NYSSA Conference Presentation: B. Caldwell
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Transcript of NYSSA Conference Presentation: B. Caldwell
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Translating Stem Cell Research into Commercial Products
Translating Stem Cell Research into Commercial Products
December 2006December 2006
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This presentation contains “forward-looking statements” as defined under the federal securities laws. Actual results could vary materially. Factors that could cause actual results to vary materially are described in our filings with the Securities and Exchange Commission.
You should pay particular attention to the “risk factors” contained in documents we file from time to time with the Securities and Exchange Commission. The risks identified therein, as well as others not identified by the Company, could cause the Company’s actual results to differ materially from those expressed in any forward-looking statements.
Cautionary Statement Concerning Forward-Looking StatementsCautionary Statement Concerning Forward-Looking Statements
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Advanced Cell TechnologyAdvanced Cell Technology
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BackgroundBackground
� Scientific Leader in Regenerative Medicine
� First Proven Alternative Method for Deriving Human Embyronic Stem Cells
� Driving Human ES Cell Therapies to the Clinic
� Focus on RPE, Hemangioblast and Dermal Product Programs
� Proprietary Human ES Cell Technology
� Over 300 Patents and Patent Applications
� Award Winning Team of Scientists and Management
� Dr. Mike West and Dr. Robert Lanza are Recognized Thought Leaders
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The Vision of Advanced Cell Technology
The Vision of Advanced Cell Technology
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Driving Human ES Cell Therapies to the ClinicDriving Human ES Cell Therapies to the Clinic
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Potential of ACT Embryonic Stem CellsPotential of ACT Embryonic Stem Cells
Unlimited Expansion of Stem Cell Source
Master Cell BankMaster Cell Bank
Working Cell Working Cell BankBank
Bank of Differentiated CellsBank of Differentiated Cells
Bank of Differentiated CellsBank of Differentiated Cells
Bank of Differentiated CellsBank of Differentiated Cells
Single BlastomereSingle Blastomere
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Commercialization of ACT TherapiesCommercialization of ACT Therapies
DermalDermalRetinal Retinal Pigmented Pigmented EpitheliumEpithelium
HemangioblastHemangioblast
Cryopreserved Cell BankCryopreserved Cell BankWorking Cell BankWorking Cell BankCryopreserved Cell BankCryopreserved Cell Bank
Cryopreserved Cell BankCryopreserved Cell Bank
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Our Product Development PathwayOur Product Development Pathway
Cell Discovery and ValidationFinding Cell Types with Therapeutic Potential
Select Target Indications
“GMP” Cell ProductionDerive Human ES Cells under GMP
Differentiate Target Cell Type under GMP
Preclinical and Clinical Testing Functional and Safety Preclinical TestingDevelop Plan with FDA for Human Clinical Trials
target cell typestarget cell types
target cell linestarget cell lines
20062006
20072007
2004 2004 20052005
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Stem Cell Therapy: The MarketplaceStem Cell Therapy: The Marketplace
� $30 billion estimated market for successful stem cell therapies
� Total costs of some major illnesses:� Heart Disease - $175 billion� Diabetes - $98 billion� Alzheimer’s - $87 billion� Vision Disorders - $68 billion� Stroke - $43 billion
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Regenerative MedicineCuring Devastating DiseaseRegenerative MedicineCuring Devastating Disease
800,000 Parkinson's patients 750,000
liver disease patients
20,000,000 heart disease patients
20,000,000 diabetes patients
5,000,000 epilepsy patients
4,000,000 Alzheimer's patients
2,000,000 stroke patients
300,000 kidney disease patients
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Driving Human ES Cell Therapies to the ClinicDriving Human ES Cell Therapies to the Clinic
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Our Clinical Testing PathwayOur Clinical Testing Pathway
Research
Development
Process Validation
Process Understanding
Pre-Clinical Testing
Clinical Testing- Phase 1
Clinical Testing- Phase 2
Clinical Testing- Phase 3
Commercialization (Phase 4) Marketing
BLA
IND
Amendments
Updates
Letters
Submissions
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Our GMP StrategyOur GMP Strategy
