October 2012LEADINGREGENERATIVEMEDICINE
This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced CellTechnology Inc”, or “the Company”) sa...
ACT is a publicly-traded biotechnology company• Develop cellular therapies for the treatment of diseases andconditions tha...
Multiple Pluripotent Cell PlatformsSingle Blastomere-derivedEmbryonic Stem Cell LinesGenerating hESC linesWITHOUT DESTRUCT...
RPE Clinical Program
6Structure of RetinaThe Retina the light-sensitivetissue lining the inner surface ofthe eyeRetina
7Life Support to PhotoreceptorsProvides nutrients and growth factors• photoreceptors see no bloodRecycles Vitamin A• maint...
8Life Support to PhotoreceptorsLoss of RPE cellsBuild up of toxic wasteLoss of photoreceptorsDry AMDBruch’s Mem. dehiscenc...
9RPE Therapy- RationaleDry AMDrepresents more than90%of all cases of AMDNorth America & Europealone have more than 30 mill...
RPE cell therapy may impactover 200 retinal diseases10RPE Therapy- Rationale• Massive unmet medical need• Unique measuring...
11RPE Therapy- RationaleU.S. PATIENT POPULATIONEARLY STAGE(10-15M)INTERMEDIATE(5-8M)LATE STAGE(1.75M)ACT’S RPE Cell Therap...
GMP process for differentiation and purification of RPE– Virtually unlimited supply from stem cell source– Optimized for m...
Preclinical - Examples13control treatedInjected human RPE cellsrepair monolayer structure ineyePhotoreceptorlayerphotorece...
Phase I - Clinical Trial Design14SMD and dry AMD Trials approved in U.S., SMD Trial approved in U.K.12 Patients / trialasc...
Participating Retinal Surgeons15Steven D. Schwartz, MDChief, Retina DivisionAhmanson Professor of OphthalmologyDirector, D...
Participating Retinal Surgeons16Dean Eliott, MDAssociate Director, Retina ServiceProfessor, Harvard Medical SchoolMassachu...
Surgical Overview17Procedure:• 25 Gauge Pars PlanaVitrectomy• Posterior Vitreous Separation(PVD Induction)• Subretinal hES...
Preliminary Results18No Adverse EventsNo signs of hyperproliferation,abnormal growth, rejection or retinaldetachment.Persi...
19Engraftment and Survival: SD-OCT image collected at month 3show survival and engraftment of RPESMD0013mo post-opPrelimin...
20Preliminary Results – StructuralBaseline Month 1 Month 2Injection siteSD-OCT confirms RPE engraftmentadjacent Bruch’s me...
Preliminary Results – Functional21Visual Acuity Measurements• SMD Patient: BCVA improved from hand motions to 20/800 andim...
Current Safety Profile – Stargardt’s Trial229 SMD Patients Treated (as of 27 October 2012)3 patients (50K cells cohort) tr...
Current Safety Profile – Dry AMD Trial234 dry AMD Patients Treated (as of 27 October 2012)3 patients (50K cells cohort) tr...
RPE Program Milestone Objectives24Key upcoming milestones• Continue to treat and review patient data• Define efficacy endp...
Intellectual Property – RPE ProgramTreatment Dominant Patent Position for Treating RetinalDegenerationManufacturing Broad ...
Price Justification26Unmet Therapeutic NeedEfficacyPatient PrevalencePharmacoeconomicsPatient AdvocacyPricing Justificatio...
RPE Program - Investment Thesis27• Immense unmet medical need• Small Doses• Immunoprivileged – permits central (allogeneic...
Therapeutic Pipeline -Ocular Programs
Retinal Pigment Epithelial Cells Macular Degeneration - dry AMD Retinitis Pigmentosa Photoreceptor protectionHemangiobl...
Mesenchymal Stem CellProgram30
Mesenchymal Stem Cells in Therapy31Mesenchymal stem cells (MSCs) regulate immune responsesprovide therapeutic potential fo...
hESC- and iPS – derived MSC32ACT Proprietary Process• hESC- and iPS-derived MSCs• virtually inexhaustible source of starti...
hESC- and iPS – derived MSC33Substantially decreases and reverses diseaseconditions in autoimmune models.• Far more potent...
