Lineage Cell Therapeutics, Inc. · AMEX:LCTX
Lineage Cell Therapeutics — BioTime — Corporate Update (November 2014)
137 slides from Lineage Cell Therapeutics's corporate update, Nov 4, 2014
Slide 1: Shareholder Update: The Regenerative Medicine Revolution
Shareholder UpdateThe RegenerativeMedicine Revolution November 4, 2014
Slide 2: Safe Harbor Statement
Safe Harbor Statement 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
Slide 3: The Rise of the Aged Baby Boomers
The Rise of the Aged Baby Boomers Source of data on projected growth of US population: US Census Bureau Investment Highlights
Slide 4: Rectangularization of Survivorship Curve
4 Rectangularization of Survivorship Curve Investment Highlights
Slide 5: Aging: The Rogue Wave
5 Aging: The Rogue Wave Investment Highlights
Slide 6: The Tsunami of Chronic Age-Related Degenerative Disease
6 The Tsunami of Chronic Age-Related Degenerative Disease Investment Highlights CDD due to lack of cellular regeneration80% Americans have CDD of aging50% of Americans have two CDDs95% costs in aging is CDD
Slide 7: Advent of Regenerative Medicine
Advent of Regenerative Medicine Recombinant DNA Monoclonal Antibodies Regenerative Medicine Advent: 1974: Gene cloning technology developedHurdle: 1976: moratorium on rDNA research Launch: 1989: EPO is first billion dollar product2014: products using rDNA technology are ubiquitous>140 clinical trial
Slide 8: The Mission
The Mission 8 To lead in the application of pluripotent stem cell technology for regenerative therapeutic applications in age-related degenerative disease.
Slide 9: The Technology Leader in Regenerative Medicine
9 Poised to commercialize one of the largest revolutions in medicineTargeting large markets in degen-erative diseases ($billion markets)Multiple clinical milestones in 2H2014Balance of near-term and longer-term products in developmentLeader in core pluripotent stem cell technology with >600 patents/
Slide 10: hESC-Based Manufacturing
hESC-Based Manufacturing 10 Any functional cell type can be producedPermanently functional replacement cells Scalable batch production Pluripotency Indefinite Replicative Capacity Not Possible With “Tissue Sourced” Stem Cells Two Defining Characteristics of hESCs Not Possible With “Tissue Sourced” S
Slide 11: Pluripotent vs. Adult Stem Cells
Pluripotent vs. Adult Stem Cells 11 PluripotentStem Cells PluripotentStem Cells PluripotentStem Cells AdultStem Cells Pluripotent stem cells allow for the first time in history the capacity of medicine to produce all human cell types
Slide 12: Value of the Pluripotent Platform
Value of the Pluripotent Platform 12 Cell therapy: In growth phase – 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
Slide 13: OPC1: hESC-Derived Oligodendrocyte Progenitor Cells
Safe and feasible in world’s first clinical trial of human embryonic stem cell-derived therapyFDA clearance for expansion of Phase I/IIa trial in cervical SCIInitiation of CIRM-funding of 3 yr $14M grantAsterias listed NYSE MKTFollow-on opportunities in MS, Stroke, other neurodegenerative diseases 1
Slide 14: Milestones – VAC2 & OpRegen
Milestones –VAC2 & OpRegen 14 Complete Process Development of VAC2CRUK to fund Phase I/II clinical trial in Lung CAOpRegen IND Cleared for Phase I/IIa OpRegen (RPE Cells) VAC2 (Dendritic Cells)
Slide 15: Near-Term Strategic Products HyStem Matrices
Near-Term Strategic Products 15 HyStem Matrices
Slide 16: LifeMap Sciences, Inc.
