BioEurope Presentation, March 2010


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BioEurope Presentation, March 2010

  1. 1. Advanced Cell TechnologyCorporate PresentationMarch 20101
  2. 2. This presentation is intended to present a summary of ACT’s (“ACT”, or “AdvancedCell Technology Inc”, or “the Company”) salient business characteristics.The information herein contains “forward-looking statements” as defined under thefederal securities laws. Actual results could vary materially. Factors that could causeactual results to vary materially are described in our filings with the Securities andExchange 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 Concerning Forward-LookingStatements2
  3. 3. ACT TherapeuticsACT Proprietary HumanTherapeutic ProgramsTreatment Clinical StageBlastomere ProgramDevelopment of embryonic stem cell lines withoutdestruction of embryoPre-ClinicalRetinal Pigment Epithelium(RPE) ProgramTreatment of Age-related Macular Degeneration(AMD) and Retinal Degenerative DiseasesClinical INDawaitingFDA approvalMyoblast ProgramTreatment of Heart Disease, Heart Attack andHeart FailurePhase IIHemangioblast programTreatment of Diseases and Disorders of theCirculatory and Vascular SystemsPre-Clinical3
  4. 4. PendingMultiple next generation technologies complementary toexisting patents and claimsPendingMultiple use patents for heart diseaseStatusUse PatentsPendingCombinations of myoblasts and stem cellsPendingCombinations of myoblasts and muscle survival factorsPendingCatheter delivery of cellsAwardedMedia for preservation and transplant of muscle cellsAwardedStem cells capable of forming nerve or muscle cellsAwardedMuscle cellsStatusComposition of Matter PatentIntellectual Property ProtectionACT owns or licenses over 150 patents and patent applications related tostem cell and regenerative therapies4
  5. 5. Institutional CollaboratorsAdvanced Cell’s Institutional Collaborators include:Casey Eye Institute Mayo ClinicMoran Eye Institute UCSFHarvard Johns HopkinsStanford Sloan KetteringUniversity of Florida University of IowaUniversity of Illinois U.C. BerkeleyColorado State University5
  6. 6. Blastomere Program: Synthesizing Stem Cellswithout Harm to the Embryo•Company scientists successfully generate stemcell lines without destruction of embryo•Cell lines retain potential to form all cells in thehuman body•Potential to treat a range of human diseases•Technology has been reproduced and peer-reviewed on several occasions•National Institute of Health (NIH) proposingexpanded funding regulations to accommodateACT’s lines6
  7. 7. First Proven Alternative hESC Method•Enables Derivation of New hESC Linesvia Pre-implantation Genetic Diagnosis(PGD) Method, Preserving DevelopmentPotential of the Embryo•7 hESC lines awaiting NIH approval forfunding•Technology is used to develop RPE cells forour clinical trials for Stargardt’s diseaseHemangioblastsDifferentiated fromBlastomere hESC Lines7
  8. 8. RPE Program: Why RPE?• Pigmented epithelium cells are easy to identify• Small dosage vs. other therapies• The eye is an immune-privileged site, thus no riskof rejection at the injection site• Ease of administration– Doesn’t require a separate approval by the FDA (universal applicator)– Procedure is already used by eye surgeons; no new skill set requiredfor doctors• RPE cell therapy may positively impact over 200 retinal diseases8
  9. 9. RPE Program StatusComplete Stage 1 of Phase I Clinical Trial forStargardt’s disease2010 – 4th QuarterDeveloped the “Proof of Concept” for ACT’s RPEcells in RCS animal model2006Pre – IND meeting with FDA2008Filed IND for Stargardt’s disease2009FDA approval2010 – 3rd Quarter9
