ACTC Company Presentation, July 2007
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ACTC Company Presentation, July 2007 ACTC Company Presentation, July 2007 Presentation Transcript

  • Company PresentationJuly 2007
  • 2This presentation contains “forward-looking statements” as defined underthe federal securities laws. Actual results could vary materially. Factorsthat could cause actual results to vary materially are described in our filingswith the Securities and Exchange Commission.You should pay particular attention to the “risk factors” contained indocuments we file from time to time with the Securities and ExchangeCommission. The risks identified therein, as well as others not identified bythe Company, could cause the Company’s actual results to differ materiallyfrom those expressed in any forward-looking statements.Legal NoticeLegal NoticeCautionary Statement ConcerningForward-Looking Statements
  • 3Stem Cell Based TherapyStem Cell Based TherapyScientists worldwide believe stem cell-based therapies have the potential torestore healthy function of diseasedor damaged tissuesGoal is to develop therapies that workby transplanting cells and tissuesderived from stem cells into thebody, using the body’s naturalpowers of regeneration and healing
  • 4Company MissionCompany MissionTranslating World Class Stem CellResearch into Proprietary HumanTherapeutic Programs
  • 5Recent EventsRecent EventsDriving Forward into the ClinicAcquiring Mytogen and Myoblast ProgramPhase II Clinical Trial for the Treatment of Chronic Heart FailureCommencing GLP Studies for RPE ProgramPreclinical Safety Studies for the Treatment of AMD and RetinalDegenerative DiseaseCommencing Preclinical Studies for Hemangioblast ProgramPreclinical Studies for the Replacement of Red Blood Cells andthe Treatment of Eye Disease (Diabetic Retinopathy and BranchVein Occlusion)
  • 6Key Highlights of Mytogen AcquisitionKey Highlights of Mytogen AcquisitionTarget Market for Myoblast ProgramChronic Heart Failure - treat scar tissuecaused by heartattackClearance from FDA to Proceed withPhase II Clinical TrialsExperienced Mytogen DevelopmentTeam – Together for over Ten YearsStrong Intellectual Property – MytogenLicensed from GenVec who Acquiredfrom DiacrinTransaction to Close in July – ACTAcquiring Mytogen for Common StockCharlestown, MA GMP Facility
  • 7Key Highlights of Mytogen AcquisitionKey Highlights of Mytogen AcquisitionGained Clearance from FDA to Proceedwith Phase II Multi-Center Clinical Trial3 Phase I Clinical Studies CompletedDosed in 42 patientsNo safety concernsStrong indications of efficacyPhase lb Catheter Trial Nearly CompleteNo adverse effects associated with cells,no arrhythmia concernsHighly significant improvement in NYHAclassificationImprovement in ventricular volumes2. ControlAverageNYHAClass3 mos: p = 0.0009, n = 236 mos: p < 0.0001, n = 23A decrease in NYHA score represents animprovement in symptoms and an increaserepresents a worsening.Phase Ib Clinical Trial Results – NYHA Classification
  • 8Key Investment HighlightsKey Investment HighlightsACT Proprietary Human Therapeutic ProgramsMyoblast Program Treatment of Heart Attack Phase IIand Heart FailureRPE Program Treatment of AMD and Retinal PreclinicalDegenerative DiseasesHemangioblast Treatment of Disease and Disorders PreclinicalProgram of the Blood and Vascular SystemDermal Program Treatment of Wounds, Burns Preclinicaland Scars
  • 9Key Investment HighlightsKey Investment HighlightsACT Proprietary Enabling Technology PlatformsBlastomere Program First Proven Alternative Method for hESC DerivationACTCellerate High Throughput Derivation and Differentiation ofProgram Therapeutic Cell TypesGMP Manufacturing Producing GMP-Compliant, Pathogen-Free BanksProgram of hESCs and Therapeutic Cell Types
  • 10World Class Scientific Team Lead By:Dr. Michael West President and Chief Scientific OfficerDr. Robert Lanza VP of Research & Scientific DevelopmentDr. Pedro Huertas Chief Development OfficerIndustry-Leading Intellectual Property PortfolioMore than 300 patents and patent applications related tostem cell therapy and regenerative medicineKey Investment HighlightsKey Investment Highlights