LifeMap Sciences, Inc. • More than 14 Million page views from more than 3,000 institutions world-wide, including academia, research hospitals, patent offices, and leading biotech and pharma• Premium services now being offered for a feeLifeMap Solutions developing mobile health applications 16 Integr
Slide 17: Targeting High Medical Need
Targeting High Medical Need OPC1 – Spinal cord injury, oligodendrocytes$20B annual economic impact of spinal cord injury in the U.S.Developed by Asterias Biotherapeutics (BioTime owns 70.6%)VAC2 – Cancer, telomerase immunotherapy $38B economic impact of non-small-cell lung cancer in the U.STargeted
Slide 18: Balanced Strategy
Balanced Strategy 18 Diversified, lower-cost, lower-risk strategyNot a bet on a single clinical programMultiple, growing revenue streams currentlySeven clinical phase programs by Q4 2014Internal focus on low-cost, low-risk, near-term programsPartnering strategy for select high-cost potential blockbu
Slide 19: BioTime – Investment Opportunity
BioTime – Investment Opportunity 19 Positioned as the technology leader in the coming regenerative medicine revolution
Slide 20: Asterias Biotherapeutics (NYSE MKT: AST)
Asterias Biotherapeutics(NYSE MKT: AST)Leveraging Proprietary Regenerative Medicine Platforms to Address Significant Unmet Medical Needs
Slide 21: Forward Looking Statements
Forward Looking Statements Statements pertaining to future financial and/or operating results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects express
Slide 22: Key Investment Highlights
Two potential transformative platforms: 1) industry leading Pluripotent Stem Cells; 2) Allogeneic Dendritic Cell ImmunotherapyTwo products entering dose escalation clinical trialsPartnerships with leading scientific institutions provide validation and access to significant, non-dilutive capital (~$4
Slide 23: Additional Investment Considerations
Regenerative medicine in emerging growth phase and approaching valuation inflection pointClinical development focus on de-risked, proof-of-concept initial product indicationsDeep, experienced management team with scientific and business expertise 4 Additional Investment Considerations
Slide 24: Recent Key Accomplishments
Successfully completed stock and warrant distribution to GERN shareholders, became publicly traded and listed on NYSE MKT under ticker “AST”Received FDA clearance for Phase 1/2a dose escalation trial of AST-OPC1 in complete cervical spinal cord injuryLaunched partnership with California Institute of
Slide 25: Asterias' Programs Built on Transformative Technology Platforms
Asterias’ Programs are Built on Potentially TransformativeTechnology Platforms 6 Major scientific validation by top scientists in multiple countriesDe-risked by extensive nonclinical data & long term clinical safety data
Slide 26: Asterias' Programs Built on Transformative Technology Platforms
Asterias’ Programs are Built on Potentially TransformativeTechnology Platforms 7 Major scientific validation by top scientists in multiple countriesDe-risked by clinical data for first generation, autologous product
Slide 27: Large Markets with Unmet Needs: AST-OPC1
Asterias’ Products Address Large Markets with Significant Unmet Medical Needs AST-OPC1 for Neurodegenerative Diseases Lead indication: Spinal Cord InjuryDevastating injury affecting 12,000 patients per year in US1No currently approved therapiesLifetime cost of care per patient of $2-4 mil1Healthcare
Slide 28: Large Markets with Unmet Needs: AST-VAC2
Asterias’ Products Address Large Markets with Significant Unmet Medical Needs AST-VAC2 (Cancer Immunotherapy) Unique mode of action in emerging field of Immune Cancer TherapeuticsCancer Immunotherapy market projected to reach $35 bil in annual revenues by 20241Partial HLA mismatch from allogeneic va
Slide 29: AST-OPC1: Oligodendrocyte Progenitor Cells
AST-OPC1: Oligodendrocyte Progenitor Cells 10 First hESC-Derived Product in Clinic Phase 1 successfully completedExtensive data in animal studies demonstrates:Engraftment over periods of at least 1 yearExtensive cavity filling and myelinationImproved locomotor functionNo observed toxicity or immunog
Slide 30: AST-OPC1 Phase 1 Trial in Complete Thoracic Injuries
AST-OPC1 Phase 1 Trial in Complete Thoracic Injuries 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 immunosuppressionNo unexpected neurological changes to dateNo adverse changes on M
Slide 31: AST-OPC1 Phase 1/2a Trial in Complete Cervical SCI
AST-OPC1 Phase 1/2a Trial in Complete Cervical SCI 12
Slide 32: Allogeneic hESC-DC Vaccine Platform
Asterias’ Allogeneic hESC-DC Vaccine Platform 13 A New Class of Cancer Immunotherapy Existing proof-of-concept data for dendritic cell vaccines from first generation, autologous productsSignificant scalability, cost and feasibility advantages of hESC-derived allogeneic dendritic cell platformPotenti
