BIO-Europe, Barcelona, Spain, March 12, 2013
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3/12/2013

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BIO-Europe, Barcelona, Spain, March 12, 2013 Presentation Transcript

  • 1. LEADINGREGENERATIVEMEDICINE
  • 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. RPE Clinical Program
  • 4. There is a tremendousUNMET MEDICAL NEEDfor treatments of dry AMD& other forms of macular degeneration
  • 5. RPE Program - Investment Thesis5Dry AMD: More than 50 million patients in major markets.1% market penetrationmay represent $5-10B market opportunity.Orphan indications: 10% market penetration of SMD alone may be a $100+million/year product. Orphan status provides options for early authorization.Immense UnmetMedical NeedsSmall Doses &Globally Scalable ColdChainImmune PrivilegedInjection Site
  • 6. 6Structure of RetinaThe Retina the light-sensitivetissue lining the inner surface ofthe eyeRetina
  • 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. 8Life Support to PhotoreceptorsFailure of RPE cellsresults in manydegenerative diseasesAge-related macular degeneration (AMD)Myopic Macular DystrophyStargardt’s disease
  • 9. RPE cell therapy may impactover 200 retinal diseases9RPE Therapy- Rationale• Massive unmet medical need• 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• Unique measuring and observation environment
  • 10. Preclinical Models10Injected human RPE cellsrepair monolayerstructure in eyeTransplanted cellsengraft and formcorrect anatomicalstructureMouse model for macular degeneration
  • 11. Preclinical Models11untreated treatedPhotoreceptorlayerphotoreceptorlayer is lostTransplanted RPE cellsprotect photoreceptors andprevent loss of visionRat model formacular degeneration•Untreated animals go blind•Treated animals maintain70-80% of normal vision
  • 12. Phase I - Clinical Trial Design12SMD 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 Cells100K CellsFDA Approved “Cohort 2a”Inclusion Criteria: vision 20/100+
  • 13. 13Participation by the leadingretinal surgeons in the worldJules Stein(UCLA)MassEye & EarInfirmaryWills EyeInstituteBascomPalmer EyeInstituteMoorfieldsEyeHospitalEdinburghRoyalInfirmary
  • 14. Surgical Overview14Procedure:• 25 Gauge Pars Plana Vitrectomy• Posterior Vitreous Separation (PVDInduction)• Subretinal hESC-derived RPE cellsinjection• Bleb Confirmation
  • 15. Preliminary Results15No 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.
  • 16. 16Engraftment and Survival: SD-OCT image collected at month 3show survival and engraftment of RPESMD0013mo post-opPreliminary Results – Structural
  • 17. BaselineInjection siteMonth 1 Month 2FUNDUS PHOTOGRAPH17
  • 18. Current Safety Profile1812 SMD Patients Treated6 patients (50K cells cohort) treated – US&UK Trials > Cohort Complete6 patient (100K cells cohort) treated – US&UK Trials > Cohort Complete6 dry AMD Patients Treated3 patients (50K cells cohort) treated > Cohort Complete3 patient (100K cells cohort) treated > Cohort CompleteNo reports of any adverse events or complications due to cells• No evidence of inflammation or infiltration• No evidence of ectopic tissue formation• No evidence of retinal detachment
  • 19. RPE Program Milestone Objectives19Key upcoming milestones• Continue to treat and review patient dataFDA has recently approved vision as good as20/100 in treated eye for patient inclusion criteria• Treat earlier stage disease to determine curativepower of dissociated cell injections• Define efficacy endpoints and targeted patient visualcriteria• Simplify shipping and cell-prep to enhance scaleddistribution platform
  • 20. Expanding Clinical Programs20Myopia 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
  • 21. 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 improvements21
  • 22. ACTBlood ComponentsProgram22
  • 23. Generation of Blood Products23Hemangioblasts RBCsHemangioblasts EnucleatedRBC’sProcess generates largequantities of functionalred blood cellsandmegakaryocytes &platelets
  • 24. The Case for Platelets24Platelets are key elements ofhemostasis and thrombosis as well astissue regeneration after injury or surgery.• Wound repair and treatment of trauma• Thrombocytopenia• Reconstructive, plastic and joint replacement surgery• Current supply limits use: expanded use of platelets predicts amarket for several million more units of platelets yearlyPlatelets are the blood product most difficultto maintain – cannot be refrigerated or frozen
  • 25. Clinical Program Status25• Animal model studies showproper in vivo function• Achieved clinical dosescale manufacturing• Completely feeder-free process(bioreactor capable!)• Pre-IND meeting with FDASC-derived plts Incorporate into clot
  • 26. Platelets 2.026Ability to control platelet manufacturing providesopportunities to improve storage, fine tuneplatelets for use in wound healing applications, as wellas to engineer new roles for platelets beyondtraditional involvement in wound healing.• Improve cryopreservation of platelets• Make lyophilization of platelets tractable solution• Utilize platelets for drug delivery• Utilize platelets in imaging (load with contrast agents)
  • 27. Therapeutic Pipeline27Retinal 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
  • 28. ACT Corporate Overview
  • 29. Financial Update – Strong Balance Sheet29• 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
  • 30. 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 Media30
  • 31. 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 professor31World Class Board of DirectorsHighly-regarded Ethics Advisory Board
  • 32. Thank youFor more information, visit www.advancedcell.com