Exhibit 99.1
Leading the Revolution in Regenerative Medicine NYSE MKT: BTX February, 2015
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 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.
Timing of Investment in Biotech Recombinant DNA Monoclonal Antibodies Regenerative Medicine Advent: 1974: Gene cloning technology developedOpportunity: Industrial-scale manufacture of all proteinsHurdle: 1976: moratorium on rDNA research Launch: 1989: EPO is first billion dollar product2015: products using rDNA technology are ubiquitous>140 clinical trials$75bn current global market Advent: 1975: Hybridoma technology developed Opportunity: Manufacture of diverse & specific antibodiesHurdle: HAMA responseLaunch: 1997: Rituximab first billion-dollar product2014: 8 of the 20 best-selling biotechnology drugs are therapeutic monoclonal antibodies>200 clinical trials$44bn current global market Advent: 1998: Isolation of pluripotent stem cellsOpportunity: Manufacture of diverse & specific cell types“It is not too unrealistic to say that this research has the potential to revolutionize the practice of medicine.” – H. Varmus 1998Hurdle: 2001: U.S. federal funding restriction (reversed in 2009) 2010: First-in-human trial of OPC1 Biotech Revolutions: 15-20 Years to Commercialization *
Value of the Pluripotent Platform 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 at a Glance Pluripotent stem cell platform allows industrial manufacture of all human cell typesSignificant competitive barriers: >600 patents/apps worldwideTargeting large degenerative disease markets Multiple near-term clinical milestonesSolid balance sheetUnique subsidiary structure provides multiple opportunities for value creation
* Includes shares owned by BioTime, Asterias, and ESI. Subsidiary Company BTX Ownership 67% 63%(1) 75% Key BioTime Subsidiaries
* Addressing large market opportunities Product Unmet Medical Need Patient Population (US) OpRegen Dry AMD 8,000,000 (prevalence) OPC1 Spinal cord injury rehabilitation 12,000/yr VAC2 Non small cell lung cancer ~200,000/yr Renevia Cell transfer matrix for HIV lipoatrophy >360,000 (prevalence) PanC-Dx Screening diagnostics for bladder, lung, and breast cancer >40 million/yr Multiple Products in Development
* 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
Age-Related Macular Degeneration (AMD) OpRegen Photoreceptor function and angiogenesis inhibition depend on RPE cells Loss of RPE cells can cause either the dry or wet forms of AMD Retinal PigmentEpithelium (RPE) Choroid Photoreceptors Drusen 9
Strategy: hES cell-derived RPE cell replacement therapy for the dry form of AMDMechanism of Action: Integration into subretinal space and replacement of missing RPE cellsPrevalence: Wet AMD represents only 10% of the cases; Dry AMD represents ~ 90% of casesMarket Potential: Wet AMD therapies currently generate ~ $7 billionThere are no FDA-approved Dry AMD therapies Therapy for Dry-AMD The OpRegen Opportunity PMEL17HuNu *
OpRegen Clinical Trial Design Phase I/IIa dose escalation safety and efficacy study of OpRegen transplanted subretinally in patients with advanced dry-form of 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 cells in saline delivered into the subretinal space. Part 1Cohort 1: 3 Patients, BCVA 20/200 or less, 50,000 cellsCohort 2: 3 Patients, BCVA 20/200 or less, 200,000 cellsCohort 3: 3 Patients, BCVA 20/200 or less, 500,000 cellsPart 2Cohort 4: 6 Patients, BCVA 20/100, 500,000 cells *
Oligodendrocyte Progenitors OPC1 Demyelination of neurons impairs rehabilitation from spinal cord injury, plays a role in multiple sclerosis, and other diseases Transplantation of oligodendrocyte progenitors can remyelinate damaged nerve axons improving recovery from spinal cord injury in extensive rat model studies Oligodendrocyte Nerve CellAxon *
OPC1 – Previous Phase 1 Trial * Feasible and Safe Five subjects received 2 mil OPC1 cells, followed for >4 years Clean safety profile observed to date:No serious adverse events related to surgery, OPC1, or immunosuppressionNo unexpected neurological changesNo adverse changes on MRIMonitoring through one year shows no evidence of immune responses to OPC1Potential evidence of biological activity:MRI results in 4 of 5 subjects are consistent with prevention of lesion cavity formation
