Lineage Cell Therapeutics, Inc. · AMEX:LCTX
Lineage Cell Therapeutics — Investor Presentation (January 2020)
56 slides from Lineage Cell Therapeutics's investor presentation, Jan 13, 2020
Slide 1: Lineage Cell Therapeutics Corporate Overview
=_ == LINEA —- CELL THERAPEUTICS www.lineagecell.com Lineage Cell Therapeutics Corporate Overview NYSE American: LCTX ail
Slide 2: Forward-Looking Statements
-Looking Statements This presentation is for informational purposes only and is not an offer to sell or a solicitation of an offer to buy any securities of Lineage Cell Therapeutics, Inc. (“Lineage”). This presentation includes certain information obtained from trade and statistical services, third-
Slide 3: Lineage is a clinical-stage cell therapy company
Lineage is a clinical-stage cell therapy company which manufactures and transplants various cell types to treat injuries and disease OpRegen® OPC1 VAC2 Immunotherapy is AMD is the leading Lifetime care for an "poised to cause of irreversible SCI patient can cost revolutionize vision loss in the US n
Slide 4: Why Invest in Lineage?
Why Invest in Lineage? Lineage is well-positioned for near-term growth and long-term value © © 3 clinical-stage World class One of the Funded well Leader in the programs with in-house GMP largest patent into 2021* with emerging field billion dollar manufacturing portfolios in cell cost-efficient of
Slide 5: Our Cell Therapy Programs
Our Cell Therapy Programs Three Allogeneic (“Off-the-Shelf”) Treatments for Three Serious Conditions OpRegen® OPC1 VAC2 RPE cells for Dry Age- Related Macular Degeneration with GA (dry AMD) Oligodendrocytes for Dendritic cells for Oncology Spinal Cord Injury (SCI) (Non-Small Cell Lung Cancer, NSCLC)
Slide 6: Validating Partnerships and Funding
Validating Partnerships and Funding Partnerships Programs Phase | Phase Il & External Funding FEY nowina nw & AL > Israel Innovation Dry Age-Related Macular Degeneration L_______y < 47 Authority with GA (Dry AMD) $16M OpRegen® OPC1 : Cl R mM Spinal Cord Injury (SCI) eee” >$14M 2 CANCER 4 RESEARCH UK
Slide 7: Lineage Technology Platform
Lineage Technology Platform * The Lineage Platform starts with | ie normal pluripotent cell lines & ¢ Pluripotent cells have the capacity to become any human cell type ~~ Liver Cells - ¥ Vascular / Smooth Muscle Cells ¢ Ahighly controlled process generates only the desired cell A * No genomic manipu
Slide 8: Technology Partners & Collaborators
Technology Partners & Collaborators ThermoFisher AJINOMOTO EN STEMCELL™ GE Healthcare Mi AGEX Advanced » Hadasit g IMMUNOMIC THERAPEUTICS 2 BioMatrix Bio-Holdings Lie GCiver GYROSCOPE Chg Heaithcare BicGa’ ‘Therapeutics ae my . = MICHIGAN STATE OD iistyessey WISCONSIN UCTrvINE fm SCHISANSTATE waniin
Slide 9: In-House cGMP Production Capabilities
In-House cGMP Production Capabilities Extensive experience directing the lineage of pluripotent cells into terminally differentiated, specialized cell types such as retinal cells, glial cells, etc. * Cell banking and handling * Process development * Manufacture of clinical material * Scale-up in mul
Slide 10: In-House cGMP Manufacturing
In-House cGMP Manufacturing The lineage of an established line of pluripotent cells can be controlled to create a population of substantially pure and differentiated cells (e.g. RPE cells) Identity Assay (purity) Functional Assay (phagocytosis) LINEAGE CELL THERAPEUTICS Fluorescence 7 0.06% wy 99.5%
Slide 11: OpRegen: A Cell Therapy Product Candidate for Dry AMD
