Lineage Cell Therapeutics, Inc. · AMEX:LCTX
Lineage Cell Therapeutics — Biotime — Investor Presentation (February 2015)
28 slides from Lineage Cell Therapeutics's investor presentation, Feb 24, 2015
Slide 1: Leading the Revolution in Regenerative Medicine
Leading the Revolution in Regenerative Medicine NYSE MKT: BTX February, 2015
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 n
Slide 3: Timing of Investment in Biotech
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: product
Slide 4: Value of the Pluripotent Platform
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 Extensive
Slide 5: BioTime at a Glance
* 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 pro
Slide 6: Key BioTime Subsidiaries
* Includes shares owned by BioTime, Asterias, and ESI. Subsidiary Company BTX Ownership 67% 63%(1) 75% Key BioTime Subsidiaries
Slide 7: Addressing large market opportunities
* 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-D
Slide 8: Near-Term Value Creation
* 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 9: Age-Related Macular Degeneration (AMD) OpRegen
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
Slide 10: The OpRegen Opportunity: Therapy for Dry-AMD
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 current
Slide 11: OpRegen Clinical Trial Design
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,
Slide 12: Oligodendrocyte Progenitors OPC1
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
Slide 13: OPC1 – Previous Phase Trial
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
Slide 14: OPC1 – Current Phase 1/2a Trial
OPC1 – Current Phase 1/2a Trial *
Slide 15: VAC1 - Proof of Concept
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, incre
Slide 16: VAC2: ESC-Derived Dendritic Cells in Non-Small Cell Lung Cancer
* 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
Slide 17: VAC2 — Trial Design
VAC2 — Trial Design Indication: -small Cell Lung Cancer Objectives: Safety and preliminary efficacy SCASTERIAS Immune blockade inhibitor trials demonstrate sensitivity of lung cancer to immunotherapy Proof of concept for telomerase antigen in lung cancer High level of unmet medical need with current
Slide 18: Renevia - Unmet Medical Needs
Renevia - Unmet Medical Needs * Injectable 3-Dimensional Tissue Matrix
Slide 19: Renevia
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
Slide 20: Renevia Pivotal Trial Design
* 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
Slide 21: PanC-Dx Noninvasive Cancer Screening Diagnostics
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 scr
Slide 22: Near-Term Commercialization
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
Slide 23: Financial De-Risking
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 fo
Slide 24: Subsidiary Strategy: Unlocking Subsidiary Value
* 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 25: Unlocking Subsidiary Value
Unlocking Subsidiary Value e ° BIOTIME Asterias Biotherapeutics is the first of BioTime’s subsidiaries to be publicly traded Created subsidiary . to acquire Hired a Geron seasoned stem cell management assets team Closed acquisition of assets Executed clinical and financial de-risking strategies Appo
Slide 26: Building Commercialization Team
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 Step
Slide 27: BioTime Key Statistics
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.
Slide 28: Summary
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 B