ARM's Regen Med Investor Day, New York City, April 17, 2013


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ARM's Regen Med Investor Day, New York City, April 17, 2013

  1. 1. This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced CellTechnology 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 we filefrom time to time with the Securities and Exchange Commission. The risks identifiedtherein, as well as others not identified by the Company, could cause the Company’sactual results to differ materially from those expressed in any forward-lookingstatements. Ropes GrayCautionary Statement Concerning Forward-Looking Statements1LEADINGREGENERATIVEMEDICINE
  2. 2. 050100150200250Per1000populationArthritis/other musculoskeletalHeart/other circulatory VisionExemplary Chronic Health ConditionsSource: U.S. Census Bureau Publication “65+ in the United States”, P23-209218-4445-6465-7475+AgeGroups
  3. 3. 133.7 142.2 173.4 202.7 207.4 205.537.452.964.881.9105.3 121214.6274.6395.9568.5752.2870.434.453.280.1121198.5305.32010 2020 2030 2040 20502000 2010 2020 2030 2040 2050200080 and over65 to 79Population Aged 65 and Older for Developed andDeveloping Countries(in millions)Developed Countries Developing CountriesSource: U.S. Census Bureau Publication “65+ in the United States”, P23-2093
  4. 4. 4Structure of RetinaThe Retina the light-sensitivetissue lining the inner surface ofthe eyeRetina
  5. 5. 5Life Support to PhotoreceptorsProvides nutrients and growth factors• photoreceptors see no bloodRecycles Vitamin A• maintains photoreceptor excitabilityDetoxifies photoreceptor layerMaintains Bruch’s Membrane• natural antiangiogenic barrier• immune privilege of retinaAbsorbs stray light / protects from UVRPE Layer hasmultiplecritical rolesin thehealth andfunctionof photoreceptors andthe retina as a whole.
  6. 6. 6Life Support to PhotoreceptorsFailure of RPE cellsresults in manydegenerative diseasesStargardt’s diseaseMyopic Macular DystrophyAge-related macular degeneration (AMD)
  7. 7. 7RPE Therapy- Rationale• Massive unmet medical need• Easy to identify – aids manufacturing• Small dosage size – less than 200K cells• Immune-privileged site - minimal immunosuppression• Ease of administration - no separate device approval• Unique measuring and observation environment
  8. 8. Preclinical Models8Injected human RPE cellsrepair monolayerstructure in eyeTransplanted cellsengraft and formcorrect anatomicalstructureMouse model for macular degeneration
  9. 9. Preclinical Models9untreated treatedPhotoreceptorlayerphotoreceptorlayer is lostTransplanted RPE cellsprotect photoreceptors andprevent loss of visionRat model formacular degeneration•Untreated animals go blind•Treated animals maintain70-80% of normal vision
  10. 10. 10Participation by the leadingretinal surgeons in the worldJules Stein(UCLA)MassEye & EarInfirmaryWills EyeInstituteBascomPalmer EyeInstituteMoorfieldsEyeHospitalEdinburghRoyalInfirmary
  11. 11. Surgical Overview11Procedure:• 25 Gauge Pars Plana Vitrectomy• Posterior Vitreous Separation• Subretinal hESC-derived RPE cellsinjection• Bleb Confirmation• Day Surgery/Sedation
  12. 12. Preliminary Results12No Adverse EventsNo signs of hyperproliferation,abnormal growth, rejection or retinaldetachment.Persistence of cellsAnatomical evidence of hESC-RPEsurvival and engraftment.Increased pigmentation within the bedof the transplant.Impact on AcuityRecorded functional visualimprovements in both patients.