OpRegen”: A Cell Therapy Product Candidate for Dry AMD www.linéagecell.com ee NYSE American: LCTX ail
Slide 12: Dry Age-Related Macular Degeneration (AMD)
Dry Age-Related Macular Degeneration (AMD) * Dry AMD involves the loss of specialized retina cells (RPE), causing impaired vision and blindness * OpRegen is formulated as a ready-to-inject suspension of RPE cells delivered to the sub-retinal space Photoreceptor iyhen oa —— recovery — legeneration me
Slide 13: OpRegen - Generating Evidence of a Treatment Effect
OpRegen - Generating Evidence of a Treatment Effect Stepwise progress has been made to support the expectation of a clinically-meaningful treatment effect from the transplant of RPE cells ina comparative clinical trial. Any Evidence? Durable Engraftment Yes Human Structural Improvement Yes Human Dru
Slide 14: Engraftment and Survival of RPE Cells in vivo
Engraftment and Survival of RPE Cells in vivo * OpRegen cells counter-stained with DAPI (red line) * OpRegen cells form a stable monolayer in multiple species RCS Rat NOD-SCID Mouse post-transplantation 2 mo ost-transplantation 2 months po Se ee ee anti-GFP anti-human Nuclei Human specific marker Tr
Slide 15: Improved Visual Function in RCS Rat Model
Improved Visual Function in RCS Rat Model Dose-dependent rescue of vision can be observed via optokinetic nystagmus 0.6 Control ° @ 25K Cells 0.4 ie m 100K Cells 0.3 = 200K Cells 0.2 O41 Spatial Frequency (cycles per degree) ° 60 100 150 200 Age (Days) onl _ o a McGill TJ, et al. Transl Vis Sci Tech
Slide 16: Ongoing Phase I/IIa OpRegen Clinical Trial
Ongoing Phase I/Ila OpRegen Clinical Trial Purpose: To evaluate the safety and efficacy of subretinally transplanted RPE cells in patients with advanced dry AMD with geographic atrophy (GA) Design: Open label, single-arm, and multi-center (5 sites) Dose and Administration: One 50-100 ul dose of cell
Slide 17: OpRegen Phase I/IIa Clinical Trial
OpRegen Phase I/Ila Clinical Trial Patient Characteristics Currently Enrolling Target Patient Population Part 1 - Cohorts 1-3 Part 2 - Cohort 4 Parameter legally blind) (le N ed dise n=12 n=4 Best Corrected Visual Acuity (BCVA) WEEERGOn GURL [23 letters is =20/400] [55 letters is =20/80] Mean GA Are
Slide 18: Patient Data: Cell Engraftment
OpRegen Phase I/Ila Patient Data: Cell Engraftment Bleb border (boundary of transplanted OpRegen cells) Punctate shaded areas are indicative of stable engraftment of pigmented cells for more than 24 months through Color Fundus Photography 1
Slide 19: Patient Data: Drusen Reduction
OpRegen Phase I/Ila Patient Data: Drusen Reduction * Drusen are deposits of waste material associated with higher risk of dry AMD ¢ Drusen accumulation is observed at pre-treatment (wrinkled white line) ¢ Areduction or change to drusen is observed through 12 months in some patients Untreated Treated
Slide 20: Patient Data: Reduced Growth of GA
OpRegen Phase I/Ila Patient Data: Reduced Growth of GA Before and after tracing of the area of GA shows asymmetric growth Gray = pre-treatment Purple = 12 months Green line marks the area of P as transplanted OpRegen cells Treated Area (slower growth) Untreated Area (normal growth) CELL THERAPEUTICS
Slide 21: Patient Data: 12-Month Change in BCVA Cohort 4
OpRegen Phase I/Ila Patient Data: 12-Month Change in BCVA Cohort 4 (n=5) =—= Treated Eye Fellow Eye 30 a3 «6 Ph 5 5 ois F F 10 oo be 2 © 5 0 5 0 --f----------------- 3 3 - * - ° 2 10 2 10 a5 -as o 123 4 6 9 2 0123 4 6 9 2 Time Post-Implantation (months) Time Post-Implantation (months) ae 30 30 / \ y
