EX-99.1 2 ex99_1.htm EXHIBIT 99.1

Exhibit 99.1
 
 NYSE MKT: BTX  Rodman & Renshaw16th Annual GlobalInvestment ConferenceSeptember 10, 2014 
 

 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. 
 

 *  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/ patent applications worldwideNo approval pathway for generics or biosimilars to our cell therapies  The Technology Leader in Regenerative Medicine  Investment Highlights  Pluripotent Stem Cells  OrthopedicsArthritisIVD diseaseTendon repair  EndocrinologyDiabetesObesity  NeurologySpinal cord injuryStrokeParkinsons  CardiologyHeart failureIschemiaHypertension  OphthalmologyMacular degeneration  HematologyImmunotherapy  OncolologyLung Cancer 
 

 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 trials$75bn current global market  Advent: 1975: Hybridoma technology developed Hurdle: HAMA responseLaunch: 1997: Rituximab is 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 cellsHurdle: 2001: U.S. federal funding restriction (reversed in 2009) 2010: First-in-human trial of OPC1 Future: First billion-dollar product  *  Biotech revolutions often take 15-20years from conception to commercialization 
 

 Value of the Pluripotent Platform  *  Cell therapy: In growth phase – potentially long lifespan due to lack of regulatory pathway for generics or biosimilars 
 

 Targeting High Medical Need  VAC2 – Cancer, telomerase immunotherapy $38B economic impact of non-small-cell lung cancer in the U.STargeted by Asterias Biotherapeutics (BioTime owns 70.6%)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%)OpRegen – Dry AMD, retinal pigment epithelial cells>$5B annual spend on drug therapies for wet AMDCell Cure Neurosciences (BioTime owns 62.5%)PanC-Dx – Cancer diagnostic, breast, lung, bladder CA$7B annual cost of mammography in the USTargeted by OncoCyte (BioTime owns 75.3%)Renevia – HyStem, device for lipoatrophy>3M HIV+ patients with lipoatrophyRenevia developed by BioTime (owns 100%)  * 
 

 Recent Milestones  3Q14: FDA clearance to initiate Phase 1/2a clinical trial of OPC1 for complete cervical spinal cord injury3Q14: ASTY becomes first publicly-traded subsidiary of BioTime3Q14: FDA premarket notification clearance for Premvia™3Q14: OncoCyte initiates large multi-site clinical trial forPanC-Dx ™ bladder cancer diagnostic2Q14: ASTY awarded $14.3MM research grant from CIRM to fund Phase 1/2a clinical trial of OPC12Q14: OncoCyte partners with Weill Cornell for expanded lung cancer diagnostic clinical study2Q14: ASTY presents OPC1 Phase 1 clinical data at ASGCT scientific meeting2Q14: ASTY raises $13MM through sales of equity1H14: BTX raises $10MM through sales of equity  * 
 

 Rapid Cadence of Milestones  2H14: ASTY anticipates completing negotiations for partnership to fund Phase 1/2a of VAC2 development2H14: Cell Cure Neurosciences files OpRegen® IND with FDALate 2014: Three clinical trials for OncoCyte’s PanC-Dx ™ (bladder, breast, and lung cancers) expected to be completed4Q14: Renevia™ pivotal clinical trial4Q14-2Q15: results of OncoCyte’s three PanC-Dx ™ trials expected to be published in peer-reviewed journals and presented at major scientific meetings1Q15: OPC1 clinical trial patient enrollment expected to beginMid-2015: Potential PanC-Dx ™ commercializationLate 2015: Potential Renevia™ commercialization  * 
 

 Major Therapeutics  *  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  *  Developing Breakthrough Technologies for Regenerative MedicinePedro Lichtinger, CEOFormer CEO Optimer Pharmaceuticals, and President of Pfizer’s Global Primary Care Unit  * 
 

 OPC1 – Unmet Medical Needs  *  The First hESC-Derived Product in the Clinic  Safe and feasible in world’s first clinical trial of human embryonic stem cell-derived therapyExtensive data in non-clinical animal studies demonstrates:-- Engraftment periods of > 1 year-- Extensive cavity filling and myelination-- Improved locomotor function-- No observed toxicity, immunogenicityCleared to enter Phase1/2a study in target population for initial commercial indication (complete cervical SCI)Follow-on opportunities in MS, Stroke, other neurodegenerative diseases  AST-OPC1 
 

