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The PIPEs Conference Presentation, October 2005

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The PIPEs Conference Presentation, October 2005

  1. 1. ConfidentialConfidentialThe PIPEs Conference 2005October 27, 2005
  2. 2. 2This presentation contains “forward-looking statements” as defined under thefederal securities laws. Actual results could vary materially. Factors that couldcause actual results to vary materially are described in our filings with the Securitiesand Exchange Commission.You should pay particular attention to the “risk factors” contained in documents wefile from time to time with the Securities and Exchange Commission. The risksidentified therein, as well as others not identified by the Company, could cause theCompany’s actual results to differ materially from those expressed in any forward-looking statements.Cautionary Statement ConcerningForward-Looking Statements
  3. 3. 4Investment 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
  4. 4. Realizing the Potential ofHuman Embryonic Stem Cells
  5. 5. 6The Potential of Human ES Cells - Pluripotency
  6. 6. 7ES Cells Form Complex TissuesScience 2002 295: 819PNAS 2003, 100 Suppl 1:11911Intestine Muscle RetinaHair / Skin Cartilage Bone
  7. 7. 8The Potential of Human ES Cells
  8. 8. Regenerative Medicine“The New Frontier”
  9. 9. 10Regenerative MedicineCuring Devastating Disease800,000Parkinsons patients 750,000liver disease patients20,000,000heart disease patients16,000,000 diabetespatients5,000,000epilepsy patients4,000,000Alzheimers patients2,000,000stroke patients300,000kidney disease patients
  10. 10. 11Growing 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 Other State Initiatives MA, CT, NJ, NY, IL, MO, KS, FL Federal Legislation Bi-partisan House of Representatives Bill H.R. 810 Federal Funding for Alternative Methods of Deriving Human ES Cells
  11. 11. 12The Next Frontier in Medicine"If the potential of stem cell research is realized, it would mean an end to the suffering ofmillions of people. If stem cell research succeeds, there isn’t a person in the country whowon’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 theworld 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 ourscientists 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)
  12. 12. ACT Business Strategy
  13. 13. 14Focused 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
  14. 14. 15Driving Human ES Cell Therapies to the Clinic
  15. 15. 16Driving Human ES Cell Therapies to the Clinic
  16. 16. 17Driving Human ES Cell Therapies to the Clinic
  17. 17. 18Our Product Development PathwayCell Discovery and Validation“GMP” Cell ProductionPreclinical and Clinical Testingtarget cell typestarget cell typestarget cell typestarget cell types
  18. 18. 19Cell Therapy – RPE cellsAge-related diseases of the eyeOver 10 million cases MDG in U.S.Early functional tests show therapeuticpotentialGMP cell production – next step
  19. 19. 20Cell Therapy – Hemangioblast CellsPotential treatment for multiplecardiac and blood disordersPublished data showstherapeutic potential in mousemodelLarge market potentialIn cell discovery nowCirculatory Research 2004, 94: 820
  20. 20. 21Cell Therapy – Dermal CellsUnique capacity of early skin cellsLarge applications in burns, woundrepair and surgeryIn cell discovery now
  21. 21. 22Solving Immune Rejection and the Ethical Debate Two Greatest Challenges to Regenerative Medicine Today Rejection of cell therapies by a patient’s immune system Ethical debate surrounding the derivation of human ES cells ACT’s Novel Scientific Discoveries Address Challenges SCNT for custom-tailored cell therapy Parthenogenesis for immediate or “off-the-shelf” cell therapy Single Cell Biopsy as an alternative method to derive ES cells Transdifferentiation has the potential to solve both challenges
  22. 22. 23 Chronic Needs or Indications Requiring Custom Therapy Today - Somatic Cell Nuclear Transfer (“SCNT”) Tomorrow – Transdifferentiation Acute Needs or Indications Requiring Immediate Therapy Using Parthenogenesis to create a Reduced Complexity Library (“RCL”) In either case the patient wins!Solving Immune Rejection“Chronic versus Acute”
  23. 23. 24Somatic Cell Nuclear Transfer – “SCNT”
  24. 24. 25Reduced Complexity LibraryACT Reduced Complexity LibraryHLA matched cellline for patientAct 1Act 1Act 2Act 2Act 5Act 5Act 6Act 6Act 7Act 7Act 3Act 3Act 8Act 8Act 4Act 4
  25. 25. 26Strong IP in Alternative Technologies10.17.05Scientists Find New Ways toDerive Embryonic StemCells- ForbesScientists Devise New Stem CellMethods to Ease Concerns- The New York Times
  27. 27. 28Expanding Academic Collaborationsand Corporate PartnershipsCollaboration potential:•Existing scientific andacademic relationships•CA Proposition 71•Large BIO / PharmaDIFFERENTIATION/COW OOCYTEHISTOCOMPATIBILITY/SCNTpatient-specificpatient-specificALTERNATIVE/PGDTRADITIONAL ES CELLS CELL DIFFERENTIATIONrperpedermaldermalhemangioblasthemangioblastrclrclescellsescellsPatient-specificPatient-specificDIFFERENTIATION/COW OOCYTEHISTOCOMPATIBILITY/SCNTTECHNOLOGIESTECHNOLOGIESINTELLECTUAL PROPERTYINTELLECTUAL PROPERTYSCIENTIFIC TEAMSCIENTIFIC TEAM
  28. 28. 29Key Financial Information Strong Cash Position of $16 million Funding Status Funded into 2007 Potential additional $8.875 million in Convertible Notes – Funding into 2008 Conversion of notes at $4.60 per share stock price Public Market Status OTC-BB:ACTC Apply for listing on Amex or NASDAQ SmallCap Recent share price of $2.65 Approximate fully-diluted market cap of $125 - 150 million
  29. 29. 30Senior Management TeamMr. William Caldwell, IV, Chief Executive Officer30 Year Career in Management and FinanceDr. Michael West, Chairman, Chief Scientific Officer and PresidentFounder of Geron and ACTDr. Robert Lanza, VP of Medical & Scientific Development25 Year Career in Biomedical and Scientific ResearchMr. James Stewart, SVP, Chief Financial Officer25 Year Career in Accounting, FinanceMr. Jon Atzen, SVP, General Counsel15 Year Legal Career in Corporate and Securities Law
  30. 30. 312005 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 Formed California Collaborations Defined Product Areas of Focus Retinal, Dermal and Hemangioblasts Published on Alternatives for Derivation of ES Cells – Nature 2005
  31. 31. 322006 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 Expand Technology Platform with Scientific Breakthroughs Achieve Listing on AMEX or NASDAQ SmallCap Announce Corporate Partnership Raise Additional Capital
  32. 32. 33Summary 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
  33. 33. ConfidentialConfidential