Lineage Cell Therapeutics, Inc. · AMEX:LCTX
Lineage Cell Therapeutics — Biotime — Rodman & Renshaw 16th Annual Global Investment Conference (September 2014)
37 slides from Lineage Cell Therapeutics's conference presentation, Sep 10, 2014
Slide 1: Rodman & Renshaw 16th Annual Global Investment Conference
NYSE MKT: BTX Rodman & Renshaw16th Annual GlobalInvestment ConferenceSeptember 10, 2014
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: The Technology Leader in Regenerative Medicine: Investment Highlights
* 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/
Slide 4: Advent of Regenerative Medicine
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 trial
Slide 5: Value of the Pluripotent Platform
Value of the Pluripotent Platform * Cell therapy: In growth phase – potentially long lifespan due to lack of regulatory pathway for generics or biosimilars
Slide 6: Targeting High Medical Need
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
Slide 7: Recent Milestones
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 fo
Slide 8: Rapid Cadence of Milestones
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 compl
Slide 9: Major Therapeutics
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
Slide 10: OPC1 & VAC2
OPC1 & VAC2 * Developing Breakthrough Technologies for Regenerative MedicinePedro Lichtinger, CEOFormer CEO Optimer Pharmaceuticals, and President of Pfizer’s Global Primary Care Unit *
Slide 11: OPC1 – Unmet Medical Needs
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 myelina
Slide 12: OPC1 Phase 1/2a Trial Design
* 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,
Slide 13: OPC1 Opportunity: SC Injury
* OPC1 Opportunity: SC Injury
Slide 14: 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 * Phase 1: prostate cancerDukeJ. Immunol 2005, 174:3798 Phase 2: acute myelogenous leukemia Multi-centerK
Slide 15: VAC2 – Unmet Medical Needs
VAC2 – Unmet Medical Needs * Advantages of VAC2 Platform
Slide 16: VAC2 Opportunity: NSC lung cancer
* 1 Source: Cancer Research Technologies2 Source: National Institutes of Health3 Source: Decision Resources VAC2 Opportunity: NSC lung cancer
Slide 17: OpRegen
OpRegen * Advancing Medicine Through Stem Cell TherapiesCharles Irving, Ph.D., CEO *
Slide 18: OpRegen – Unmet Medical Needs
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 (A
Slide 19: OpRegen Phase 1/2a Trial Design
* 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 subretinal
Slide 20: OpRegen Opportunity: Dry AMD
OpRegen Opportunity: Dry AMD * 1 Source: http://www.amd-therapy.de/en/investment/market-and-competition
Slide 21: Device & Diagnostic Pipeline
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 medica
Slide 22: HyStem - Unmet Medical Needs
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
Slide 23: Renevia - Medical Need
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 autolog
Slide 24: Renevia – Pivotal Clinical Trial
* 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 adipocyte
Slide 25: 2014 Milestones – HyStem
2014 Milestones – HyStem * Initiation of Renevia™ pivotal clinical trial510(k) clearance for Premvia™2014-15 file 510(k) application for ReGlyde™
Slide 26: Renevia Opportunity: Lipoatrophy
* 1 Source: AIDS Care Vol. 21, No. 5, May 2009, 664-671 Renevia Opportunity: Lipoatrophy
Slide 27: PanC-Dx
PanC-Dx * Advanced Noninvasive Cancer DiagnosticsJoseph Wagner, Ph.D., CEO *
Slide 28: PanC-Dx – Cancer Diagnostics
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
Slide 29: PanC-Dx – Breast Cancer Study
* 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 •
Slide 30: PanC-Dx – Bladder 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
Slide 31: PanC-Dx – Lung 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 dat
Slide 32: PanC-DX - Opportunity #1: Breast CA
PanC-DX™ - Opportunity #1: Breast CA * Blood-based screening diagnostic measures sera protein biomarkers using ELISA. Clinical study underway & in discussions with additional sites
Slide 33: PanC-DX - Opportunity #2: Bladder CA
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
Slide 34: PanC-DX - Opportunity #3: Lung CA
PanC-DX™ - Opportunity #3: Lung CA * Blood-based lung cancer screen for high-risk populationMulti-center study underway with high interest from KOLs
Slide 35: BioTime (NYSE Market: BTX) Key Statistics
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 ma
Slide 36: Long-Term Value Creation Strategy
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
Slide 37: BioTime – Investment Opportunity
BioTime – Investment Opportunity * Positioned as the technology leader in the coming regenerative medicine revolution
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