Slide 22: Patient Data: Mean Change in BCVA
OpRegen Phase I/Ila Patient Data: Mean Change in BCVA (Treated and Fellow Eye) Change in BCVA - Treated and Fellow Eye 35 30 25 Change in number of ETDRS letters from baseline ) 1 2 3 4 6 9 12 Time Post-Implantation (months) Treated Eye ===Fellow Eye
Slide 23: Clinical Considerations: Cohort Patients at Months
Clinical Considerations: Cohort 4 Patients at 12 Months Phase I/Ila Study: Absolute Changes in Best Corrected Visual Acuity at 12-Month Timepoint* bject # Treatment Route 16 + 25 letters Month 6 Orbit SDS 17 +2 letters Month 1 Orbit SDS * Patients are assessed at 15 months and at years 2-5, but 6- a
Slide 24: Real-World Relevance of Letter Improvement
Real-World Relevance of “Letter Improvement” ETDRS Visual Acuity Chart Initial Visual Acuity Score (20/160) 25 Letter Improvement (20/50)
Slide 25: Patient Data: Correlating BCVA and GA
OpRegen Phase I/Ila Patient Data: Correlating BCVA and GA (Subject #13) Best Corrected Visual Acuity Change in Area of (BCVA) Geographic Atrophy (GA) # of ETRDS letters mm 3 6 9 2 15 3 6 9 2 Months Post-Implementation Months Post-Implementation mums Treated Eye —— Fellow Eye (Control) Long-term foll
Slide 26: Subretinal Delivery Solution
Subretinal Delivery Solution * Standard subretinal injection technique requires vitrectomy and retinotomy — Known complications include retinal detachment and other adverse events ¢ Lineage has begun using a vitrectomy-free subretinal injection device: — “For subretinal delivery of RPE cells for the
Slide 27: Orbit SDS (Suprachoroidal Approach)
Orbit SDS (Suprachoroidal Approach) Orbit Subretinal Injection Device Flexible Needle Advancement ~_ ae — ~ » Saline Injection —_ e — Slide Lock Infusate Injection—_ Valve Lever Cannula ~~ Microneedle — Flexible cannula (no puncture made to retina) Chor Microneedle Image used with permission from Gy
Slide 28: OpRegen Clinical Study: Orbit SDS Subjects
Phase I/Ila OpRegen Clinical Study: Orbit SDS Subjects (n=2) * First subretinal injection of OpRegen with Orbit performed July 2019 — No operational complications — No unexpected post-op complications — Subject doing well, no unexpected AEs as of 6 months post-op — Demonstrated signs of improved vis
Slide 29: OpRegen Clinical Trial Highlights
Phase I/Ila OpRegen Clinical Trial Highligh Treatment with OpRegen: Summary Findings Structural Improvement Some patients show signs of structural improvement in the retina and decreases in drusen density * Photoreceptor layer and ellipsoid zone assumed a more regular structural appearance in areas
Slide 30: Significant Unmet Medical Need
Significant Unmet Medical Need ¢ AMD afflicts ~11 million people in the United States ~ ~$6B in sales of approved wet AMD therapies: Lucentis’ and Eylea’ ~ But 90% of AMD patients have the dry form ~ Currently, there are no approved therapies for dry AMD aside from nutritional supplements! Sources:
Slide 31: Dry AMD Approaches: In The Clinic
Dry AMD Approaches: In The Clinic Cell Therapy Has Potential for Disease Reversal and One-Time Treatment Cell Therapy Lineage Cell Therapeutics (P1/2) Astellas (P1/2) Regenerative Patch Technologies (P1/2) Cell therapy has the potential for both disease REVERSAL and one-time treatment Oxidative Stre
Slide 32: OpRegen Well Positioned in Dry AMD
OpRegen Well Positioned in Dry AMD OpRegen’s advantages compared to other cell therapies in development (manufacturing, route of administration) Company Stage Types of Route of Patients Astellas Phase 1 (n=9) 20/200+ Trans-vitreal injection Phase 1 complete (new cell line) Phase 2 (n=150) Phase 2 on