  10. 10. GMP-compliant Human RPE Cells Derived from Embryonic Stem CellLines Rescue Visual Function In a Rat Model for PhotoreceptorDegeneration“Our results show that a well-characterized GMP-compliant embryonic stem cell-derived RPEcells can survive after transplantation to the sub-retinal space of RCS rats, integrate into hostRPE layer without migration into the retina and continue to express at least some of themolecules characteristic of RPE. The cells are effective in rescuing photoreceptors fromdegeneration and result in significant rescue of visual function over sham-operated animals forthe long term.”CASEY EYEINSTITUTE10
  11. 11. Myoblast Program HighlightsTarget Market for Myoblast ProgramSufferers of Heart Failure, Chronic HeartFailure and patients with scarred orischemic (dead) heart tissue caused byor related to heart attackProgram StatusClearance from FDA to Proceed withPhase II Clinical Trials in the U.S.Charlestown, MA GMP Facility11
  12. 12. Myoblast Program: Clinical Trial SummaryPhase 1LVADPhase 1CABGPhase 1bCABGPhase 1bCatheterPatients 6 patients 12 patients 12 patients23 Patients(12 treated,11 control)IndicationLVAD Bridgeto Transplant+CABG +CABG Catheter InjectionDoseSingle Dose -300 MM CellsEscalating –10, 30, 100 and300 MM CellsSingle Dose -300 MM CellsEscalating -30, 100, 300 and600 MM CellsPrimaryEndpointSafety Safety Safety SafetyResultDemonstratedcell survival inhumansDoseescalation, Safeat all dosesSurvival of cells(cardiacimaging),improvedsymptoms andfunctionSafe at all doses,improvedsymptoms andfunction12
  13. 13. Myoblast Program: Phase II Clinical Trial 80 total patients (1:1 treatment vs. control) Double-blind, placebo-controlled (sham procedure) Catheter delivery of cells: percutaneous targeted delivery Three-month, six-month, and 12-month follow-up Endpoints: Improvement in HF symptoms and VentricularVolumes Primary endpoint: Improvement in heart failure symptomsmeasured by “Kansas City Cardiomyopathy Questionnaire” Supporting endpoints: Ventricular volumes, Six minute walk test Interim data analysis at six-months by Independent Review Board13
  14. 14. Hemangioblast Program: Partnership• Joint Venture with leading Korean stem celldeveloper CHA Biotech Co.• The J.V., ‘Stem Cell & RegenerativeMedicine International’, is focused on thedevelopment of human blood cells andrelated products14
  15. 15. Hemangioblast Program: OverviewThe HG cell is the precursor to all cell types in thecirculatory and vascular systems15
  16. 16. Hemangioblast Program: Synthesizing Blood Cells16
  17. 17. The ACT Therapeutic TimelineTimetable for Therapeutic Program Milestones PeriodReceived Orphan indication from Department of Healthand Human ServicesQ1 2010Completion of end of life tumorgenicity & biodistributionQ1 2010Receive FDA clearance to commence with Phase I ClinicalTrialsQ3 2010Treat 1st Patient in history with a hECS-based therapy Q3 20101st Quadrant Safety Data on Treated Patients Q4 2010File 2nd Phase I IND for another Retinal Disease2nd half2010Announce 2nd cellular-based IND in area other thanretinal program2nd half2010studies for RPE17
  18. 18. The Advanced Cell Technology TeamWorld Class Scientific Team lead bySeasoned Management Team:Dr. Robert Lanza, M.D. – Chief Scientific OfficerDr. Jonathan Dinsmore, PhD – Myoblast Project AdvisorEdmund Mickunas – Vice President of RegulatoryWilliam Caldwell IV – Chief Executive OfficerRita Parker – Director of OperationsRoger Gay, PhD – Senior Director of ManufacturingMatthew Vincent, PhD – IP / Licensing Advisor18
  19. 19. State of the Company• Two years of cash available• Phase I/II trial fully funded• Anticipating cell lines will qualify forfederal funding• Actively pursuing development partnersoffshore to accelerate development ofprograms19
  20. 20. Thank you for your timeFor more information, visit www.advancedcell.comAdvanced Cell Technology is traded on the OTC BB, symbol: ACTC