nyssa conference presentation: b. caldwell

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Translating Stem Cell Research into Commercial Products Translating Stem Cell Research into Commercial Products December 2006 December 2006

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Page 1: NYSSA Conference Presentation: B. Caldwell

Translating Stem Cell Research into Commercial Products

Translating Stem Cell Research into Commercial Products

December 2006December 2006

Page 2: NYSSA Conference Presentation: B. Caldwell

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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

Page 3: NYSSA Conference Presentation: B. Caldwell

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

Page 5: NYSSA Conference Presentation: B. Caldwell
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Page 7: NYSSA Conference Presentation: B. Caldwell

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

Page 12: NYSSA Conference Presentation: B. Caldwell

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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

Page 13: NYSSA Conference Presentation: B. Caldwell

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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

Page 14: NYSSA Conference Presentation: B. Caldwell

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

Page 26: NYSSA Conference Presentation: B. Caldwell

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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