Slide 33: 2020 — OpRegen Upcoming News and Events
2020 — OpRegen Upcoming News and Events * DSMB meeting to review the Phase I/Ila study protocol — Seeking approval to perform concurrent patient enrollment * Opening additional clinical sites in Phase I/Ila study — Cincinnati Eye Institute — Wills Eye Hospital * Complete patient enrollment in Orbit
Slide 34: OPC1: A Cell Therapy Product Candidate for Spinal Cord Injury
OPC1: A Cell Therapy Product Candidate for Spinal Cord Injury www.linéagecell.com — NYSE American: LCTX ail
Slide 35: Lucas' Story
Lucas’ Story
Slide 36: Spinal Cord Injury (SCI) Unmet Need
Spinal Cord Injury (SCI) Unmet Need Spinal cord segments * SCI creates a significant burden for patients and caregivers* Cervical nerves — 67% of patients are unemployed 10 years post-injury — Lifetime healthcare costs can reach $5 million for one patient Thoracic nerves * Motor level improvements t
Slide 37: OPC1 Overview
OPC1 Overview OPC1 is a population of “off the shelf” oligodendrocyte progenitor cells (OPCs) OPCs are precursors to the cells which provide electrical insulation for nerve axons in the form of a myelin sheath OPC1 has RMAT and Orphan Drug Designations from the FDA Program has received >$14M from cI
Slide 38: OPC1 Potential Mechanisms of Action
OPC1 Potential Mechanisms of Action shiverer mouse shi mouse + OPC1 Control Media
Slide 39: Completed Studies in Spinal Cord Injury
Completed Studies in Spinal Cord Inju Pr inical Clinical (n=30) 28 Animal Studies Phase 1 Thoracic Study Cells survive in the spinal cord Improves locomotor activity Reduces parenchymal cavitation Migrates up to 5cm in spinal cord No distribution outside of CNS Does not increase mortality Does not i
Slide 40: SCiStar Study Enrollment & Cohort Progression
SCiStar Study Enrollment & Cohort Progression * Open Label (n=25) * Primary Assessment: Safety * Traumatic cervical SCI (C4-C7 * Secondary Assessment: Neurological Function * Dosed 21-42 days post injury (ISNCSCI exams) ° Ages 18-69 * Exploratory Assessments: SCIM, GRASSP. ° AlSAor AISB I Cohort 1 }
Slide 41: Safety and Efficacy from OPC1 Phase 1/2a Study
Safety and Efficacy from OPC1 Phase 1/2a Study ee Cell Engraftment Motor Function Gain (cohorts 2-5 at 12 months, n=22) (cohorts 2-5 at 12 months, n=22) 32% Two or More Levels To date, there have been no serious adverse events related to the OPC1 cells
Slide 42: Real-World Impact from +2 Motor Level Gain
Real-World Impact from +2 Motor Level Gain Activities of Daily Living (ADLs) +2 Motor Level Improvement Function Capability C1-C3 C4 cs (a C7-C8 Bowel Bladder Bed Mobility Transfers Pressure Relief Eating Dressing Grooming Bathing Wheelchair Car transport Daily Home Care 24 hr attendant Total Assist
Slide 43: SCiStar Study Year Results
SCiStar Study — 2 Year Results (Nov 2019 Update) * Overall safety profile continues to be excellent (21 subjects) - MRI scans show no evidence of adverse changes - No unexpected serious adverse events related to the OPC1 cells - No study subjects had worsening of neurological function ¢ Motor Level
Slide 44: SCiStar Study - Overall Summary
SCiStar Study - Overall Summary ¢ Excellent overall safety profile * 96% durable engraftment through 1 year post-injection ¢ MRI scans available through 24 months show no evidence of adverse changes (21 subjects) * No subjects had a decline in motor function from Year 1 to Year 2 * 95% of patients e
Slide 45: 2020 — OPC1 Upcoming News and Events
