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Rodman & Renshaw
Global Healthcare Conference
May 16, 2006
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
4
Investment Considerations
 A Leader in Regenerative Medicine – the Next Frontier in Medicine
 Product Focused – Driving Human ES Cell Therapies to the Clinic
 Intellectual Property – Proprietary Human ES Cell Technology
 Human Capital – Talented Team of Scientists and Management
 Financial Resources – Strong Cash Position and Public Currency
Realizing the Potential of
Human Embryonic Stem Cells
6
Stem Cells are a Proven Therapy
 First successful bone marrow transplant (BMT) in 1968
 Tens of thousands of patients have been cured with BMT
 Human embryonic stem cells first isolated in 1998
 hESC’s have significant potential to treat diseases
7
The Potential of Human ES Cells - Pluripotency
8
ES Cells Form Complex Tissues
Science 2002 295: 819
PNAS 2003, 100 Suppl 1:11911
Intestine Muscle Retina
Hair / Skin Cartilage Bone
9
The Potential of Human ES Cells
Beating heart cellsBeating heart cells
Regenerative Medicine
“The New Frontier”
11
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
12
Growing Legislative Support
 California & Proposition 71
 Voters approve $3.0 billion in funding over next ten years
 Formation of CA Institute of Regenerative Medicine (CIRM)
 Recent awards of initial training grants
 Court trial complete
 CIRM public discussions – funding 2007
 Other State Initiatives
 MA, CT, NJ, NY, IL, FL
 Federal Legislation
 Bi-partisan House of Representatives Bill H.R. 810
 Federal Funding for Alternative Methods of Deriving Human ES Cells
13
The Next Frontier in Medicine
"If the potential of stem cell research is realized, it would mean an end to the suffering of
millions of people. If stem cell research succeeds, there isn’t a person in the country who
won’t benefit, or know somebody who will."
Michael J. Fox
“[Stem cell research] is the most promising research in healthcare perhaps in this history of the
world and we should not be left behind in this research.”
Senator Orin Hatch, (R-Utah)
“Science has presented us with a hope called stem-cell research, which may provide our
scientists with answers that have so long been beyond our grasp.”
Nancy Reagan
“This is not a matter of using a human embryo that has the potential to produce life. Rather,
these otherwise discarded embryos have the potential to save lives.”
Senator Arlen Specter (R-PA)
ACT Business Strategy
15
Focused Business Strategy
 Driving Human ES Cell Therapies to the Clinic
 Solving Immune Rejection and the Ethical Debate
 Leveraging Proprietary Technology Platform
 Expanding Collaborations and Corporate Partnerships
16
Driving Human ES Cell Therapies to the Clinic
17
Driving Human ES Cell Therapies to the Clinic
18
Driving Human ES Cell Therapies to the Clinic
19
Our Product Development Pathway
Cell Discovery and Validation
Finding Cell Types with Therapeutic Potential
Initial FDA Discussion for Target Indication
“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
20
Cell Therapy – RPE cells
Age-related diseases of the eye
Over 10 million cases MDG in U.S.
Early functional tests show therapeutic
potential
Next steps:
Publish animal data in 2006
GMP cell production in MA
21
Cell Therapy – Hemangioblast Cells
Potential treatment for multiple
cardiac and blood disorders
Published data shows
therapeutic potential in mouse
model
Large market potential
Cells isolated in MA labs
Next steps:
Initiate key collaborations
Publish animal data
Circulation Research 2004, 94: 820
22
Cell Therapy – Dermal Cells
Unique capacity of early skin cells
Large applications in burns, wound
repair and surgery
Cells isolated in CA labs
Next steps:
Initiate key collaborations
Publish animal data
23
Solving Immune Rejection and the Ethical Debate
 Two Significant Challenges to Regenerative Medicine Today
 Solving Rejection of Cell Therapies by a Patient’s Immune System
 Addressing the Ethical Debate Surrounding the Derivation of Human
ES Cell
 ACT Uses Proprietary Cellular Reprogramming to Address Both
Challenges
24
 Chronic Diseases or Indications Requiring Custom Therapy
 Today - Somatic Cell Nuclear Transfer (“SCNT”)
 Tomorrow – Transdifferentiation
 Result - a custom-tailored, patient-specific cell therapy for patient
 Acute Injuries or Indications Requiring Immediate Therapy
 Tomorrow – Our Reduced Complexity Library (“RCL”)
 Result – cell therapies for the immediate treatment of acute injuries which will
provide a close match for most patients
Solving Immune Rejection – Cellular
Reprogramming for “Chronic versus Acute”
25
Somatic Cell Nuclear Transfer – “SCNT”
26
Reduced Complexity Library
ACT Reduced Complexity Library
HLA matched cell
line for patient
Act 1Act 1
Act 2Act 2
Act 5Act 5
Act 6Act 6
Act 7Act 7Act 3Act 3
Act 8Act 8Act 4Act 4
27
Strong IP in Alternative Technologies
10.17.05
Scientists Find New Ways to
Derive Embryonic Stem
Cells
- Forbes
Scientists Devise New Stem Cell
Methods to Ease Concerns
- The New York Times
28
No Damage to the Embryo
29
Leveraging Proprietary Technology Platform
escellsescells
patient-specificpatient-specific
hemangioblashemangioblas
tt
dermaldermal
ALTERNATIVES / PGD
TRADITIONAL ES CELLS
CELL DIFFERENTIATION
HISTOCOMPATIBILITY / SCNT
TRANSDIFFERENTIATION
TECHNOLOGIESTECHNOLOGIES
INTELLECTUAL PROPERTYINTELLECTUAL PROPERTY
SCIENTIFIC TEAMSCIENTIFIC TEAM
RCLRCL
escellsescells
RPERPE
30
Expanding Academic Collaborations
and Corporate Partnerships
Collaboration potential:
•Existing scientific and
academic relationships
•CA Proposition 71
•Large BIO / Pharma
DIFFERENTIATION/COW OOCYTE
HISTOCOMPATIBILITY/SCNT
patient-specificpatient-specific
ALTERNATIVE/PGD
TRADITIONAL ES CELLS CELL DIFFERENTIATION
rperpe
dermaldermal
hemangioblasthemangioblast
rclrcl
escellsescells
Patient-specificPatient-specific
TRANSDIFFFERENTIATION
HISTOCOMPATIBILITY/SCNT
TECHNOLOGIESTECHNOLOGIES
INTELLECTUAL PROPERTYINTELLECTUAL PROPERTY
SCIENTIFIC TEAMSCIENTIFIC TEAM
31
Significant Collaboration Potential
32
Key Financial Information
 Strong Cash Position – recent PIPE financing
 Funding Status
 Funded into 2007
 Potential additional $8.875 million in Convertible Notes
 Conversion of notes at $4.60 per share stock price
 Public Market Status
 OTC-BB:ACTC
 Apply for listing on Amex or NASDAQ Capital Market
 Recent share price of $1.30 – 1.50
 Approximate fully-diluted market cap of $80 - 95 million
33
Senior Management Team
Mr. William Caldwell, IV, Chief Executive Officer
30 Year Career in Management and Finance
Dr. Michael West, Chairman, Chief Scientific Officer and President
Founder of Geron and ACT
Dr. Robert Lanza, VP of Medical & Scientific Development
25 Year Career in Biomedical and Scientific Research
Mr. James Stewart, SVP, Chief Financial Officer
25 Year Career in Accounting, Finance
Mr. Jon Atzen, SVP, General Counsel
15 Year Legal Career in Corporate and Securities Law
34
2005 Corporate Milestones - Achieved
 Expanded Senior Management and Scientific Team
 Raised over $25 million in capital
 Series A Financing of $8.0 million in January
 Convertible Note Financing of $17.75 million in September
 Opened California location – significiant GMP capacity
 Formed California Collaborations
 Defined Product Areas of Focus
 Retinal, Dermal and Hemangioblasts
 Published on Alternatives for Derivation of ES Cells – Nature 2005
35
2006 Corporate Milestones - Objectives
 “GMP” Cell Production
 Derive Human ES Cells under “GMP”
 Differentiate and Scale Production of “GMP” cells
 Launch Preclinical and Clinical Plan
 Recruit Senior Clinical Executive
 Conduct Preclinical Testing of “GMP” cells
 Work with FDA to expedite human clinical trials
 Define clear path to initial product IND
 Expand Technology Platform with Scientific Breakthroughs
 Achieve Listing on AMEX or NASDAQ Capital Market
 Announce Corporate Partnership
36
Summary
 A Leader in Regenerative Medicine – the Next Frontier in Medicine
 Product Focused – Driving Human ES Cell Therapies to the Clinic
 Intellectual Property - Proprietary Human ES Cell Technology
 Human Capital – Talented Team of Scientists and Management
 Financial Resources – Strong Cash Position and Public Currency
Rodman & Renshaw Global Healthcare Conference Presentation, May 2006

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Rodman & Renshaw Global Healthcare Conference Presentation, May 2006

  • 1. Rodman & Renshaw Global Healthcare Conference May 16, 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
  • 3.
  • 4. 4 Investment Considerations  A Leader in Regenerative Medicine – the Next Frontier in Medicine  Product Focused – Driving Human ES Cell Therapies to the Clinic  Intellectual Property – Proprietary Human ES Cell Technology  Human Capital – Talented Team of Scientists and Management  Financial Resources – Strong Cash Position and Public Currency
  • 5. Realizing the Potential of Human Embryonic Stem Cells
  • 6. 6 Stem Cells are a Proven Therapy  First successful bone marrow transplant (BMT) in 1968  Tens of thousands of patients have been cured with BMT  Human embryonic stem cells first isolated in 1998  hESC’s have significant potential to treat diseases
  • 7. 7 The Potential of Human ES Cells - Pluripotency
  • 8. 8 ES Cells Form Complex Tissues Science 2002 295: 819 PNAS 2003, 100 Suppl 1:11911 Intestine Muscle Retina Hair / Skin Cartilage Bone
  • 9. 9 The Potential of Human ES Cells Beating heart cellsBeating heart cells
  • 11. 11 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
  • 12. 12 Growing Legislative Support  California & Proposition 71  Voters approve $3.0 billion in funding over next ten years  Formation of CA Institute of Regenerative Medicine (CIRM)  Recent awards of initial training grants  Court trial complete  CIRM public discussions – funding 2007  Other State Initiatives  MA, CT, NJ, NY, IL, FL  Federal Legislation  Bi-partisan House of Representatives Bill H.R. 810  Federal Funding for Alternative Methods of Deriving Human ES Cells
  • 13. 13 The Next Frontier in Medicine "If the potential of stem cell research is realized, it would mean an end to the suffering of millions of people. If stem cell research succeeds, there isn’t a person in the country who won’t benefit, or know somebody who will." Michael J. Fox “[Stem cell research] is the most promising research in healthcare perhaps in this history of the world and we should not be left behind in this research.” Senator Orin Hatch, (R-Utah) “Science has presented us with a hope called stem-cell research, which may provide our scientists with answers that have so long been beyond our grasp.” Nancy Reagan “This is not a matter of using a human embryo that has the potential to produce life. Rather, these otherwise discarded embryos have the potential to save lives.” Senator Arlen Specter (R-PA)
  • 15. 15 Focused Business Strategy  Driving Human ES Cell Therapies to the Clinic  Solving Immune Rejection and the Ethical Debate  Leveraging Proprietary Technology Platform  Expanding Collaborations and Corporate Partnerships
  • 16. 16 Driving Human ES Cell Therapies to the Clinic
  • 17. 17 Driving Human ES Cell Therapies to the Clinic
  • 18. 18 Driving Human ES Cell Therapies to the Clinic
  • 19. 19 Our Product Development Pathway Cell Discovery and Validation Finding Cell Types with Therapeutic Potential Initial FDA Discussion for Target Indication “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
  • 20. 20 Cell Therapy – RPE cells Age-related diseases of the eye Over 10 million cases MDG in U.S. Early functional tests show therapeutic potential Next steps: Publish animal data in 2006 GMP cell production in MA
  • 21. 21 Cell Therapy – Hemangioblast Cells Potential treatment for multiple cardiac and blood disorders Published data shows therapeutic potential in mouse model Large market potential Cells isolated in MA labs Next steps: Initiate key collaborations Publish animal data Circulation Research 2004, 94: 820
  • 22. 22 Cell Therapy – Dermal Cells Unique capacity of early skin cells Large applications in burns, wound repair and surgery Cells isolated in CA labs Next steps: Initiate key collaborations Publish animal data
  • 23. 23 Solving Immune Rejection and the Ethical Debate  Two Significant Challenges to Regenerative Medicine Today  Solving Rejection of Cell Therapies by a Patient’s Immune System  Addressing the Ethical Debate Surrounding the Derivation of Human ES Cell  ACT Uses Proprietary Cellular Reprogramming to Address Both Challenges
