Lineage Cell Therapeutics, Inc. · AMEX:LCTX
Lineage Cell Therapeutics — Investor Presentation (November 2020)
42 slides from Lineage Cell Therapeutics's investor presentation, Nov 10, 2020
Slide 1: Corporate Overview
The future of cell therapy. Corporate Overview November 10, 2020
Slide 2: Forward-Looking Statements
Forward-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,
Slide 3: Management Biographies
Management Biographies Mr. Culley joined Lineage as CEO in September 2018. Prior to joining Lineage, Mr. Culley served from August 2017 to September 17, 2018 as Interim Chief Executive Officer at Artemis Therapeutics, Inc., a, where he was responsible for the management of the company. Mr. Culley pr
Slide 4: Lineage Cell Therapeutics – Investment Overview
Lineage Cell Therapeutics – Investment Overview Innovative Transplanting differentiated cells to treat a wide range of serious medical Approach conditions World-class expertise and IP; ability to manufacture an unlimited supply Unique Advantage of pluripotent stem cells into specialized cell types T
Slide 5: Technology Overview
“We aim to pioneer a new branch of medicine, based on transplanting specific cell types into the body” Technology Overview
Slide 6: Lineage Technology Platform
Lineage Technology Platform “Off-the-shelf” cell therapies to treat serious diseases and conditions • The Lineage Platform starts Undifferentiated with a frozen vial of stem cells self-renewing stem cells LINEAGE • These unique cells can become TECHNOLOGY any cell type in the body • Lineage’s propri
Slide 7: In-House Manufacturing
In-House Manufacturing Lineage’s competitive advantage is the differentiation of an unlimited supply of pluripotent stem cells into specialized cell types Capabilities Facilities • Cell banking and handling • Process development • Manufacture of clinical trial material • Scale-up in multi-liter bior
Slide 8: Pipeline and Validating Partnerships
Pipeline and Validating Partnerships Financial Clinical Programs Support Phase 1 Phase 2a Next Steps Received OpRegen® (RPE Cells) Complete Dry AMD with enrollment by year Geographic Atrophy (GA) $16M end Trial complete, OPC1 (Oligodendrocytes) planning phase Spinal Cord Injury (SCI) 2b/3 $14M VAC2
Slide 9: OpRegen: Cell Therapy for Dry AMD
AMD is the leading cause of irreversible vision loss in the US Source: aao.org ® OpRegen : Cell Therapy for Dry AMD
Slide 10: Dry AMD Can Lead Rapidly to Blindness
Dry AMD Can Lead Rapidly to Blindness Visual acuity over time… The area of geographic 20/20 atrophy or “GA” grows (normal) larger as retinal cells die 2012 2013 2014 2015 2017 2019 20/200 (legally blind in 3 years) Dry AMD involves the progressive loss of retina cells, which can lead rapidly to blin
Slide 11: Multi-Billion Dollar Market Opportunity in the U.S.
Multi-Billion Dollar Market Opportunity in the U.S. Age-related Macular Degeneration (AMD) (all forms) afflicts ~11 million people in the United States Type of AMD % of AMD Cases FDA Approved Therapies Wet AMD 10 – 15% Lucentis & Eylea ($10 Billion in annual sales) Dry AMD 85 – 90% None 85 - 90% of
Slide 12: Lineage Approach – OpRegen, an RPE Cell Transplant
Lineage Approach – OpRegen, an RPE Cell Transplant • Dry AMD involves the loss of retina cells, • OpRegen is a injection of RPE cells creating an area of geographic atrophy (GA), delivered to the retina, to replace lost which causes impaired vision and blindness retinal cells and preserve or improve
Slide 13: Promising Results
Promising Results (As of Sep. 14, 2020 Update) • The transplanted OpRegen cells have been well tolerated • No reports of rejection of the transplanted cells or acute or delayed inflammation • 4 out of 5 “Cohort 4” patients (the intended commercial population) have better vision at their 1-year time
Slide 14: Cohort 4 Patients – Improved Visual Acuity
Cohort 4 (Better Vision) Patients – Improved Visual Acuity (As of ARVO May 2020 Update) Sustained Increase in Vision in Treated Eye Versus Untreated Eye 30 Change in number of ETDRS letters P=0.0283 P=0.0200 15 from baseline +12.3 letters 0 n=5 n=4 n=3 -15 0 1 2 3 4 6 9 12 15 Treated Eye Untreated E
Slide 15: Real-World “Letters of Improvement”
Real-World “Letters of Improvement” Legally Blind (20/200) Initial Visual Acuity Example (20/125) +10 letters (20/80) +15 letters (20/63) ETDRS Visual Acuity Chart #3 15