2020 — OPC1 Upcoming News and Events ¢ Enhance the OPC1 program commercial readiness — Introduce enhancements to the manufacturing process (robust and commercially viable improvements to scale, purity, and reproducibility) — Develop a Thaw-and-Inject formulation (eliminates need for dose preparation
Slide 46: VAC2: A Cell Therapy Product Candidate for Cancer
-z= LINEAGE @m CELL THERAPEUTICS VAC2: A Cell Therapy Product Candidate for Cancer (Immuno-Oncology) www.linéagecell.com ~~ NYSE American: LCTX al
Slide 47: VAC Immuno-Oncology (I-O) Program
VAC Immuno-Oncology (I-O) Program * The VAC platform uses mature dendritic cells (DC) to increase a patient’s tumor response * VAC is an allogeneic (“off the shelf”) vaccine; cells are manufactured from a pluripotent cell line and not derived from the patient (time and cost advantages) * Mature dend
Slide 48: VAC2 Clinical Program
VAC2 Clinical Program * Based on encouraging survival data generated in an antecedent autologous VAC1 program in Acute Myeloid Leukemia* * VAC2 is partnered with Cancer Research UK, which is responsible for the costs and conduct of manufacturing and the clinical trial * Primary endpoint: safety and
Slide 49: Potential Advantages of the VAC2 Approach
Potential Advantages of the VAC2 Approach Attribute Single master cell bank for scalability and consistency v Available ‘off-the-shelf’, on demand No known significant off-target effects Lower anticipated COGS than CAR-T v v Low AE-related cost of treatment v v v Use in combination with chemotherapy
Slide 50: Renevia
Renevia® CE Mark Granted September 2019 www.linéagecell.com ~ / NYSE American: LCTX a
Slide 51: Renevia - Medical Aesthetics Program
Renevia® - Medical Aesthetics Program * A3-D scaffold designed to support adipose (fat) tissue transplant and retention ¢ 50-patient, HIV-Associated Lipoatrophy Pivotal Study: — Renevia in combination with fat-derived SVF cells for facial volume augmentation — Increase in hemifacial volume measured
Slide 52: Financial Overview
Financial Overview * Cash and cash equivalents and marketable securities — $35.7 million (as of 9/30/2019, last reported quarter) — Sold $5.0 million of OncoCyte (OCX) holdings on 1/2/2020 * Provides funding into 2021 assuming on-time payment of Juvenescence note * Value of Remaining Equity Holdings
Slide 53: Lineage 2019 Key Accomplishments
Lineage 2019 Key Accomplishments A Year of Transformation OBJECTIVES SET Expand/diversify our cell therapy pipeline Divest/cease non-core activities Enhance competitive advantage of OpRegen program Re-brand the company, invigorate with new executive team Enhance IP portfolio Intelligent cost-cutting
Slide 54: Lineage 2020 Key Goals
Lineage 2020 Key Goals A Year of Major Inflection Points GOALS Advance OpRegen program Advance OPC1 Program VAC2 Program Efficient use of resources Expand BD activities EUTIC = LINEAGE CELL THERAP 3 TIMING Qi 2020 Qi 2020 Q2 2020 2H 2020 Throughout 2020 Qi 2020 2020 Throughout 2020 Throughout 2020 S
Slide 55: Why Invest in Lineage?
Why Invest in Lineage? Lineage is well-positioned for near-term growth and long-term value © © 3 clinical-stage World class One of the Funded well Leader in the programs with in-house GMP largest patent into 2021* with emerging field billion dollar manufacturing portfolios in cell cost-efficient of
Slide 56: The future of cell therapy.
-z= LINEAGE @m CELL THERAPEUTICS The future of cell therapy. ————— www.linéagecell.com y, NYSE American: LCTX ail
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