Slide 33: Proof of Concept from First Generation AST-VAC1
Proof of concept for Dendritic Cell Telomerase Immunotherapy from First Generation AST-VAC1 Product Prostate Cancer Acute Myelogenous Leukemia Phase 1, Single center (Duke) Phase 2, Multi-center Investigator-sponsored Industry-sponsored 20 patients 21 patients 95% Patients Develop Immune Responses t
Slide 34: Rationale for Telomerase
Rationale for Telomerase 15 Telomerase Antigen:Expressed in >90% of human cancersRarely expressed in normal adult cellsPlays critical role in conferring replicative immortality to cancer cellsEvidence for immunogenicity from previous trials Hanahan and Weinberg, “The Hallmarks of Cancer”
Slide 35: AST-VAC2 Phase 1/2a Trial in NSCLC
AST-VAC2 Phase 1/2a Trial in Non-small Cell Lung Cancer (NSCLC) 16
Slide 36: Partnerships Provide Validation and Non-dilutive Funding
Partnerships Provide Validation and Non-dilutive Funding at Minimal Cost to AST Shareholders 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 cos
Slide 37: Asterias Milestones and Newsflow Next 24 Months
18 Asterias Milestones and Newsflow Next 24 Months 1H’15 2H’15 1H’16 Initiate AST-OPC1 P1/2a Trial 6 mo efficacy data from AST-OPC1 10M cell cohort Initiate AST-VAC2 P1/2a Trial MHRA Clearance for AST-VAC2 P1/2a Trial 30 day safety of 2M cell cohort; dose escalate to 10M cell cohort Complete AST-VAC
Slide 38: Opportunities for Future Pipeline Expansion
19 Significant Opportunities for Future Pipeline Expansion AST-OPC1 Broad potential applications in neurodegenerative disease:Multiple sclerosisStrokeAmyotrophic lateral sclerosisLeukodystrophies (e.g. Canavan disease) AST-VAC2 Telomerase immunotherapy in additional cancer indicationsUse of hESC-DC
Slide 39: Asterias Has Sufficient Funds to Advance Lead Programs
Asterias Has Sufficient Funds to Advance its Lead Programs in 2014 and 2015 20 6.9* 4.4ᵻ Cash 12.4** BioTimestock PotentialWarrantProceeds 11.7*** 12-16 $14.3 M in non-dilutive funding for AST-OPC1 project in 2014-2018 from CIRMEstimated $20-30M cost avoidance from AST-VAC2 CRUK deal * As of 6/30/14
Slide 40: Capital Structure Profile
Capital Structure Profile 21 Total 30.9 million outstanding shares valued at $130 mil ($4.20 per share) as of October 31, 2014Additional 5 million Warrants @ $2.34/warrant expiry June 2015 – held by George Karfunkel Trust and Broadwood Partners, LLPAdditional 3.5 million Warrants @ $5.00/warrant exp
Slide 41: Asterias Executive Team
Asterias Executive Team: Business Focused, with Best-in-Class Cell Therapy Product Development Experience Name Title Summary of experience Pedro Lichtinger Chief Executive Officer Former President & CEO, Optimer Pharmaceuticals. 25 year career at Pfizer including as President of Global Primary Care
Slide 42: Asterias
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Slide 43: Strong, Multilayer IP and Market Protection
Strong, Multilayer IP and Market Protection for Asterias’ Products 24 Confidential Additional market protection expected from:Lack of biosimilars pathway for cellular therapiesOrphan designations (7 year market exclusivity) for OPC1 in SCI, VAC2 in certain cancer indications Example U.S. issued pate
Slide 44: AST-OPC1 Is Safe and Efficacious in Animal Models of SCI
AST-OPC1 Is Safe and Efficacious in Animal Models of SCI 25 Control AST-OPC1 Uninjured Injury + OPC1 Injury + Control Four restorative functions observed:Secretes factors to stimulate nerve growthIncreases myelination of axonsStimulates new blood vessels in injury siteReduces injury-related cavitati
Slide 45: AST-OPC1 Reduces Cavity Formation and Induces Myelination
Asterias Neurology Program AST-OPC1 Reduces Cavity Formation and Induces Persistent Myelination Myelinated Bundles of Nerve Fibers Traverse Injury 9 months vehicle hNucEC 1 mm 100 µm 50 µm Cavity forms in untreated SCI lesion Myelinated axons do not extend across cavity Brown: antibody to human nucl
Slide 46: OPC: Evidence for Efficacy in Cervical SCI
OPC: Evidence from Literature for Efficacy in Cervical SCI Asterias: Proprietary and Confidential 27 Transplant Vehicle Transplant Vehicle Spared White Matter at Epicenter Spared Gray Matter at Epicenter Area (um2) Area (um2) p<0.01 1,000,000800,000600,000400,000200,000 3,000,0002,000,0001,000,0000
Slide 47: SCOPE Consortium Defined Clinical Development Path
Work of the SCOPE Consortium Has Defined Clinical Development Path in Complete Cervical Spinal Cord Injury 28 Target Product Profile for AST-OPC1 in Complete Cervical SCI: Increase of ≥20% in the percentage of patients regaining two or motor levels of function Enables powering of trial with only ~20
Slide 48: AST-OPC1 Improves Locomotor Recovery in SCI Rats
AST-OPC1 Improves Locomotor Recovery in SCI Rats Increased Weight BearingImproved HL-FL Coordination Improved Hind Paw Clearance Improved Trunk StabilityDecreased Tail Drag Increased Stride LengthDecreased Stride WidthDecreased Paw RotationDecreased Toe Spread 2.8 29 Confidential
