Slide 1: Leading the Regenerative Medicine Revolution
LEADING THEREGENERATIVE MEDICINE REVOLUTION May 2015NYSE MKT: BTX
Slide 2: 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 limited to the succes
Slide 3: Investment Highlights: Building Value at BioTime
RIGHT TIME to consolidate leadership in regenerative medicine ROBUST PIPELINE addressing large, degenerative diseases UNIQUE CELL DELIVERY PLATFORM: ReneviaTM pivotal trial underway MULTIPLE CLINICAL TRIALS advancing products to commercialization UNLOCKING SUBSIDIARY VALUE (NYSE MKT: AST, $230M1) LE
Slide 4: Regenerative Medicine: The Next Revolution
Gene cloningtechnology developed Hybridomatechnology developed Isolation of pluripotentstem cells 2015 1970 1980 1990 2000 2010 1974 1975 1998 RECOMBINANT DNA MONOCLONAL ANTIBODIES REGENERATIVE MEDICINE 2010: FIRST-IN-HUMAN TRIAL OF OPC1 $44BNCURRENT GLOBAL MARKET $75BNCURRENT GLOBAL MARKET Regenera
Slide 5: Cell Therapy Has Long Asset Life Potential
Small MoleculesTechnology in decline Monoclonal AntibodiesTechnology in growth phase Cell TherapyTechnology in clinical trials phase Cell Therapy Has Long Asset Life Potential Product Recombinant ProteinsTechnology in maturity phase hESC MasterCell Bank 5 Lack of regulatory pathway for generics or b
Slide 6: Stem Cell Platform Enables Multiple Opportunities
Pluripotent stem cell platform allows industrial manufacture of all humancell typesSignificant competitive barriers:>600 patents/apps worldwideLarge degenerativedisease markets Multiple near-term clinical milestonesBioTime and subsidiaries have multiple opportunities for value creation Stem Cell Pla
Slide 7: Broad Portfolio Advancing in Clinic
HIV-associated lipoatrophy CELL THERAPIES Broad Portfolio Advancing in Clinic CELL DELIVERY MATRIX Renevia DIAGNOSTICS PLATFORM PanC-Dx Pre-Clinical Phase I Phase II Phase III/Pivotal OpRegen OPC1 VAC2 Dry AMD Spinal cord injury rehabilitation NSC lung cancer Screening diagnostics for bladder, lung,
Slide 8: OpRegen has Proven Mechanism in Dry AMD
Retinal Pigment Epithelium (RPE) Choroid Photoreceptors Drusen Age-Related Macular Degeneration (AMD) OpRegen has Proven Mechanism in Dry AMD Photoreceptor function, angiogenesis inhibition depend on RPE cells Loss of RPE cells can cause dry or wet AMD OpRegen integrates into subretinal space to rep
Slide 9: Targeting Larger Dry AMD Opportunity
hES Cell-Derived RPE Cell Replacement Therapy for Dry AMD Targeting Larger Dry AMD Opportunity 9 800,000 Cases in U.S. Annually 90% Dry AMDNo Approved Therapy 10% Wet AMD>$7B Market Globally
Slide 10: OpRegen Phase I/IIa: First Patient Dosing 2Q15
OpRegen Phase I/IIa: First Patient Dosing 2Q15 Cohort 3 ● 3 Patients BCVA 20/200 or less 500,000 cells TRIAL DESIGNPart 1 Cohort 4 ● 6 Patients BCVA 20/100 or less 500,000 cells Cohort 2 ● 3 Patients BCVA 20/200 or less 200,000 cells Cohort 1 ● 3 Patients BCVA 20/200 or less 50,000 cells Part 2 Phas
Slide 11: OPC1: Promise in Spinal Cord Rehabilitation
Demyelination of neurons impairs rehabilitation from spinal cord injury, plays a role in multiple sclerosis, other diseases Transplantation of oligodendrocyte progenitors can remyelinate damaged nerve axons, improving recovery from spinal cord injury in extensive rat model studies Oligodendrocyte Pr
Slide 12: OPC1: Phase I/IIa Trial Underway
after 30 Days dose five pts with 20 million AST-OPC1 cells OPC1: Phase I/IIa Trial Underway TRIAL DESIGNSequential Cohort, Dose Escalation Dose three pts with two millionAST-OPC1 cells after 30 Days dose five pts with 10 million AST-OPC1 cells Subject to FDA clearance, expansion of second and third
Slide 13: VAC1: Proof-of-Concept in Multiple Cancers