  • ACT Therapeutics:Myoblast Program
  • 12Myoblast Stem Cell TherapyMyoblast Stem Cell TherapyOutpatient therapy to improvecardiac function in heart failurepatientsHarvest muscle stem cells(Myoblasts) from patient’s thighExpand cells using proprietaryculturing processIdentify and map scarred regionsof the heart with 3-D imagingtechniqueInject cells with minimally-invasivecardiac catheter: no generalanesthesia
  • 13Myoblast Clinical Trial SummaryMyoblast Clinical Trial SummaryEscalating -30, 100, 300 and600 MM CellsSingle Dose -300 MM CellsEscalating –10, 30, 100 and300 MM CellsSingle Dose -300 MM CellsDoseSafe at all doses,improvedsymptoms andfunctionSafetyCatheter Injection23 Patients(12 treated,11 control)Phase 1bCatheterResultPrimaryEndpointIndicationPatientsPhase 1bCABGPhase 1CABGPhase 1LVADSurvival of cells(cardiac imaging),improvedsymptoms andfunctionDose escalation,Safe at all dosesDemonstratedcell survival inhumansSafetySafetySafety+CABG+CABGLVAD Bridge toTransplant12 patients12 patients6 patients
  • 14Phase Ib Catheter Trial SummaryPhase Ib Catheter Trial SummaryFirst U.S. randomized trial of catheter-delivered muscle cellsCore laboratory analysis of objective endpointsOnly trial with direct injection into scar (other stem cells mustbe delivered to viable tissue for benefit)Catheter delivery process without any complicationsNo deaths, no loss to follow-upOngoing follow-up data
  • 153-D NOGA Mapping of Myoblast Injections3-D NOGA Mapping of Myoblast Injections5.7 mV 8.6 mVInjections of 600 million cells intoinfarct region shown in red3 months post reveals significantimprovement in viability of the heart
  • 16Phase Ib Clinical Trial ResultsPhase Ib Clinical Trial Results2. ControlAverageNYHAClass3 mos: p = 0.0009, n = 236 mos: p < 0.0001, n = 23A decrease in NYHA score represents an improvement insymptoms and an increase represents a worseningNYHA Classification of Heart Failure SymptomsBaseline 3mos 6mos Baseline 3mos 6mos
  • 17Phase Ib Clinical Trial ResultsPhase Ib Clinical Trial Results-0.6-0.4-1-1.40.2-1.6-1.4-1.2-1-0.8-0.6-0.4-ß-blockersACE InhibitorsCRTMyoblastsControl ß-blockers* ACE Inhibitors#CRT†Myoblasts* Lombardo et al., 2006 Am J Cardiovasc Drugs 6:259-63†McAllister et al., 2004 Ann Intern Med 141:381-90# Sliwa et al., 2004 JACC 44:1825-30Change in NYHA at 6mos post treatmentComparative Effects of Heart Failure Treatments on NYHA
  • 18Phase Ib Clinical Trial ResultsPhase Ib Clinical Trial ResultsMinnesota Living with Heart Failure Questionnaire (MLHFQ)47 47284601020304050Myoblast Controlp = 0.017, n = 23Baseline 6mos Baseline 6mos* Improvement in patient quality of life (QOL) scores for all patients
  • 19Phase Ib Clinical Trial ResultsPhase Ib Clinical Trial ResultsAverage change in distance walked in 6 minutes from 3 months to 6 monthsfor all patients81-9-50050100FeetMyoblast Control6 Minute Walk Test
  • 20Phase Ib Clinical Trial ResultsPhase Ib Clinical Trial ResultsVentrical Volumes: End Systolic and End Diastolic DiametersEnd Systolic Diameter at 3 and 6 mos as measured byindependent echocardiography core lab for all patients5.524.895.44.995.415.084.84.955. Controlcmp = 0.06, n = 23Baseline 3mos 6mos Baseline 3mos 6mosEnd Diastolic Diameter at 3 and 6 mos as measured byindependent echocardiography core lab for all patients6.416.26.386.276.396.3766. Controlcmp = 0.07, n = 23Baseline 3mos 6mos Baseline 3mos 6mos
  • 21Phase Ib Clinical Trial ResultsPhase Ib Clinical Trial ResultsNYHA Classification at 6 month vs Baseline for all Patients36%64%0%Control0%17%83%MyoblastWorsenedNo ChangeImproved