OPC1 – Current Phase 1/2a Trial *
VAC1 - Proof of Concept * • Telomerase is an unprecedented target - abnormally expressed in ~95% of cancer types• Patient-specific dendritic cells can train immune cells to attack cancer• Phase I study in prostate cancer appeared safe, increased PSA times1• Phase II study in AML appeared safe, increased DFS2• Off-the-shelf hES cell-derived DCs could improve QC, reduce costs, and speed delivery of therapy to patients J. Immunol 2005, 174:3798Khoury ASH 2010
* 1 Source: Cancer Research Technologies, 2 Source: National Institutes of Health. 3 Source: Decision Resources VAC2: ESC-Derived Dendritic Cells Application in Non-Small Cell Lung CA
* VAC2 – Trial Design
Renevia - Unmet Medical Needs * Injectable 3-Dimensional Tissue Matrix
ReneviaTM * 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 typesPivotal trial for CE mark for use in HIV-associated lipoatrophy in combination with autologous lipotransfer now underwayEstimated 3.5M people worldwide have HIV-related lipoatrophy In addition, a greater number of people have lipoatrophy due to trauma or agingMany other potential applications in combination with adult and ESC therapies Age-Related Lipoatrophy
* ReneviaTM Pivotal Trial Multicenter, randomized, controlled, single blind trial Multicenter, randomized, controlled, single blind trial Treated vs. delayed treatment control 25 completers 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 scale Enrollment First patient in Q1’15. Multiple sites in Spain Trial Design
PanC-Dx * PanC-DX™ Potential market size Status Initial Results to be Presented Breast cancer $2.5 billion Clinical study underway April 2015 Bladder cancer $500 million Clinical study underway April 2015 Lung cancer $525 million Clinical study underway 1H 2015 Novel blood and urine-based cancer screening diagnostics Designed to provide increased accuracyLess invasive and designed to be easily implementedPotential for expanded use in numerous solid tumor types Noninvasive CA Screening Diagnostics
Near-Term Commercialization PanC-Dx™ Breast Cancer Diagnostic: Blood-based screening diagnostic Panel of protein markersPanC-Dx™ Bladder Cancer Diagnostic:Urine-based screening diagnosticPanel of RNA markersPanC-Dx™ Lung Cancer Diagnostic:Blood-based screening diagnostic Panel of RNA markers * Three Diagnostic Products Planned to Launch within 12 Months
AST-OPC1 AST-VAC2 $14.3 Million Grant Includes funding for:Execution of Phase 1/2a studyAssay developmentFacilities and indirect costsPotential follow-on grants to expand and accelerate trial $40-50 Million of Total Non-dilutive Funding 15 Financial De-Risking $20-30 Million CRUK provides funding for personnel, cGMP manufacturing, regulatory filing, Phase 1/2a trialAsterias has first option to reacquire program on preset, reasonable terms or majority revenue share OpRegen ~ $5 Million Funding of pre-clinical studies leading to IND filingNondilutive grant with potential for follow-on funding
* 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
Unlocking Subsidiary Value * Asterias Biotherapeutics is the first of BioTime’s subsidiaries to be publicly traded BTX owns 72% of ~$120M AST Market CapBTX has seven other subsidiaries whose value could be unlockedGoal: Unlock value of one additional subsidiary in 2015 Alternative slide of previous slide with Graphic image to show sequence of timeline starting from left to right.
Building Commercialization Team * 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
BioTime (NYSE Market: BTX) * $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)No debt Revenues of $1.2M and $3.4M during the three and nine months ended September 30, 2014, respectivelyBTX market cap approx. $220M, owns 72% of AST (market cap approx $156M (as of October 16, 2014) Long-term investors hold approx. 45% of BTX stock Strong Board, Management, and Financial Structure Key Statistics:
Summary Powerful pipeline based on pluripotent stem cellsEmphasis on degenerative diseases with relatively large market potentialBalance of near- and long-term products significant milestones in 2015Executing clinical/financial de-risking strategiesBuilding commercially-focused management team and Board Unlocking subsidiary value (AST first listing on NYSE)Long potential asset life due to lack of biosimilars *