  13. 13. Preliminary Results13Persistence of cellsAnatomical evidence of hESC-RPEsurvival and engraftment.Increased pigmentation within the bedof the transplant.Engraftment and Survival: SD-OCT image collected at month 3show survival and engraftment of RPE
  14. 14. Preliminary Results14BaselineInjection siteMonth 1 Month 2Increased pigmentation within the bedof the transplant.Persistence of cells
  15. 15. Preliminary Results15• Varying degrees of improvement in visual acuity acrosspatientsPersistence for 18+ months in first patients• Increased letters on ETDRS Charts• Color perception• Contrast• Low light visionThese are very late stage patients with a high degree ofheterogeneity in degree of “rescueable” photoreceptorsImpact on Visual Acuity
  16. 16. Halfway Point16Based on safety and functional data from first 18 patients,FDA has approved new 4 patient cohorts in each trial.Best Vision Inclusion Criteria for new Cohort 2aincludes patients with vision as good as 20/100.50K Cells 100K Cells 150K Cells 200K Cells100K CellsFDA Approved “Cohort 2a”Inclusion Criteria: vision 20/100+
  17. 17. Current Safety Profile1714 SMD Patients Treated6 patients (50K cells cohort) treated – US&UK Trials > Cohort Complete6 patient (100K cells cohort) treated – US&UK Trials > Cohort Complete1 patient (150K cells cohort) treated – US1 patient (“Cohort 2a”) treated – US7 dry AMD Patients Treated3 patients (50K cells cohort) treated > Cohort Complete3 patient (100K cells cohort) treated > Cohort Complete1 patient (150K cells cohort) treated – US
  18. 18. RPE Program Milestone Objectives18Key upcoming milestones• Continue to treat and review patient data• Treat earlier stage disease to determinecurative power of dissociated cell injections• Define efficacy endpoints and targetedpatient visual criteria• Simplify shipping and cell-prep to enhancescaled distribution platform
  19. 19. Expanding Clinical Programs19Myopia creates a higher risk of permanent vision loss dueto Myopic Macular Degeneration (MMD)• Severe near-sightedness causes elongation of the eyeball --which can cause fissures in RPE layer.January 2013 - FDA ApprovedMMD Phase I/II studyJules Stein Eye Institute (UCLA) and ACT
  20. 20. Intellectual Property – RPE ProgramDominant Patent Position for Treating Retinal DegenerationBroad Coverage for Manufacturing RPE CellsBroad protection of pharmaceutical preparations• RPE cell suspensions• scaffolded RPE layers.RPE Cells derived from other pluripotent stem cellsVigilant filing on improvements20
  21. 21. Therapeutic Pipeline21Retinal Neural Progenitor cells& Isolated Protective FactorsPhotoreceptor Loss, Modulation of Müller CellsProtection of Retinal Ganglion cells (Glaucoma)Corneal EndotheliumCorneal DiseaseHemangioblast cellsIschemic retinopathy– diabetic retinopathy, vascular occlusionsMesenchymal Stromal CellsOcular - Glaucoma, Uveitis, Retinitis PigmentosaAutoimmune DiseasesInflammatory Diseases or disorders
  22. 22. Generation of Blood Products22Hemangioblasts RBCsHemangioblasts EnucleatedRBC’sProcess generates largequantities of functionalred blood cellsandmegakaryocytes &platelets
  23. 23. ACT Corporate Overview
  24. 24. Financial Update – Strong Balance Sheet24• Company ended 2012 Q4 with $40 million incash or availability of cash through financingcommitments• $16 million annual cash-burn rate(funded through early 2015)• Settled nearly all litigation hangover fromprevious management
  25. 25. ACT Management TeamHighly Experienced and Tightly Integrated Management TeamGary Rabin – Chairman & CEODr. Robert Lanza, M.D. – Chief Scientific OfficerEdmund Mickunas – Vice President of Regulatory AffairsDr. Irina Klimanskaya, Ph.D. – Director of Stem Cell BiologyDr. Shi-Jiang (John) Lu, Ph.D. – Senior Director of ResearchDr. Roger Gay, Ph.D. - Senior Director of ManufacturingKathy Singh - ControllerRita Parker – Director of OperationsDr. Matthew Vincent, Ph.D. – Director of Business DevelopmentBill Douglass – Dir. of Corporate Communications & Social Media25
  26. 26. Dr. Ronald M. Green: ChairmanDr. Judith BernsteinDr. Jeremy B.A. GreenDr. Robert KauffmanDr. Carol A. TauerACT LeadershipGary Rabin: Chairman & CEODr. Robert S. Langer, ScD: Prolific medical inventor; Chair – ACT SABGregory S. Perry: EVP – ImmunogenMichael Heffernan: CEO – Collegium PharmaZohar Loshitzer: CEO Presbia; Founder LifeAlert MedicalDr. Alan C. Shapiro: Renowned business school professor26World Class Board of DirectorsHighly-regarded Ethics Advisory Board
  27. 27. Thank youFor more information, visit