Slide 49: Rapid Execution Expanding Clinical Development of AST-OPC1
Asterias has Rapidly Executed on Expanding Clinical Development of AST-OPC1 to Complete Cervical SCI Worked with FDA to define path to clinic for hESC-derived therapyPerformed >20 nonclinical studies to enable first in man testing of AST-OPC1Resolved two clinical holds to satisfaction of FDAEnrolled
Slide 50: Spinal Cord Injury Represents a Substantial Market Opportunity
Spinal Cord Injury Represents a Substantial Market OpportunityDue to A High Level of Unmet Need, Lack of Competing Therapies 31 Confidential Estimated Market Opportunity of $300M-$3B in U.S. aloneDriven by magnitude of impact on high lifetime costs of care for cervical ($3-4M) and thoracic ($2.3M) i
Slide 51: AST-VAC2 Has Properties of Mature Dendritic Cells
AST-VAC2 Has Expected Properties of Mature Dendritic Cells Confidential and Proprietary 32 Expresses Markers of Mature Dendritic CellsActivates T CellsMigrates in Response to InflammationCan be Cryopreserved and Irradiated Without Loss of Function g-IFN AST-VAC2 AST-GFP AST-VAC2 Stimulates Telomeras
Slide 52: AST-VAC2 Phenotypically Similar to PBM-Derived DC
hESC-derived Mature DC PBM-derived Mature DC AST-VAC2 and PBM-derived mature DC show similar DC marker expression profiles Tseng et al, Regen Med. 2009 AST-VAC2 is Phenotypically Similar to PBM-Derived DC Confidential 4.2 33
Slide 53: hESC-DC Vaccines Likely Synergistic With Checkpoint Inhibitors
hESC-DC Vaccines Are Likely to Be Synergistic With Immune Checkpoint Inhibitors 34 Immune Checkpoint Inhibitors Block Pathways that Tumors Use to Evade Immune Surveillance AST-VAC2 is the Only Allogeneic Dendritic Cell Cancer Immunotherapy That Educates the Immune System to Recognize Antigens on Tum
Slide 54: POC Data Enables Broad Development of hESC-DC Platform
POC Data from Phase 1/2a Study of AST-VAC2 in NSCLC Enables Broad Development of hESC-DC Platform 35 Demonstration of safety and immune responses in NSCLC study Demonstration of safety and immune responses in NSCLC study Phase 2 and pivotal trials of AST-VAC2 in NSCLC Expansion of AST-VAC2 into addi
Slide 55: Human embryonic stem cell-derived products for retinal and neurodegenerative diseases
Human embryonic stem cell –derived products for the treatment of retinal and neurodegenerative diseases Dr Charles Irving, CEO 04 Nov 2014 BIOTIME ANNUAL MEETING SHAREHOLDER UPDATE
Slide 56: Safe Harbor Statement
Safe Harbor Statement 1 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 n
Slide 57: Dry AMD: A Major Incurable Disease of Aging
Leading eye disease responsible for visual impairment of the elderly in the US, EU and AustraliaLoss of fine visual detail; Central vision blind spots; Mostly affects both eyes; 10% progress to wet AMD and legal blindness In the US:1.6 million new cases/yr of dry-AMD7.3 million with early stage dry-
Slide 58: Types of Age-Related Degeneration
Types of Age-Related Degeneration Dry AMD (non-neovascular) is characterized by drusen and RPE changes which lead to central atrophy of photoreceptors. Geographic atrophy (GA) is the severe form of dry-AMD.No FDA approved therapy.Wet AMD (neovascular) can develop from dry-AMD. Pathological neovascul
Slide 59: Role of RPE Cells in Dry AMD
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 Role of Retinal Pigment Epithelium (RPE) Cells in Dry AMD (RPE)
Slide 60: RPE Transplantation Therapy
October ‘09 RPE RPE Transplantation Therapy: A Promising ApproachedThat Lacked a Reliable Source of RPE Cells Healthy RPE cells can replace old, dysfunctional RPEsHuman Embryonic Stem Cells (hESCs) could provide an unlimited supply of well-characterized RPE cells, if a method better than spontaneous
Slide 61: Production of RPE by Directed Differentiation of hESCs
Production of RPE by Directed Differentiation of hESCsy Adherent Polygonal Pigmented Cell Culture Clinical Grade hESCsGrown on Cord Feeders Spheroid Bodies NicotinamideLow O2 Nicotinamide + Activin ALow O2 Adherent Culture (Pigmented and Non-Pigmented Areas) Selection of Pigmented Areas and Expansio
Slide 62: IP Status of Directed Differentiation Method
IP Status of Directed Differentiation Method Cell Cure has exclusive license from Hadasit Ltd.Publ. #: WO2008/129554; Publ. Date: 30-Oct-2008GrantedJapan – 5,395,058; Australia – 2008242106; China -200880020748.0; Israel -201600 AllowedEurope PendingUSA, Canada, Hong Kong, India, various divisionals
Slide 63: World's Largest Patent Estate in Pluripotency
The BioTime family of companies has the world’s largest patent estate in pluripotency with >600 patents and patent applications worldwide.Examples of relevant IP:Feeder free culture of hES cellsDirected differentiation methodsNeuronal differentiation (NCAM positive lineages)License to WARF estateRPE