VAC2 is the next-generation allogeneic vaccine advancing toward clinic studies 13 Telomerase is an unprecedented target - abnormally expressed in ~95% of cancer types VAC1: Proof-of-Concept in Multiple Cancers Patient-specific dendritic cells can train immune cells to attack cancer Phase I study in
Slide 14: Need for Cell Delivery is Significant
Most cell therapies without matrix support have reported engraftment of less than 5% Most human cells naturally die if not rapidly attached to matrix Need for Cell Delivery is Significant 14 Current known options not easily adapted to include cells and matrix for delivery to tissues in vivo using a
Slide 15: Renevia: Novel Injectable Cell Delivery Matrix
3-Dimensional Cell Delivery Matrix Renevia™: Novel Injectable Cell Delivery Matrix 15 Renevia™: Novel Injectable Cell Delivery Matrix
Slide 16: Applications in Multiple Types of Facial Atrophy
Applications in Multiple Types of Facial Atrophy ReneviaTM designed to safely produce 3-D tissue in vivoCells remain where placed by surgeon~350,000 in EU have HIV-related lipoatrophy$5,000-7,000 spent per patient annually for currently available treatment optionsTrauma or age-related lipoatrophy ev
Slide 17: Renevia: Pivotal Trial Initiated 1Q15
Renevia™: Pivotal Trial Initiated 1Q15 Secondary Endpoint- Mid-face volume deficit score - - Global aesthetic improvement scale - TRIAL DESIGNMulticenter, randomized, controlled, single-blind trial Treated vs. delayed treatment control25 completers in each group with treatment effect measured at 1,
Slide 18: PanC-Dx: Platform with Broad Oncology Potential
8 Million Patients Screened Annually in U.S. LUNG CANCER 18 PanC-Dx™: Platform with Broad Oncology Potential Noninvasive Cancer Screening Diagnostics Less invasive, easily implemented, novel blood and urine-based diagnostic Potential for expanded use in numerous solid tumor types 3.5 Million Diagnos
Slide 19: Financial Strength for Growth
Financial Strength for Growth ASSETS $283M Liquid Assets $26M Cash1 $27M Registered BTX shares2 held by subsidiaries $230M AST shares2(BTX owns 68%) No Debt ~$452M BioTime Market cap3 ~45% Shares owned bylong-term stockholders 1 At March 31, 2015, 2 Fair value of AST shares and BTX shares based on M
Slide 20: Financial De-Risking: Non-Dilutive Funding
$20-30 million $14.3 million Grant Financial De-Risking AST-OPC1 AST-VAC2 OpRegen $42-52 million of Total Non-Dilutive Funding Includes funding for: Execution ofPhase 1/2a studyAssay developmentFacilities and indirect costs CRUK provides fundingfor personnel, cGMP manufacturing, regulatory filing, P
Slide 21: Significant Stakes in Key Subsidiaries
Significant Stakes in Key Subsidiaries 68% % BTX Ownership as of March 31, 2015 63%2 75% 1 Fair value of AST shares held by BioTime based on May 1, 2015 2 Includes shares owned by BioTime, Asterias, and ESI 21 $230M 1
Slide 22: Leadership Team
Leadership TeamScientific, Clinical Development, Commercial Expertise LEADER ROLE EXPERIENCE Michael D. West, Ph.D. President andChief Executive Officer Ocata Therapeutics, Geron (Founder) Adi Mohanty Chief Operating Officer Shire, Baxter EXPERIENCED BOARD OF DIRECTORS + 3 RECENT ADDITIONS WITH COMM
Slide 23: Near-Term Value Creation
Near-Term Value Creation Results from multiple clinical studies Commercializing cancer screening diagnostics Completing Renevia pivotal clinical trial enrollment Implementing clinical and financial de-risking strategies Unlocking subsidiary company value for BioTime shareholders Building management
Slide 24: Leading the Regenerative Medicine Revolution
LEADING THEREGENERATIVE MEDICINE REVOLUTION NYSE MKT: BTX