  • 22Plan for Phase II Clinical TrialPlan for Phase II Clinical Trial160 total patients (2:1 treatment vs control)Double-blind, placebo (sham procedure) controlledCatheter delivery of cells: percutaneousThree month, six month, and 12 month follow-upEndpoints: Improvement in HF symptoms and Ventricular VolumesPrimary endpoint: Improvement in quality of life measure “Kansas CityCardiomypathy Questionaire”Secondary endpoints: Ventricular volumes, Six minute walk testInterim data analysis at six months by Independent Review Board
  • ACT Therapeutics:RPE Program
  • 24RPE Anatomy and FunctionRPE Anatomy and FunctionRPELayerDeterioratesin Patientswith AMDand otherRetinalDegenerativeDiseasesabsorption of stray lightbarriervitamin A metabolism andtransportphagocytosis of shedphotoreceptor segmentssome functions of RPE
  • 25RPE ProgramRPE Program2006 MilestoneAchieved:Published DataShowing hESC-derived RPECells RescueVisual FunctioninRCS Rats
  • 26RPE Rescue in the RCS RatRPE Rescue in the RCS RatRPE: essential for the survival of photoreceptors in the retina;believed to play a critical role in healthy visionRCS retina at P100 withRPE-9 injection:A: low power view of retinashowing extensive rescue;B: high power of outline bshowing rescuedphotoreceptors;C: high power of outline cshowing non-rescue area.PhotoreceptorRescue in RCS Ratby transplantedH9-RPE cells
  • 27RPE Program CollaborationRPE Program Collaboration2007 Milestone Achieved - Preclinical Development Collaborationwith Casey Eye Institute at Oregon Health & Science UniversityDr. Raymond Lund Leading Researcher in RetinalDegenerative DiseaseDr. Peter Francis Senior Researcher and ClinicianDr. Richard Weleber Senior ClinicianOHSU Team is Conducting Dosage Studies in the RCS RatOHSU Team is Advising on RPE Program Safety StudiesAdditionally, OHSU Team is Advising on Plans for IND andPhase I Human Clinical Trials
  • 28RPE Program - GLP Safety StudiesRPE Program - GLP Safety Studies2007 Milestone Achieved – Hired MPI Research to conduct our GLPSafety Studies for the RPE ProgramInitial Pilot Studies Commenced initial studies todemonstrate cell survival and validationof controlsPivotal Studies Full safety and tumorigenicity studies todemonstrate safety of cells in animalmodelsFDA Dialogue Company has initiated discussions withFDA and is planning for furtherdiscussions in 2007 in regard to INDsubmission
  • ACT Therapeutics:Hemangioblast Program
  • 30The Hemangioblast CellThe Hemangioblast CellPre-cursor to all cell types of the blood and thevascular system
  • 31Hemangioblast ProgramHemangioblast Program2006 Milestone Achieved: Hemangioblast CellsGenerated from Blastomere Derived hESC Lines40X 10X
  • 32Hemangioblast ProgramHemangioblast Program2007 MilestoneAchieved:Published DataShowingHemangioblastFunction In Vivo inNature Methods
  • 33Hemangioblast ProgramHemangioblast ProgramRepair of Ischemic Retinal VasculaturesMouse Rat
  • 34Hemangioblast ProgramHemangioblast ProgramRestoration of Blood Flow in Ischemic Limbs (Rat)
  • 35Hemangioblast ProgramHemangioblast ProgramIncrease in Survival Rate in Infacted Heart (Mouse)
  • 36Hemangioblast ProgramHemangioblast ProgramSignificant Breakthrough in ScaledManufacture of Hemangioblasts CellsWell characterized - make all cellsof the blood and vascular tissuesSignificant Collaborations underwayto generate proof of concept data in awide range of indicationsPlan to generate Hemangioblasts underGMP in 2007 and commence additionalpreclinical studies for target indications
  • ACT Therapeutics:Dermal Program
  • 38Skin: Dermal Cells for Wound and Burn RepairSkin: Dermal Cells for Wound and Burn RepairES dermal cells possess uniquehealing capacities of early skin cellsto heal wounds without scarring.ACTC is researching ES dermal celltechnology that may someday repairskin.Large application for therapies to repairdamage from burns, wounds and surgicalprocedures, quickly and without scarringCells successfully isolated in our California labs
  • ACT Technology:Blastomere Program
  • 40Blastomere Program–Most RecognizedBlastomere Program–Most Recognized
  • 41Success in HumanSuccess in Human2006 MilestoneAchieved:Derivation ofHuman hESCLines Publishedin Nature
