AST”  BTX owns 72% of ~$120M AST Market CapBTX has 7 other subsidiaries whose value could be unlockedGoal: Unlock value of one additional subsidiary in 2015 
 

 Building Commercialization Team  31  Appointed Pedro Lichtinger as President and Chief Executive Officer of Asterias, a former Pfizer senior executive with successful drug development and commercialization experienceAdi Mohanty, former Shire executive, appointed COO of BioTimeMichael H. Mulroy and Stephen L. Cartt joined BioTime’s Board of Directors. Mr. Mulroy and Mr. Cartt both had successful careers in senior management at Questcor Pharmaceuticals, Inc.Angus C. Russell, former Chief Executive Officer of Shire plc, appointed to BioTime's Board of Directors   Focused on adding biopharmaceutical industry executives with expertise in clinical development and commercialization  
 

 Improved Product Manufacture  32  Advantages of Pluripotency Platform      Pluripotent Stem CellMaster Cell Bank    Limitless expansionSource of every cell typeCentralized productionUniform geneticsReduced COGsImproved QC  DifferentiationFactors  Frozen Inventory  Point of Use    Clinical:Oligodendrocyte progenitors (SCI)RPE cells (AMD)Preclinical:Dendritic cells (Cancer)Brown adipocytes (Type 2 diabetes)Vascular cells ischemic diseaseCartilage cells (spinal disc repair)Bone cells (spinal fusion) 
 

 Value of the Pluripotent Platform  33      Cell therapy: the next wave – with a potentially long lifespan due to lack of regulatory pathway for generics or biosimilars  Recombinant ProteinsTechnology in maturity phase  Small MoleculesTechnology in decline  Unique and proprietary Pluripotent Stem Cells Extensively characterized to Strict Regulatory Requirements.  hESC MasterCell Bank  Product  Monoclonal AntibodiesTechnology in growth phase  Cell TherapyTechnology in clinical trials phase 
 

 BioTime (NYSE Market: BTX)  34  $156M liquid assets: ~$36M in cash, $3M in registered BTX shares held by subsidiaries, & $117M AST shares held by BTX (as of October 16, 2014)    BTX market cap approx. $290M, owns approx. 70% of AST (market cap approx. $130M (as of January 9, 2015)No debt      Long-term investors hold approx. 45% of BTX stock  Strong Board, Management, and Financial Structure    Key Statistics: