Lineage Cell Therapeutics, Inc. · AMEX:LCTX
Lineage Cell Therapeutics — Biotime — Investor Presentation (January 2015)
35 slides from Lineage Cell Therapeutics's investor presentation, Jan 12, 2015
Slide 1: Regenerative Medicine Through Pluripotency
Regenerative Medicine Through PluripotencyNYSE MKT: BTX January, 2015
Slide 2: Safe Harbor Statement
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 n
Slide 3: Leading Regenerative Medicine
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 -
Slide 4: Near-Term Value Creation
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
Slide 5: Clinical Milestones in 1H 2015
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 opp
Slide 6: Multiple products, each addressing large 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 Lip
Slide 7: Subsidiary Strategy
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 t
Slide 8: Major Therapeutics
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
Slide 9: OPC1 & VAC2 Developing Breakthrough Technologies for Regenerative Medicine
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
Slide 10: OPC1 – Phase 1 Results Feasible and Safe
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-O
Slide 11: OPC1 — Phase 1/2a Trial
OPC1 — Phase 1/2a Trial ASCASTERIAS Indication: + High level of unmet medical need — no approved therapies, high Complete level of disability & lifetime cost of care Cervical Spinal * Clear path to market — endpoint measurements established by Cord Injury SCOPE initiative Establish safety of AST-OPC
Slide 12: OPC1 Opportunity: SC Injury
12 OPC1 Opportunity: SC Injury
Slide 13: VAC2 - Proof of Concept
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-center
Slide 14: VAC2 – Unmet Medical Needs
VAC2 – Unmet Medical Needs 14 Advantages of VAC2 Platform
Slide 15: AST-VAC2 — Trial Design
AST-VAC2 — Trial Design FRASTERIAS . . . + Immune blockade inhibitor trials demonstrate sensitivity of lung Indication: cancer to immunotherapy Non-small Cell + Proof of concept for telomerase antigen in lung cancer Lung Cancer + High level of unmet medical need with current therapeutic regime Objec
Slide 16: VAC2 Opportunity: NSC lung cancer
16 1 Source: Cancer Research Technologies2 Source: National Institutes of Health3 Source: Decision Resources VAC2 Opportunity: NSC lung cancer
Slide 17: OpRegen Advancing Medicine Through Stem Cell Therapies
OpRegen 17 Advancing Medicine Through Stem Cell TherapiesCharles Irving, Ph.D., CEO 17
Slide 18: AMD and Loss of RPE Cells
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)
Slide 19: OpRegen: Therapy for Dry-AMD
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
Slide 20: Trial Design OpRegen Phase I/IIa
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
Slide 21: Device & Diagnostic Pipeline
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 Indicatio
Slide 22: HyStem - Unmet Medical Needs
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
Slide 23: Renevia Pivotal Trial
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
Slide 24: Renevia Pivotal Trial Design
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 End
Slide 25: Advanced Noninvasive Cancer Diagnostics PanC-Dx
25 Advanced Noninvasive Cancer DiagnosticsJoseph Wagner, Ph.D., CEO 25 PanC-Dx
Slide 26: PanC-Dx – Cancer Diagnostics
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
Slide 27: Balance of Near-Term Products
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 mar
Slide 28: Clinical De-Risking Strategies
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 permissio
Slide 29: Financial De-Risking: Clinical Development Partnership
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
Slide 30: Unlocking Subsidiary Value
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 sig
Slide 31: Building Commercialization Team
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 Ste
Slide 32: Improved Product Manufacture
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 progenitor
Slide 33: Value of the Pluripotent Platform
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 Extens
Slide 34: Strong Board, Management, and Financial Structure
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 invest
Slide 35: Summary
Summary = BIOTIME * Balance of near- and long-term products ¢ Emphasis on degenerative diseases with relatively large market potential * Significant milestones in 2015 ¢ Executing clinical/financial de-risking strategies * Building commercially-focused management team and Board ¢ Unlocking subsidiar