 *  Indication: OPC1 is a cryopreserved cell product containing human embryonic stem cell (hESC) derived oligodendrocyte progenitor cells (OPCs) for the treatment of subacute spinal cord injury (SCI). OPC1 has three reparative functions (production of neurotrophic factors, induction of remyelination, & induction of vascularization) that promote tissue repair & return of function in the injured spinal cord.  Multicenter, open-label, dose escalation trial Three consecutive dose cohorts, 13 total subjectsTreatment effect measured at 6 and 12 monthsPrimary endpoint: Safety with respect to: OPC1, the surgical procedure for administration, and the transient immunosuppressionSecondary endpoint:Neurological function as measured by upper extremity motor scores and motor level in the cervical spinal cord3-4 sites in the United StatesDuration (enrollment and follow up): About 36 months  OPC1 – Phase 1/2a Clinical Trial 
 

 *  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  *    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  *  Advantages of VAC2 Platform 
 

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

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

 OpRegen® – Unmet Medical Needs  *  Current wet AMD market estimated at > $5B  7.3 million have early stage dry AMD in US (10x wet form)Any effective treatment expected to achieve blockbuster salesOpRegen® – Suspension of retinal pigment epithelial (RPE) cells for dry age-related macular degeneration (AMD) OpRegen® Plus – Matrix bound RPE cells for dry AMDPartnered with TEVA  http://www.amd-therapy.de/en/investment/market-and-competition 
 

 *  Indication: OpRegen® is a suspension of allogeneic human retinal pigment epithelium (RPE) cells intended to replace diseased or missing RPE cells in patients with geographic atrophy, the severe stage of dry form age-related macular degeneration. OpRegen® is intended to be transplantated subretinally using existing surgical skills.   • Dose escalation safety trial • 15 patients divided into three cohorts each of three legally blind patients, followed by a fourth cohort of six patients with better vision that will receive the maximum dose. All patients will be followed up for 12 months post-transplantation. • Primary endpoint: Safety and tolerability of OpRegen®• Secondary endpoint: Measure of the ability of OpRegen® cells to engraft, survive, and moderate disease progression • Single site – Hadassah Medical Center, Jerusalem • Duration (enrollment and initial follow up): 23-26 months  OpRegen® – Phase I/IIa Clinical Trial 
 

 OpRegen Opportunity: Dry AMD  *  1 Source: http://www.amd-therapy.de/en/investment/market-and-competition 
 

 Device & Diagnostic Pipeline  *  Partner  Platform  Pre-Clinical  Design control /IND enabling  Clinical development  Clinical development  Clinical development  Regulatory Pathway  Platform  Pre-Clinical  Design control /IND enabling  Safety  Efficacy  Pivotal  Regulatory Pathway  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  Clinical Validation  Clinical Validation  Commercialization  Breast      Clinical Studies in Progress  Clinical Studies in Progress  Clinical Studies in Progress    Bladder      Clinical Studies in Progress  Clinical Studies in Progress  Clinical Studies in Progress    Lung      Clinical Studies in Progress  Clinical Studies in Progress  Clinical Studies in Progress    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  *  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 
 

 Renevia - Medical Need  *  Based on animal studies ReneviaTM is expected to have numerous applications in the stable engraftment of CNS, heart, osteochondral, liver, adipose, and other cell types.BioTime initially is seeking a CE mark to apply the technology to HIV-associated lipoatrophy using autologous ASF cells.An estimated 33M people worldwide have HIVAn estimated 35% of patients on ARV therapy have lipoatrophyA greater number of people in the US have age-related lipoatrophy  Age-Related Lipoatrophy 
 

 *  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 adipocytes.   • Multicenter, randomized, controlled, single blind, trial • Treated vs. delayed treatment control, 25 - 45 subjects in each group with treatment effect measured a 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 • Two sites in Palma de Mallorca, Spain • Duration (enrollment and follow up): 18 - 24 months  ReneviaTM – Pivotal Clinical Trial 
 

 2014 Milestones – HyStem  *  Initiation of Renevia™ pivotal clinical trial510(k) clearance for Premvia™2014-15 file 510(k) application for ReGlyde™ 
 