ACT Vascular Program
Hemangioblast Program: Overview35Hemangioblast: common precursor to hematopoietic and endothelial cells.Multipotent cell p...
Generation of Blood Products36Hemangioblasts RBCsHemangioblasts EnucleatedRBC’sProcess generates largequantities of functi...
ACT Corporate Overview
Financial Update – Strong Balance Sheet38• Company ended 2012 Q3 with $42 million in cash oravailability of cash through f...
ACT Management TeamHighly Experienced and Tightly Integrated Management TeamGary Rabin – Chairman & CEODr. Robert Lanza, M...
Dr. Ronald M. Green: ChairmanDr. Judith BernsteinDr. Jeremy B.A. GreenDr. Robert KauffmanDr. Carol A. TauerACT LeadershipG...
Thank youFor more information, visit www.advancedcell.com
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Stem Cell Meeting on the Mesa, Oct. 29, 2012

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10/29/2012

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Stem Cell Meeting on the Mesa, Oct. 29, 2012

  1. 1. October 2012LEADINGREGENERATIVEMEDICINE
  2. 2. 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 the federalsecurities laws. Actual results could vary materially. Factors that could cause actual resultsto 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 fromtime to time with the Securities and Exchange Commission. The risks identified therein, aswell as others not identified by the Company, could cause the Company’s actual results todiffer materially from those expressed in any forward-looking statements. Ropes GrayCautionary Statement Concerning Forward-Looking Statements2
  3. 3. ACT is a publicly-traded biotechnology company• Develop cellular therapies for the treatment of diseases andconditions that impact hundreds of millions of people worldwide• Cell therapies are traditionally derided for being high cost, high touchand having lack of scalability. Not our approach at all• Premier scientific and research development teamAbout ACT:– Ticker – ACTC– Market Capitalization: $170 million– Principal lab and GMP facility: Marlborough, MA– Corporate HQ: Santa Monica, CA– Only hESC-derived tissue trials ongoing worldwide– Nearly completed corporate turnaround from disastrous financial and legaldecisions3Company Overview
  4. 4. Multiple Pluripotent Cell PlatformsSingle Blastomere-derivedEmbryonic Stem Cell LinesGenerating hESC linesWITHOUT DESTRUCTION OF EMBRYOUtilizes aSINGLE CELL BIOPSYInduced Pluripotency Stem Cells (iPS)• Early Innovator in Pluripotency (before iPS was even a term!)• Controlling Filings (earliest priority date) to use of OCT4 for inducing pluripotency4Final Product Definition: hESC-derivedproducts will be manufactured using a cellline made in 2005 from single cell isolatedwithout the destruction of any embryos
  5. 5. RPE Clinical Program
  6. 6. 6Structure of RetinaThe Retina the light-sensitivetissue lining the inner surface ofthe eyeRetina
  7. 7. 7Life 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.
  8. 8. 8Life Support to PhotoreceptorsLoss of RPE cellsBuild up of toxic wasteLoss of photoreceptorsDry AMDBruch’s Mem. dehiscenceChoroidal neovascularizationWet AMD
  9. 9. 9RPE Therapy- RationaleDry AMDrepresents more than90%of all cases of AMDNorth America & Europealone have more than 30 million dry AMD patientswho should be eligible for our RPE cell therapyWhat if we couldreplacemissing RPE cells?