Slide 64: Xeno-Free, Clinical-Grade hESCs for Manufacturing RPE Cells
Xeno-Free, Clinical-Grade, NIH -Registered hESCs: A Source for Manufacturing Xeno-free RPE Cells 102
Slide 65: Cell Cure's RPE Cell Product for Dry AMD: OpRegen
Cell Cure’s RPE Cell Product for Dry AMD: OpRegen® OpRegen®: Cell replacement therapy for the dry form of age–related macular degeneration (dry-AMD) utilizing a suspension of RPE cells produced from human embryonic stem cells (hESCs). Cells: Allogeneic human RPE cells produced under cGMP conditions
Slide 66: CMC Characterization of OpRegen
Morphology: Cells can form a monolayer of polygonal pigmented cellsIdentity: Cells express the RPE markersPurity: Total non-RPE cellular impurities <1.7% show using a new proprietary assay. No hESC impurity in neither the final product or even at early stage of productionPotency: Combined ability of
Slide 67: OpRegen Preclinical Studies Results
OpRegen® Preclinical Studies Results Three independent CRO studies conducted: Safety/Biodistribution , Tumorigenicity and Spiking and EfficacyNo teratomas or human tumors found neither in 328 NOD/SCID mice nor in 180 RCS rats that received a maximal feasible dose of OpRegen®No teratomas found with O
Slide 68: Preclinical Studies: Vision Rescue in RCS Rat
Highlights of Preclinical Studies: Vision Rescue in RCS Rat Historical Data:
Slide 69: Preclinical Studies: Long Term Graft Survival
Highlights of Preclinical Studies: Long Term Graft Survival Long term (9 months –post transplant) engrafting of viable OpRegen® cells (pigmented cells stained positive for HuNu and PMEL17) in NOD/SCID subretinal space
Slide 70: Preclinical Studies: Cells Form Monolayer
OpRegen® cells are clustered at the place that the bleb was formed and then organize themselves into the monolayer structure found in healthy animals Human PMEL17Human Nuclei Highlights of Preclinical Studies: Cells Form Monolayer Human PMEL17Human Nuclei Human PMEL17Human Nuclei
Slide 71: OpRegen-Mediated Rescue of Photoreceptors
OpRegen® -Mediated Rescue of Photoreceptors Vehicle OpRegen® HuNu+PMEL17+ (OpRegen®) Rescued Photoreceptors; ONL ONL Thickness (number of nuclei layers) in OpRegen® treated eyes is above vehicle control (group 2) at all time points Group 2: VehicleGroup 3: 25,000 OpRegen®Group 4: 100,000 OpRegen®Gro
Slide 72: OpRegen Mediated Phagocytosis in Treated RCS Rats
OpRegen® Mediated Phagocytosis in Treated RCS Rats Rat RhodopsinHuman PMEL17 Rat RhodopsinHuman PMEL17 Rat rhodopsin outer segments (green) rest along the grafted cells (human PMEL17, red) and rat rhodopsin within transplanted OpRegen® cells (yellow)
Slide 73: Cell Cure Phase I/IIa Clinical Study (1)
Cell Cure Phase I/IIa Clinical Study (1) 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, J
Slide 74: Phase I/IIa Clinical Study (2)
Primary Objective: Safety and tolerability of transplanted OpRegen®Secondary Objective: Initial exploration of the ability of transplanted OpRegen® to engraft, survive, and moderate disease progression Duration and Follow Up: Enrollment period plus 12 months post transplant follow-up; weekly during
Slide 75: Phase I/IIa Clinical Study Efficacy Outcome Measures
Selection of outcome measures based on an ongoing “Cohort Study” that tracks disease progression in patients, who might be candidates for the Phase I/II StudyVisual Acuity Rate of GA Progression over 6 and 12 monthsRetinal sensitivity to light in the engrafted regionsExtent and depth of central stom
Slide 76: Cell Cure Company Overview
Company Overview Platform Technology Manufacturing of diverse cell products from xeno-free, GMP-grade human embryonic stem cellsLead product - OpRegen®, human Retinal Pigment Epithelium Cells History Founded in 2005 in Jerusalem, IsraelMajor investments by BioTime starting in 2010 Present Shareholdi
Slide 77: OncoCyte Corporation Shareholder Update
Joseph Wagner, Ph.D. Chief Executive OfficerOncoCyte CorporationShareholder UpdateNovember 4, 2014
Slide 78: OncoCyte Corporation
OncoCyte Corporation 2 Our mission is to develop novel products for the diagnosis and treatment of cancer in order to improve both the quality and length of life of cancer patients Internally developed cancer gene discovery platformPlatform based on extensive microarray datasetMarker discovery princ
Slide 79: OncoCyte Technology
OncoCyte Technology 3 Many of the same genes that drive embryonic development drive tumor formation and growth A substantial proportion of the mammalian genome is exclusively activated during embryonic developmentThese embryo-exclusive genes regulate:Cell proliferationCell signalingOrgan formationAn
Slide 80: OncoCyte's Product Development Path
OncoCyte’s Product Development Path 4 Translating proprietary cancer marker platform into near-term revenue opportunities in oncology markets Cancer Marker Discovery (2011-):Assembled >700 sample microarray dataset Bioinformatics generated >3000 candidate markersProtection of Intellectual Property (