  • 24. 24  Chronic Diseases or Indications Requiring Custom Therapy  Today - Somatic Cell Nuclear Transfer (“SCNT”)  Tomorrow – Transdifferentiation  Result - a custom-tailored, patient-specific cell therapy for patient  Acute Injuries or Indications Requiring Immediate Therapy  Tomorrow – Our Reduced Complexity Library (“RCL”)  Result – cell therapies for the immediate treatment of acute injuries which will provide a close match for most patients Solving Immune Rejection – Cellular Reprogramming for “Chronic versus Acute”
  • 25. 25 Somatic Cell Nuclear Transfer – “SCNT”
  • 26. 26 Reduced Complexity Library ACT Reduced Complexity Library HLA matched cell line for patient Act 1Act 1 Act 2Act 2 Act 5Act 5 Act 6Act 6 Act 7Act 7Act 3Act 3 Act 8Act 8Act 4Act 4
  • 27. 27 Strong IP in Alternative Technologies 10.17.05 Scientists Find New Ways to Derive Embryonic Stem Cells - Forbes Scientists Devise New Stem Cell Methods to Ease Concerns - The New York Times
  • 28. 28 No Damage to the Embryo
  • 29. 29 Leveraging Proprietary Technology Platform escellsescells patient-specificpatient-specific hemangioblashemangioblas tt dermaldermal ALTERNATIVES / PGD TRADITIONAL ES CELLS CELL DIFFERENTIATION HISTOCOMPATIBILITY / SCNT TRANSDIFFERENTIATION TECHNOLOGIESTECHNOLOGIES INTELLECTUAL PROPERTYINTELLECTUAL PROPERTY SCIENTIFIC TEAMSCIENTIFIC TEAM RCLRCL escellsescells RPERPE
  • 30. 30 Expanding Academic Collaborations and Corporate Partnerships Collaboration potential: •Existing scientific and academic relationships •CA Proposition 71 •Large BIO / Pharma DIFFERENTIATION/COW OOCYTE HISTOCOMPATIBILITY/SCNT patient-specificpatient-specific ALTERNATIVE/PGD TRADITIONAL ES CELLS CELL DIFFERENTIATION rperpe dermaldermal hemangioblasthemangioblast rclrcl escellsescells Patient-specificPatient-specific TRANSDIFFFERENTIATION HISTOCOMPATIBILITY/SCNT TECHNOLOGIESTECHNOLOGIES INTELLECTUAL PROPERTYINTELLECTUAL PROPERTY SCIENTIFIC TEAMSCIENTIFIC TEAM
  • 32. 32 Key Financial Information  Strong Cash Position – recent PIPE financing  Funding Status  Funded into 2007  Potential additional $8.875 million in Convertible Notes  Conversion of notes at $4.60 per share stock price  Public Market Status  OTC-BB:ACTC  Apply for listing on Amex or NASDAQ Capital Market  Recent share price of $1.30 – 1.50  Approximate fully-diluted market cap of $80 - 95 million
  • 33. 33 Senior Management Team Mr. William Caldwell, IV, Chief Executive Officer 30 Year Career in Management and Finance Dr. Michael West, Chairman, Chief Scientific Officer and President Founder of Geron and ACT Dr. Robert Lanza, VP of Medical & Scientific Development 25 Year Career in Biomedical and Scientific Research Mr. James Stewart, SVP, Chief Financial Officer 25 Year Career in Accounting, Finance Mr. Jon Atzen, SVP, General Counsel 15 Year Legal Career in Corporate and Securities Law
  • 34. 34 2005 Corporate Milestones - Achieved  Expanded Senior Management and Scientific Team  Raised over $25 million in capital  Series A Financing of $8.0 million in January  Convertible Note Financing of $17.75 million in September  Opened California location – significiant GMP capacity  Formed California Collaborations  Defined Product Areas of Focus  Retinal, Dermal and Hemangioblasts  Published on Alternatives for Derivation of ES Cells – Nature 2005
  • 35. 35 2006 Corporate Milestones - Objectives  “GMP” Cell Production  Derive Human ES Cells under “GMP”  Differentiate and Scale Production of “GMP” cells  Launch Preclinical and Clinical Plan  Recruit Senior Clinical Executive  Conduct Preclinical Testing of “GMP” cells  Work with FDA to expedite human clinical trials  Define clear path to initial product IND  Expand Technology Platform with Scientific Breakthroughs  Achieve Listing on AMEX or NASDAQ Capital Market  Announce Corporate Partnership
  • 36. 36 Summary  A Leader in Regenerative Medicine – the Next Frontier in Medicine  Product Focused – Driving Human ES Cell Therapies to the Clinic  Intellectual Property - Proprietary Human ES Cell Technology  Human Capital – Talented Team of Scientists and Management  Financial Resources – Strong Cash Position and Public Currency