Slide 16: First Known Report of Retinal Tissue Regeneration in Dry AMD
First Known Report of Retinal Tissue Regeneration in Dry AMD Shown below are tracings of the GA boundary Imaging shows a clear reduction in size of GA after treatment Red = Perimeter before treatment Blue = Perimeter at 9 months • Area of atrophy regressed 27% from baseline. • Restoration cannot occ
Slide 17: Cohort 4 Patients – Slower GA Growth Observed
Cohort 4 (Better Vision) Patients – Slower GA Growth Observed (As of ARVO May 2020 Update) Slower Growth of Area of GA for Treated Versus Untreated Eye 4 3 Change in size of GA (mm2) 2 -34% 1 0 n=5 n=4 n=3 -1 0 1 2 3 4 6 9 12 15 Treated Eye Untreated Eye Time Post-Implantation (months) Data presente
Slide 18: Proprietary Delivery System – The Orbit SDS
Proprietary Delivery System – The Orbit SDS Lineage has an exclusive option to a delivery device which overcomes issues with the traditional method of delivering cells to the retina Traditional Method (safety issues) Orbit SDS (Lineage method) • The traditional method punctures the retina; • With th
Slide 19: Dry AMD Competitive Landscape
Dry AMD Competitive Landscape Only cell therapy has potential to restore tissue with infrequent dosing Only Lineage has shown evidence of retinal restoration Only Lineage has access to the Orbit delivery system to deliver cells Cell Therapy Complement Inhibitors • Lineage Cell (Ph1/2) • Apellis (Ph3
Slide 20: Commercial-Scale Manufacturing Capabilities
Commercial-Scale Manufacturing Capabilities • Current capabilities enable commercial-scale cGMP Production • OpRegen cells are >99% pure RPE cells - NIH approved line was established >20 years ago - Extensive characterization and karyotyping performed on each batch - No genetic modifications are mad
Slide 21: Ongoing Phase 1/2a Clinical Trial of OpRegen for Dry AMD
Ongoing Phase 1/2a Clinical Trial of OpRegen for Dry AMD Cohorts 1-3 Cohort 4 Safety Portion / Legally Blind Efficacy Portion / Impaired Vision 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 21 22 23 24 COMPLETED ENROLLMENT ONGOING* * Patient enrollment expected to complete by YE 2020 21
Slide 22: OpRegen – Positioned for Commercial Success
OpRegen – Positioned for Commercial Success OpRegen has been designed to capture a multi-billion dollar opportunity: ➢ Transplanting RPE cells may provide benefits other approaches cannot provide ➢ Market opportunity is not limited to monogenic deficiencies (for gene therapy) ➢ Treatment to date has
Slide 23: OPC1: Cell Therapy for Spinal Cord Injury
Lifetime care for an SCI patient can cost nearly $5 million Source: christopherreeve.org OPC1: Cell Therapy for Spinal Cord Injury
Slide 24: Lucas’ Story
Lucas’ Story Photo: Fox6News, Milwaukee Lucas Linder, an OPC1 clinical trial participant, was paralyzed from the neck down. The next year, he threw out the first pitch at a Major League Baseball game. 24
Slide 25: Using Lineage’s OPC1 Cells for Spinal Cord Injury
Using Lineage’s OPC1 cells for Spinal Cord Injury Loss of movement is the primary feature of a spinal cord injury. The goal of treatment with OPC1 is to provide additional upper limb & finger function and improve the quality of life for patients • OPC1 is comprised of oligodendrocyte progenitor cell
Slide 26: Cervical Spinal Cord Injury Level and Motor Function
Cervical Spinal Cord Injury Level and Motor Function brain cervical spinal cord spinal cervical nerves vertebrae Breathing and neck movement Shoulder and elbow movement C5 – elbow flexors Elbow and Wrist movement C6 – wrist extensors Hand finger C7 – elbow extensors Movement C8 – hand finger flexors
Slide 27: OPC1 Addresses the Complex Pathology of SCI
OPC1 Addresses the Complex Pathology of SCI OPC1 is a cellular therapy involving the transplant of oligodendrocyte progenitor cells (OPCs) derived from a pluripotent stem cell line • OPCs, which function to support and myelinate neurons, can be damaged and lost due to inflammatory response post inju
Slide 28: OPC1 Mechanisms of Action
OPC1 Mechanisms of Action 1. Prevention of Cavitation Control OPC1 2. Myelination of axons 3. Secretion of neurotrophic factors shiverer mouse shi mouse + OPC1 Control Media OPC1 TubIIIneurite outgrowth Promote increased 28
Slide 29: Clinical Trial Design
Clinical Trial Design (enrollment complete) Safety and dose escalation in two grades of severity • 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 F