Slide 81: OncoCyte's Business Formula
OncoCyte’s Business Formula 5 Designed to translate proprietary cancer marker platform into near-term revenue opportunities “Listen to Your Technology”“Listen to Your Customers”Create Scalable Business ModelEarly Revenues Fund Larger Trials
Slide 82: Listen to Your Technology
“Listen to Your Technology” 6 Technology platform defines product opportunities in subsectors of cancer diagnostics Potential products based on dataset structure:Screening diagnosticsRecurrence diagnosticsPotential products based on markers:Breast cancerColorectal/GI cancersBladder/urothelial cancer
Slide 83: Listen to Your Customer
“Listen to Your Customer” 7 Physician adoption is the key barrier to entry for cancer screening diagnostics User Will Adopt Tests That:Key Opinion Leaders supportResolve diagnostic dilemmasJustify the need for proceduresEliminate unnecessary proceduresHave reasonable cost/reimbursementUser Will Not
Slide 84: OncoCyte Products: Business Strategy
OncoCyte Products: Business Strategy 8 How does OncoCyte identify and prioritize its products? Strategically develop diagnostic products that will be rapidly adopted and have short- and long-term revenue potentialFocus on developing products that:Fit with proprietary technologyAre simple to perform
Slide 85: Product Development & Marketing Strategy
Product Development & Marketing Strategy 9 Create a scalable business model beginning with an immediate market with early adopters, successively expanding into larger opportunities $15M Initial Initial Use & Market:Early adopters with immediate needsCan be addressed rapidly with relatively low costC
Slide 86: Key Driver: Limitations of Current Tests
Key Driver: Limitations of Current Tests 10 Initial users/use will be to clarify diagnostic dilemmas due to uncertainties in imaging and other traditional tests Current Tests With Significant Limitations Include:Mammography for breast cancerUrine cytology for bladder cancer recurrence surveillanceLo
Slide 87: Current PanC-Dx Product Programs
Current PanC-Dx™ Product Programs 11 Simple, low cost cancer screens that address current unmet user needs in large patient markets Breast Cancer Diagnostic (BR-001):Blood-based screening diagnostic Panel of protein markersBladder Cancer Diagnostic (BL-002):Urine-based screening diagnostic for recur
Slide 88: ONC-BR-001: Breast Cancer Product
12 Initial test use late in diagnostic tree with progressive data driving test use earlier and earlier ONC-BR-001: Breast Cancer Product
Slide 89: ONC-BR-001: Breast Cancer Product
13 ONC-BR-001: Breast Cancer Product Initial test use late in diagnostic tree with progressive data driving test use earlier and earlier
Slide 90: ONC-BR-001: Breast Cancer Product
ONC-BR-001: Breast Cancer Product 14 Blood-based screening diagnostic measures sera protein biomarkers using ELISA $15M $25M Initial Use & Market:Radiologist: Management of BIRADS 3-4 patients350K tests/year in USExpanded Use & Market:Radiologist: In conjunction with all diagnostic mammographyOncolo
Slide 91: ONC-BR-001: Breast Cancer Clinical Study
ONC-BR-001: Breast Cancer Clinical Study 15 Prospective, multisite study in diagnostic mammography patients Subjects:N = 600At time of diagnostic mammographyEndpoints:Primary: Correlation with BIRADS scoreSecondary: Correlation of biomarker with pathologySites:Up to 6 totalCurrently enrolling: Ron K
Slide 92: ONC-BL-002: Bladder Cancer Product
16 Initial test use late in diagnostic tree with progressive data driving test use earlier and earlier ONC-BL-002: Bladder Cancer Product
Slide 93: ONC-BL-002: Bladder Cancer Product
17 Initial test use late in diagnostic tree with progressive data driving test use earlier and earlier ONC-BL-002: Bladder Cancer Product
Slide 94: ONC-BL-002: Bladder Cancer Diagnostic
ONC-BL-002: Bladder Cancer Diagnostic 18 Urine-based multiplex PCR recurrence diagnostic test $15M $15M Initial Use & Market:Pathologist: Resolution of indeterminate cytology during recurrence surveillance500K tests/year in USExpanded Use & Market:Urologist: All recurrence surveillance1.5M test/year
Slide 95: ONC-BL-002: Bladder Cancer Clinical Studies
ONC-BL-002: Bladder Cancer Clinical Studies 19 Multisite clinical studies in setting of surveillance cytology testing or cystoscopy Subjects:N = 100/1200At time of surveillance cytology/cystoscopyEndpoints:Correlation of markers to cytology/cystoscopy resultsSites:Cytopathology: Johns HopkinsCystosc
Slide 96: ONC-LN-003: Lung Cancer Product
20 Initial test use late in diagnostic tree with progressive data driving test use earlier and earlier ONC-LN-003: Lung Cancer Product
Slide 97: ONC-LN-003: Lung Cancer Product