  10. 10. RPE cell therapy may impactover 200 retinal diseases10RPE Therapy- Rationale• Massive unmet medical need• Unique measuring and observation environment• Easy to identify – aids manufacturing• Small dosage size – less than 200K cells• Immune-privileged site - minimal/no immunosuppression• Ease of administration - no separate device approval
  11. 11. 11RPE Therapy- RationaleU.S. PATIENT POPULATIONEARLY STAGE(10-15M)INTERMEDIATE(5-8M)LATE STAGE(1.75M)ACT’S RPE Cell Therapyshould address the full rangeof dry AMD patients:Halt progression of vision loss in earlystage patientsRestore some visual acuity in laterstage patients
  12. 12. GMP process for differentiation and purification of RPE– Virtually unlimited supply from stem cell source– Optimized for manufacturingIdeal Cell Therapy Product– Centralized Manufacturing– Small Doses– Easily Frozen and Shipped– Simple Handling by DoctorGMP Manufacturing12Product Cold Chain is Easily Scaled for Global Sales
  13. 13. Preclinical - Examples13control treatedInjected human RPE cellsrepair monolayer structure ineyePhotoreceptorlayerphotoreceptor layeris only 0 to 1 cellthick withouttreatment
  14. 14. Phase I - Clinical Trial Design14SMD and dry AMD Trials approved in U.S., SMD Trial approved in U.K.12 Patients / trialascending dosages of 50K, 100K, 150K and 200K cells.Regular Monitoring - including high definition imaging of retina50K Cells 100K Cells 150K Cells 200K CellsPatient 1 Patients 2/3DSMB Review DSMB Review
  15. 15. Participating Retinal Surgeons15Steven D. Schwartz, MDChief, Retina DivisionAhmanson Professor of OphthalmologyDirector, Diabetic Eye Disease and Retinal Vascular CenterDirector, Ophthalmic Photography Clinical LaboratoryJules Stein Eye InstituteWills Eye InstituteCarl D. Regillo, MDDirector, Retina ServiceProfessor of Ophthalmology, Jefferson Medical CollegeByron L. Lam, MDDirector, Clinical Visual PhysiologyProfessor of OphthalmologyBascom Palmer Eye Institute
  16. 16. Participating Retinal Surgeons16Dean Eliott, MDAssociate Director, Retina ServiceProfessor, Harvard Medical SchoolMassachusetts Eye and Ear InfirmaryMoorfields Eye HospitalJames Bainbridge, MA MB BChir PhD FRCOphthProfessor of Retinal Studies, UCL Institute of OphthalmologyPhilip J. Rosenfeld, MD PhDProfessor of OphthalmologyBascom Palmer Eye InstituteEdinburgh Royal InfirmaryBaljean Dhillon BMed Sci, BM BS, FRCSSurgeon, Edinburgh Royal InfirmarySurgeon, Lothian University Hospitals NHS TrustProfessor of Ophthalmology, University of EdinburghProfessor of Visual Impairment Studies, Heriot Watt University
  17. 17. Surgical Overview17Procedure:• 25 Gauge Pars PlanaVitrectomy• Posterior Vitreous Separation(PVD Induction)• Subretinal hESC-derived RPEcells injection• Bleb Confirmation• Air Fluid Exchange
  18. 18. Preliminary Results18No 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.
  19. 19. 19Engraftment and Survival: SD-OCT image collected at month 3show survival and engraftment of RPESMD0013mo post-opPreliminary Results – Structural
  20. 20. 20Preliminary Results – StructuralBaseline Month 1 Month 2Injection siteSD-OCT confirms RPE engraftmentadjacent Bruch’s membrane.
  21. 21. Preliminary Results – Functional21Visual Acuity Measurements• SMD Patient: BCVA improved from hand motions to 20/800 andimproved from 0 to 5 letters on the ETDRS visual acuity chart• Dry AMD Patient: Vision improved in the patient with dry age-related macular degeneration (21 ETDRS letters to 28)14 month Follow-up:• Visual acuity gains remain relatively stable for both patients• SMD Patient continues to show improvement.U.K. SMD01 Patient (at 9 month follow-up)• ETDRS: Improved from 5 letters to 10 letters, and stable• Subjective: Reports significantly improved ability to read text on TV
  22. 22. Current Safety Profile – Stargardt’s Trial229 SMD Patients Treated (as of 27 October 2012)3 patients (50K cells cohort) treated at UCLA – US Trial3 patients (50K cells cohort) treated at Moorfields Eye – UK Trial2 patient (100K cells cohort) treated at Wills Eye – US Trial1 patient (100K cells cohort) treated at Moorfields Eye – UK TrialNo reports of any adverse events or complications due tocells per se• No evidence of inflammation or infiltration• No evidence of ectopic tissue formation• No evidence of retinal detachment
  23. 23. Current Safety Profile – Dry AMD Trial234 dry AMD Patients Treated (as of 27 October 2012)3 patients (50K cells cohort) treated at UCLA – US Trial1 patient (100K cells cohort) treated at Wills Eye – US TrialNo reports of any adverse events or complications due tocells per se• No evidence of inflammation or infiltration• No evidence of ectopic tissue formation• No evidence of retinal detachment