� Worcester GMP Lab – utilize for pilot mfg of differentiated cell types (RPE, Hemangioblasts, etc.) for pharm/tox studies and Phase I studies
� Alameda GMP Lab – utilize for larger scale mfg of differentiated cell types for all phases of preclinical and human clinical studies
� Nov 2005 – Commenced build-out of small GMP lab in Worcester
� Feb 2006 – Opened research facility in Alameda, CA – 10,000 sq ft of GMP capable lab
� April 2006 – Completed build-out of GMP lab in Worcester
� May 2006 – Commenced Scientific Work under GMP
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Our Worcester GMP FacilityOur Worcester GMP Facility
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� Documentation� Training� Quality� Production and Process Controls� Materials (components, containers, closures)� In-process and Final Product Testing� Shipping/Stability� Labeling/Packaging Control� Equipment/Metrology� Facility� Laboratory Control
Systems for Phase I Human Clinical TrialsSystems for Phase I Human Clinical Trials
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Blastomere Program – Taking Aim at the ClinicBlastomere Program – Taking Aim at the Clinic
� Transfer of 2 Blastomere Lines into GMP Manufacturing
� Creation of Master Cell Bank and Working Cell Banks
� Derivation of RPE Cells under GMP from hESC Blastomere Lines
� Creation of Cryopreserved Cell Product
� Scientific Work Underway to Produce Additional Blastomere Lines
� Seeking Approval for NIH Federal Funding for hESC Research Utilizing Blastomere Lines
� Announced Collaboration with Wi-Cell to Make Blastomere Lines Widely Available if Eligible for Federal Funding
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Our RPE Product Development ProgramOur RPE Product Development Program
� Commenced GMP RPE Cell Production
� Published Paper on Proof of Concept RPE Study in RCS Rat
� Hired Regulatory and RPE Clinical Advisors
� Commenced Further Pharmacology Studies
� Commencing Toxicology Studies in early 2007
� Target IND Filing by Year-end 2007
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RCS retina at P100 with RPE-9 injection:A: low power view of retina showing extensive
rescue;B: high power of outline b showing rescued
photoreceptors;C: high power of outline c showing non-rescue
area.
B
C
Photoreceptor Rescue in RCS Rat by transplanted H9- RPE cells
bb
cc
AA
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Our Hemangioblast ProgramOur Hemangioblast Program
� Significant Breakthrough in Scaled Manufacture of Hemangioblasts Cells
� Thoroughly characterized - make all cells of the blood and vascular tissues
� Significant Collaborations underway to generate proof of concept data in a wide range of indications / early data is extremely promising and expect to publish results in 2007
� Plan to generate Hemangioblastsunder GMP in 2007 and commence additional preclinical studies for target indications40X
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Our Hemangioblast ProgramOur Hemangioblast Program
Hemangioblasts Generated from Single Blastomere Derived MA01s Human ES Cells
10X
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Our Dermal ProgramOur Dermal Program
� Produced scalable hESC-derived dermal lines in Alameda facility
� Collaborations under-way for proof of concept studies
� Currently considering regulatory framework for dermal applications
� Large applications in burns, wound repair and surgery
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Corporate MilestonesCorporate Milestones
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Vital StatisticsVital Statistics
� AS OF NOVEMBER 27, 2006:
� Symbol: OTC BB: ACTC� Share price: $0.88� 52-Week High: $2.44� 52-Week Low: $0.26� Shares Outstanding: 27.7
million*� Float: 17.4 million*� Market Cap: $24.4 million*� Average Volume (90 days):
980,733 shares*� *approximate
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2006 Corporate Milestones2006 Corporate Milestones
Achieved
� Completed Build-out of GMP Facility
� Began “GMP” Cell Production
� Launched Preclinical and Clinical Testing Plans
� Expanded Technology Platform with Scientific Breakthroughs
� Raised Significant Additional Capital
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2007 Corporate Milestones2007 Corporate Milestones
Setting the Bar High
� Raise Additional Capital / Achieve Listing on National Exchange
� Announce Significant Corporate Partnership
� File IND for our RPE Program
� Complete Substantial Preclinical Work for our Hemangioblast and Dermal Programs
� Build-out our Development Organization
� Gain Approval from NIH for Federal Funding for hESC Research utilizing Blastomere Lines
� Announce Scientific Breakthrough on Solving Histocompatibility
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