21 Initial test use late in diagnostic tree with progressive data driving test use earlier and earlier ONC-LN-003: Lung Cancer Product
Slide 98: ONC-LN-003: Lung Cancer Product
ONC-LN-003: Lung Cancer Product 22 Blood-based lung cancer screen for high-risk population Initial Use & Market:Radiologists/thoracic surgeons: Management of need-to-biopsy decision350K tests/year in USExpanded Use & Market:Radiologists/thoracic surgeons: Management of all LDCT+ patients1.75M tests/
Slide 99: ONC-LN-003: Lung Cancer Clinical Study
ONC-LN-003: Lung Cancer Clinical Study 23 Ongoing study in target population initiated by collaborators at the Wistar Institute Subjects:N = 600All high-risk, includes cancer-free, benign nodules, confirmed cancer patientsEndpoints:Primary: Correlation with diagnosisSecondary: Correlation with tumor
Slide 100: Adoption Strategy
Adoption Strategy 24 Superior product at lower cost to drive rapid adoption in initial markets, followed by widespread adoption KOL endorsementsDrive adoption within KOLs’ own practices as well as among other physiciansCan lead to medical society recommendationsStrong Clinical Trial DataSupports pos
Slide 101: Upcoming Development Milestones
Upcoming Development Milestones 25 Positive clinical trial results will drive commercialization efforts with first test launch in late 2015 Q4 2014: Completion of breast, bladder and lung clinical trial enrollment and data analysisPublication of lung trial results; submission of clinical study data
Slide 102: OncoCyte: Investment Highlights
OncoCyte: Investment Highlights 26 Beginning with an immediate market with early adopters, expanding into large opportunities Broad platform of cancer markersCost-effective tests using standard methodsPotential for rapid initial adoption and broad eventual adoptionScalable business model:Significant
Slide 103: HyStem Product Development
HyStem® ProductDevelopment November 4, 2014Thomas I. Zarembinski, PhD MBASenior Director, New Product Development
Slide 104: Safe Harbor Statement
Safe Harbor Statement 1 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 n
Slide 105: Introduction
Introduction 2 Background on the need for HyStemCompetitive LandscapeStrategyMarketsProduct Pipeline & Development Status
Slide 106: The Need for HyStem
The Need for HyStem 3 + = UnprecedentedOpportunities inRegenerativeMedicine Cells Matrix • Cells need a matrix for attachment or they often die• An injectable matrix capable of polymerizing in vivo into 3-D tissue could revolutionize medicine
Slide 107: The Need for HyStem: Defined Minimal ECM
The Need for HyStem 4 The Elegance of Defined Minimal ECM One Component Two Components Multi-Component ReGlyde - Adhesion prevention Potential applications in arthritis Renevia – Cell transplants Premvia – Wound manage- ment Controlled growth factor release - Stroke - Cancer Prestwich, J. Cell. Bioc
Slide 108: Two Component Matrix
Two Component Matrix 5 Biocompatible & Biodegradable Carbohydrates Collagen Cell (Griffith and Schwartz, 2006)
Slide 109: The Need for HyStem
The Need for HyStem 6 Addition of Bioactives (Growth factors, Cells) 20 min Safely polymerizes to make 3-D tissue with cells in the body
Slide 110: HyStem Technology Strengths
HyStem® Technology Strengths Strong IP – composition of matter & uses 2 U.S. patents issued with applications pending in the EU, Canada, Japan, and AustraliaEase of manufacture Modest capital investment - large gross margins (65% - 90%)Multiple products in rapidly emerging fields Stem cell applicati
Slide 111: Competitive Landscape
Competitive Landscape 8 Animal-derivedHydrogels forResearch Solid matrices
Slide 112: Competitive Landscape
9 pre-gelled dermal fillers can’t be mixed with cells and often don’t have the requisite attachment sites for cells. Dermal Fillers Viscosupplementation Competitive Landscape
Slide 113: Examples of Applications
Examples of Applications 10 Wu et al Ann Thorac Surg 2011
Slide 114: Examples of Applications
11 in a matrix Wu et al Ann Thorac Surg 2011 Examples of Applications
Slide 115: HyStem Strategy
HyStem® Strategy 12 In the US - a medical device: HyStem® alone – CDRH as a Class II (510K) or Class III (PMA). HyStem® + drug – CDER as a combination new therapy. HyStem® + cells – CBER as a combination new therapy. In the EU - a medical device: HyStem® alone – Notified Body as a Class III. HyStem®
Slide 116: Quality Management Systems Required for Regulatory Approval
Quality Management Systems 13 Required for Regulatory Approval
Slide 117: HyStem Product Pipeline
HyStem® Product Pipeline 14 Partner Platform Early Pre-Clinical Formulation Development /IND-Enabling Studies Clinical development Regulatory Pathway Safety Efficacy Pivotal Marketed HyStem® In-House R&D Medical device in US & EU ReGlyde™ N/A N/A N/A Medical device in USClass II 510(k) Premvia™ 510(
Slide 118: Premvia
Premvia 15 Premvia™- an FDA-cleared device to treat dermal wounds and burns.