 *  1 Source: AIDS Care Vol. 21, No. 5, May 2009, 664-671  Renevia Opportunity: Lipoatrophy 
 

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

 PanC-Dx – Cancer Diagnostics  *  PanC-DX™  Potential market size  Status  Results expected  Breast cancer  $2.5 billion  Clinical study underway  Late 2014  Bladder cancer  $500 million  Clinical study underway  Late 2014  Lung cancer  $525 million  Clinical study underway  Late 2014  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 
 

 *  Proposed Use: The PanC-Dx™ breast cancer diagnostic is a multi-analyte, blood-based test designed to i) detect presence of malignancy in otherwise asymptomatic patients, and ii) detect recurrence of disease in patients with a past history of breast cancer   • Single multicenter cohort-design study • Up to 800 patients will be enrolled at time of diagnostic mammography (including 200 cancer-positive) • Primary endpoint: Correlation of biomarkers with BI-RADS score • Secondary endpoint: Correlation of biomarkers with pathology • Currently enrolling at Scottsdale Medical Imaging • Anticipated completion: late 2014  PanC-Dx – Breast Cancer Study 
 

 *  Proposed Use: The PanC-Dx™ bladder cancer diagnostic is a multi-analyte, urine-based test designed to detect i) recurrence of disease in patients with a past history of bladder cancer, and ii) presence of bladder cancer in patients presenting with hematuria   • Two multicenter cohort-design studies • Up to 1100 patients will be enrolled at time of cystoscopy (including at least 150 cancer-positive) • Primary endpoint: Correlation of biomarkers with cystoscopy and cytology results • Secondary endpoint: Correlation of biomarkers with pathology • First study underway in US; sites currently being selected for second study • Anticipated completion first study: late 2014 • Anticipated completion second study: mid 2015  PanC-Dx – Bladder Cancer Study 
 

 *  Proposed Use: The PanC-Dx™ lung cancer diagnostic is a multi-analyte, blood-based test designed to distinguish benign from malignant nodules in high-risk patients undergoing surveillance low-dose CT   • Two cohort-design studies at 7 total sites • Approximately 730 patients have been enrolled to date (including over 300 cancer positive) • Primary endpoint: Correlation of biomarkers with pathology • Secondary endpoint: Correlation of biomarkers with stage, tumor type • Studies being conducted in collaboration with the Wistar Institute and Weill Cornell Medical College • Anticipated completion both studies: mid 2014   PanC-Dx – Lung Cancer Study 
 

 PanC-DX™ - Opportunity #1: Breast CA  *  Blood-based screening diagnostic measures sera protein biomarkers using ELISA. Clinical study underway & in discussions with additional sites 
 

 PanC-DX™ - Opportunity #2: Bladder CA  *  Urine-based multiplex PCR recurrence diagnostic test. Ongoing patient studies in U.S. & China with additional sites to be selected 
 

 PanC-DX™ - Opportunity #3: Lung CA  *  Blood-based lung cancer screen for high-risk populationMulti-center study underway with high interest from KOLs 
 

 BioTime (NYSE Market: BTX)  *  $24.8 MM available: ~$8.5 MM available in cash plus $16.3 MM in registered BTX shares held by subsidiaries that are available for sale (as of September 5, 2014)No debt  Revenues of $1.1 MM and $2.2 MM during the three and six months ended June 30, 2014, respectivelyBTX market cap approx. $223 MM, owns 72% of ASTY (market cap approx $191 MM (as of September 5, 2014)  Long-term investors hold approx. 40% of BTX stock  Strong Board, Management, and Financial Structure  Key Statistics: 
 

 Long-Term Value Creation Strategy    BioTime  Traditional Biotech  Potential to be the overall leader in the next generation of biotech?  YES  NO  Broad technology base with many product opportunities?  YES  NO  Products not easily vulnerable to generic or biosimilar competition?  YES  NO  Broad IP portfolio?  YES  NO  Spinoff technologies with near-term market potential?  YES  NO  Frugal spending history?  YES  NO  History of funding programs partially through non-dilutive research grants?  YES  NO  Not primarily a binary bet on a single product in clinical trials?  YES  NO  * 
 

 BioTime – Investment Opportunity  *  Positioned as the technology leader in the coming regenerative medicine revolution