NYSSA Conference Presentation: B. Caldwell
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This document summarizes the work of Advanced Cell Technology, a company focused on translating stem cell research into commercial products. It discusses ACT's scientific leadership in regenerative medicine as the first to prove an alternative method for deriving human embryonic stem cells. It outlines ACT's product development pathways for retinal pigment epithelium cells, hemangioblasts, and dermal products. The document highlights ACT's facilities and strategies for good manufacturing practice production, preclinical testing, and plans to file an Investigational New Drug Application for its retinal pigment epithelium cells by the end of 2007. It provides an overview of ACT's corporate milestones achieved in 2006 and goals for 2007.
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NYSSA Conference Presentation: B. Caldwell
- 1. Translating Stem Cell Research into Commercial Products Translating Stem Cell Research into Commercial Products December 2006December 2006
- 2. 2 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 Statements Cautionary Statement Concerning Forward-Looking Statements
- 3. Advanced Cell TechnologyAdvanced Cell Technology
- 4. 4 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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- 7. The Vision of Advanced Cell Technology The Vision of Advanced Cell Technology
- 8. 8 Driving Human ES Cell Therapies to the ClinicDriving Human ES Cell Therapies to the Clinic
- 9. 9 Potential of ACT Embryonic Stem CellsPotential of ACT Embryonic Stem Cells Unlimited Expansion of Stem Cell Source Master Cell BankMaster Cell Bank Working CellWorking 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
- 10. 10 Commercialization of ACT TherapiesCommercialization of ACT Therapies DermalDermal RetinalRetinal PigmentedPigmented EpitheliumEpithelium HemangioblastHemangioblast Cryopreserved Cell BankCryopreserved Cell Bank Working Cell BankWorking Cell BankCryopreserved Cell BankCryopreserved Cell Bank Cryopreserved Cell BankCryopreserved Cell Bank
- 11. 11 Our Product Development PathwayOur Product Development Pathway Cell Discovery and Validation Finding Cell Types with Therapeutic Potential Select Target Indications “GMP” Cell Production Derive Human ES Cells under GMP Differentiate Target Cell Type under GMP Preclinical and Clinical Testing Functional and Safety Preclinical Testing Develop Plan with FDA for Human Clinical Trials target cell typestarget cell types target cell linestarget cell lines 20062006 20072007 20042004 20052005
- 12. 12 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
- 13. 13 Regenerative Medicine Curing Devastating Disease Regenerative Medicine Curing 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
- 14. Driving Human ES Cell Therapies to the Clinic Driving Human ES Cell Therapies to the Clinic
- 15. 15 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
- 16. 16 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
- 17. 17 Our Worcester GMP FacilityOur Worcester GMP Facility
- 18. 18 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
- 19. 19 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
- 20. 20 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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- 22. 22 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
- 23. 23 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 Hemangioblasts under GMP in 2007 and commence additional preclinical studies for target indications 40X
- 24. 24 Our Hemangioblast ProgramOur Hemangioblast Program Hemangioblasts Generated from Single Blastomere Derived MA01s Human ES Cells 10X
- 25. 25 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
- 27. 27 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
- 28. 28 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
- 29. 29 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