  24. 24. RPE Program Milestone Objectives24Key upcoming milestones• Continue to treat and review patient data• Define efficacy endpoints and targeted patient visualcriteria• Treat earlier stage disease to determine curativepower of dissociated cell injections• Simplify shipping and cell-prep to enhance scaleddistribution platform
  25. 25. Intellectual Property – RPE ProgramTreatment Dominant Patent Position for Treating RetinalDegenerationManufacturing Broad Coverage for Manufacturing RPE Cells from hESCPreparations Claims directed to pharmaceutical preparations of RPECells from hESC, including both cell suspensions andscaffolded RPE layers.Sources Issued patents cover RPE Cells derived from otherpluripotent stem cells (including iPS cells)Vigilance Regularly Filing on Improvements• Extend patent life cycle, with significance to commercialization• Include composition-of-matter claims (cell preparations,pharmaceutical preparations, etc.)25
  26. 26. Price Justification26Unmet Therapeutic NeedEfficacyPatient PrevalencePharmacoeconomicsPatient AdvocacyPricing Justificationacross all categoriesof consideration
  27. 27. RPE Program - Investment Thesis27• Immense unmet medical need• Small Doses• Immunoprivileged – permits central (allogeneic) source of cells• Noninvasive monitoring of retinaMarket potential: More than 50 million patients in major markets.1% market penetration may represent $5-10B market opportunity.Orphan indications are meaningful: Estimating a 10% market penetrationwith reoccurring treatments every 3-5 years, Stargardt’s disease can be a$100+ million/year product.
  28. 28. Therapeutic Pipeline -Ocular Programs
  29. 29. Retinal Pigment Epithelial Cells Macular Degeneration - dry AMD Retinitis Pigmentosa Photoreceptor protectionHemangioblast cells Ischemic retinopathy– diabetic retinopathy, vascular occlusionsRetinal Neural Progenitor cellsIsolated Protective Factors Photoreceptor Loss, Modulation of Müller Cells Protection of Retinal Ganglion cells (Glaucoma)Corneal Endothelium, Corneal Epithelium,Descemet’s Membrane Corneal DiseaseMesenchymal Stromal Cells Glaucoma, Uveitis Retinitis Pigmentosa Management of Ocular Surfaces29Retina
  30. 30. Mesenchymal Stem CellProgram30
  31. 31. Mesenchymal Stem Cells in Therapy31Mesenchymal stem cells (MSCs) regulate immune responsesprovide therapeutic potential for treating autoimmune orinflammatory diseases.• Allogeneic - without HLA matching.• Track Record - Adult-derived MSCs already in 200+ clinical trials.Derived from Bone Marrow and Adipost TissueHowever, allogeneic adult-derived MSC’s are limitedby replicative senescence, and as it turns out, a relativelack of potency
  32. 32. hESC- and iPS – derived MSC32ACT Proprietary Process• hESC- and iPS-derived MSCs• virtually inexhaustible source of starting material• replicative capacity is more than 33,000 times greaterAdvantages for Manufacturing• Use Single Master Cell Bank• Simplifies FDA/regulatory process• No need for continually finding and qualifyingdonors• Less labor-intensive
  33. 33. hESC- and iPS – derived MSC33Substantially decreases and reverses diseaseconditions in autoimmune models.• Far more potent than adult (BM) derived MSCs.• Have longer duration of action compared to adult(BM) derived MSCs.
  34. 34. ACT Vascular Program
  35. 35. Hemangioblast Program: Overview35Hemangioblast: common precursor to hematopoietic and endothelial cells.Multipotent cell produces allcell types in the circulatory andvascular systems
  36. 36. Generation of Blood Products36Hemangioblasts RBCsHemangioblasts EnucleatedRBC’sProcess generates largequantities of functionalred blood cellsandmegakaryocytes &platelets
  37. 37. ACT Corporate Overview
  38. 38. Financial Update – Strong Balance Sheet38• Company ended 2012 Q3 with $42 million in cash oravailability of cash through financing commitments• $16 million annual cash-burn rate(funded through early 2015)• Settled nearly all litigation hangover from previousmanagement• Pending reverse split and NASDAQ listing in comingmonths
  39. 39. 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 Media39
  40. 40. 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 professor40World Class Board of DirectorsHighly-regarded Ethics Advisory Board
  41. 41. Thank youFor more information, visit www.advancedcell.com

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