  • 42First Proven Alternative MethodFirst Proven Alternative MethodEnables Derivation of New hESC Lines viaPreimplantation Genetic Diagnosis (PGD)Method, Preserving Development Potential ofthe EmbryoFirst Proven Alternative; Presents Solution tohESC ControversyBlastomere hESC Lines May Be Eligible forFederal Funding; Company Seeking RulingHemangioblastsHemangioblastsDifferentiated fromDifferentiated fromBlastomereBlastomere hESChESC LinesLines
  • ACT Technology:ACTCellerate Program
  • 44ACTCellerate – A Sleeping BeautyACTCellerate – A Sleeping Beauty2006 Milestone Achieved:Announced proprietary high throughputtechnology platform for discovering largenumber of therapeutically useful, hESC-derivedcell types
  • 45ACTCellerate ProgramACTCellerate ProgramCellSortingCellSortingGene ExpressionProfileScaling in RollershES Cell LineACTCellerate Cell LinesLinesHuman Embryo-Derived Cells
  • 46ACTCellerate ProgramACTCellerate ProgramACT162Cardiac ProgenitorCardiacMuscleACTCMYH7MYL4MYH3TNNT2NeuronalNEF3NEFLMEIS1CDH2SILVACTCellerate Cell LinesLines
  • ACT Technology:GMP Manufacturing Program
  • 48CENTRIFCT03002MicroscopeBSCSH-01002BSCSH-01001Viewing WindowSterilizerOV-05001CENTRIFCT-03001UnclassifiedPassThruWarmingOvenIN-02001-80FreezerCS-040022-8˚CRefrigeratorCS-01001Tri-GasIncubatorIN-03001CO2INCUBATORIN-01004 (top)IN-01003(bottom)CO2INCUBATORIN-01001 (top)IN-01002 (bottom)MicroscopewithComputerWorkStationSH-03001BSCSH-01003FPRM198Revision513June06Class 10.000Room 198Class 100.000Room 199GMP Manufacturing ProgramGMP Manufacturing Program2006 Milestone Achieved - Company Opens GMPFacilities in Massachusetts and CaliforniaADVANCED CELL TECHNOLOGYGMP MANUFACTURINGLABORATORY Rms 198/199
  • 49GMP Manufacturing ProgramGMP Manufacturing Program2007 Milestone Achieved – Company Hires Dr. PedroHuertas as Chief Development OfficerNoted Biotech Industry Executive – Experience atGenzyme, StemCells Inc., Novazyme, AmicusTherapeuticsHarvard – MIT Trained PhysicianM.D. from Harvard and MITPh.D. from Harvard University in Cell andDevelopmental BiologyM.B.A. from MIT’s Sloan School of MgmtTasked with leading company’s push to the clinic
  • 50GMP Manufacturing ProgramGMP Manufacturing ProgramDesign of Master and Working hESC BanksUnlimited Expansion ofStem Cell LineMasterCell BankWorkingCell BankBank of Differentiated CellsBank ofDifferentiated CellsBank of Differentiated CellsSingle Blastomere
  • 51GMP Manufacturing ProgramGMP Manufacturing Program2007 Milestone Achieved – Company Manufacturing RPECells under GMP Compliant ConditionsWorking Stem Cell Bank Cryopreserved RPE Cell BankRetinalPigmentedEpithelium
  • ACT :Corporate Milestones
  • 532007 – 2008 Corporate Milestones2007 – 2008 Corporate MilestonesRaise Additional CapitalAchieve Listing on National ExchangeCommence Phase II Clinical Trial for Myoblast ProgramFile INDs for RPE and Hemangioblast ProgramsAnnounce Significant Corporate PartnershipAnnounce Scientific Breakthrough on SolvingHistocompatibility
  • 54ACTC Value PropositionACTC Value PropositionSelected Comparable Companies (S mio., except per share data)Osiris OSIR / $14.04 27.4 $384.7 172,590Phase II/III Stem Cell Therapy NASDAQGeron Corporation GERN / $7.14 72.9 $520.5 1,660,000Phase II Cancer / Preclinical hESCs NASDAQStem Cells, Inc. STEM / $2.30 80.4 $184.9 1,215,743Phase I Stem Cell Therapy NASDAQAastrom Biosciences, Inc. ASTM / $1.35 120.0 $162.0 1,100,000Phase II/III Stem Cell Therapy NASDAQViaCell, Inc. VIAC / $5.40 38.8 $209.5 151,298Cord Blood Storage / Preclinical NASDAQAdvanced Cell Technology ACTC / $0.47 49.5 $23.3 1,006,000Phase II Stem Cell Therapy / Preclinical hESCs OTCBB* Does not include outstanding options, warrants and other common stock equivalents.VolumeAverageDailyMarketCapitalizationPrimarySharesOutstanding*Name / Description / Phase Tkr / ExchCurrentStock Price(Jun 28th)
  • Thank you for your timeFor more information, visit www.advancedcell.comAdvanced Cell Technology is traded on the OTC BB, symbol: ACTC