Slide 119: Premvia - Markets
Premvia - Markets 16 Change in Segment Share of Global Wound Mgt Market, 2012-2021 MedMarket Diligence, LLC Change in Market Share of Total, 2012-2021 -15% 0% 15% Traditional Dressings Growth Factors MedMarket Diligence, LLC
Slide 120: Premvia Hydrogel + Bioactive
17 Premvia Hydrogel + Bioactive
Slide 121: Premvia—510(k) Clearance
Premvia—510(k) Clearance 18
Slide 122: Renevia - Medical Need
Renevia - Medical Need 19 ReneviaTM is expected to have numerous applications in multiple tissue types.BioTime 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
Slide 123: Renevia – Pivotal Clinical Trial
20 Indication: Renevia™ is a resorbable matrix to be used for the delivery of autologous adipose derived cells for the treatment of HIV-related facial lipoatrophy. Renevia™ serves as a temporary 3-dimentional matrix in which the implanted cells can attach, proliferate and differentiate into adipocyt
Slide 124: Primary Clinical Trial Site
Primary Clinical Trial Site 21 …….The Stem Center, Palma de Mallorca, Spain Ramon Llull, MD, PhD - Medical Director
Slide 125: Summary of Accomplishments
Summary of Accomplishments 22 Obtained ISO-13485 Certificate of Quality SystemsPremviaTM (Wound Management): Received 510(k) clearanceReneviaTM (Repair of Contour Defects): Pivotal Trial starting soon.
Slide 126: Thank You!
Thank You! Thomas I. Zarembinski, PhD MBASr. Director, New Product Developmenttzarembinski@biotimemail.com510-521-3390
Slide 127: mHealth Industry Overview
lifemap-solutions.com © LifeMap Solutions Inc. 2014. Proprietary and confidential mHealth Industry Overview BioTime Shareholder Update November 4, 2014
Slide 128: Safe harbor statement
Safe harbor statement 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
Slide 129: LifeMap Solutions: mission
LifeMap Solutions: mission Working in partnership with the Icahn School of Medicine at Mount Sinai, LifeMap Solutions seeks to develop an mHealth platform that integrates disparate sources of information to generate an increasingly accurate picture of an individual’s health.
Slide 130: Key partner: Mount Sinai
Key partner: Mount Sinai Top-20 hospital Top-20 research medical school 36,000 employees, including 6,200 physicians 170,000 admissions yearly 2.6M non-ER outpatient visits yearly 490,000 ER visits yearly Direct partner is Icahn Institute for Genomics and Multiscale Biology, run by Eric Schadt, Ph.D
Slide 131: Experienced team
Experienced team MULTIVERSE (Netscape =r «) KONTIKI 2 ASOM Verisign
Slide 132: mHealth overview
mHealth overview Today: mostly consumer apps and “wearables”
Slide 133: Major new entrants signal 2nd phase
Major new entrants signal 2nd phase
Slide 134: Market growth is accelerating
Market growth is accelerating Source: Allied Market Research ( http://www.alliedmarketresearch.com/mobile-health-market ) Predicted Global mHealth Market Size (Billions USD)
Slide 135: Investment is accelerating
Investment is accelerating Source: RockHealth Q3 2014 Update ( http://rockhealth.com/2014/10/q3-funding-update-digital-health-rakes-3b/ ) Venture Funding of Digital Health Companies (2011 - Q3 2014)
Slide 136: Why now?
Why now? Culture Ubiquity of smartphones “Quantified Self” movement Expectation of accelerating tech Technology Powerful processors High bandwidth High-quality sensors Big data analytics and management tools
Slide 137: Beneficiaries of mHealth
Beneficiaries of mHealth Consumers Care Teams Direct Researchers Indirect Researchers All enabled by big data