Slide 30: Clinical Trial Results - Safety and Efficacy in Patients
Clinical Trial Results - Safety and Efficacy in 22 Patients Cell Engraftment Motor Function Gain (at 12 months) (at 12 months) 32% Two or More Levels 96% 96% Successful of Subjects Engraftment Reported 96% Improved Motor 64% Function One Level No Improvement (6%) No Improvement (4%) No Improvement (
Slide 31: Real-World Benefit from a Gain of 2 Motor Levels
Real-World Benefit from a Gain of 2 Motor Levels Motor level gains translate into clinically meaningful improvements in self-care and reductions in cost of care +2 Motor Level Improvement Total Assist Partial Assist Independent Activities of Daily Living across different levels of motor function aft
Slide 32: Clinical Trial – 2 Year Results
Clinical Trial – 2 Year Results (per Nov 2019 Update) • Overall safety profile of OPC1 continues to be excellent (21 subjects evaluated) - 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
Slide 33: Clinical Trial - Overall Summary
Clinical Trial - Overall Summary • Excellent overall safety profile • 96% experienced durable engraftment through 1 year post-injection • MRI scans available through 24 months show no evidence of adverse changes • No subjects had a decline in motor function from Year 1 to Year 2 • 95% of patients ex
Slide 34: OPC1 Program: Next Steps
OPC1 Program: Next Steps Key Considerations: • Compelling clinical data supports moving to later-stage clinical development • Early-stage manufacturing issues (of prior owner) needed to be addressed - Lineage advantage! • Leveraging Lineage’s manufacturing capabilities to improve purity, scale, usab
Slide 35: VAC: Cell Therapy for Immuno-Oncology and Infectious Disease
Immunotherapy is "poised to revolutionize treatment for all types of cancer" Source: cancerresearch.org VAC: Cell Therapy for Immuno-Oncology and Infectious Disease
Slide 36: VAC Platform
VAC Platform • The VAC platform consists of large-scale production of mature immune cells called dendritic cells (DCs) • DCs are manufactured and loaded with either a tumor antigen (to treat cancer) or a viral antigen (as a vaccine for infectious diseases) • Antigen presentation to the patient’s T c
Slide 37: VAC Development – Multiple Programs from a Single Platform
VAC Development – Multiple Programs from a Single Platform VAC1 and VAC2 Highlights • Positive phase 1 data in AML • High levels of immunogenicity seen • Cancer Research UK alliance Lineage • Positive phase 1 in NSCLC trial run Manufacturing Dendritic by CRUK Platform Cell VAC3, VAC4, VAC5…Opportuni
Slide 38: VAC Platform Next Steps
VAC Platform Next Steps Upcoming Events and Key Considerations: ➢ Complete dosing in ongoing clinical trial in NSCLC (n=8; 2 remain) ➢ Design and conduct VAC2 + checkpoint inhibitor clinical trial ➢ Design new products (i.e. VAC3, 4, 5, 6…) with newly discovered antigens ➢ Continue to identify poten
Slide 39: Financial Overview
Financial Overview Cash, Cash Equivalents and Marketable Securities $38 mm (As of last reported quarter 9/30/2020) Debt $0.5M PPP loan Market Capitalization ~$193 mm (As of 11/6/2020) Employees 53 (As of 11/6/2020) A new management team and prioritization of cell therapy programs has resulted in a m
Slide 40: Upcoming Milestones
Upcoming Milestones PROGRAM TIMING INITIATIVES OpRegen Q4 2020 Present updated OpRegen data at AAO (Nov. 13 – 15, 2020) Q4 2020 Complete enrollment of phase 1/2a study Q4 2020 Planning discussions with the FDA on device and manufacturing Ongoing Evaluate OpRegen partnership opportunities Ongoing Con
Slide 41: Our Goal is to Provide Life-Changing Cell Therapies to Patients
Our Goal is to Provide Life-Changing Cell Therapies to Patients Lineage Cell Therapeutics: Bringing the Promises of Cell Therapy into Clinical Reality 3 clinical-stage World class One of the Funded well Leader in the programs with in-house GMP largest patent into 2022 with emerging field billion-dol
Slide 42: The Patients Are Our Inspiration
The Patients Are Our Inspiration. View their stories at lineagecell.com/media/#patients OPC1 SCiStar Study Participants https://blog.cirm.ca.gov/2018/01/24/how-a-stem-cell-transplant-may-help-transform-lucas-lindners-life/ https://blog.cirm.ca.gov/tag/kris-boesen/ https://blog.cirm.ca.gov/2020/06/29
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