This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced Cell Technology Inc”, or “the Company”) s...
David versus Goliathis the story of everyemerging regenerativemedicine company inthis room….
3Structure of RetinaThe Retina the light-sensitivetissue lining the inner surface ofthe eyeRetina
4Life Support to PhotoreceptorsProvides nutrients and growth factors• photoreceptors see no bloodRecycles Vitamin A• maint...
5Life Support to PhotoreceptorsFailure of RPE cellsresults in manydegenerative diseasesStargardt’s diseaseMyopic Macular D...
6RPE Therapy- Rationale• Massive unmet medical need
7RPE Therapy- Rationale• Massive unmet medical need
8RPE Therapy- Rationale• Massive unmet medical need• Small dosage size– less than 200K cells• Immune-privileged site– mini...
Preclinical Models9Injected human RPE cellsrepair monolayerstructure in eyeTransplanted cellsengraft and formcorrect anato...
Preclinical Models10untreated treatedPhotoreceptorlayerphotoreceptorlayer is lostTransplanted RPE cellsprotect photorecept...
GMP Process11Harvest forCryopreservationES CellsEBFormation RPE isolation P1 P20 5 weeks 12 weeks 15 weeks 18 weeksTEST CA...
GMP Process12Extensive Safety Studies ShowsLack of Tumorigenicity
GMP Process13Normal female (46 XX) karyotypeof the clinical RPE lot.Regular Marker and KaryotypeConfirmation
GMP Process14Cell potency of each lot is assessed by phagocytosis(critical function in vivo) of fluorogenic bioparticles.F...
GMP Process15y = 0.0141x + 0.00070.000.501.001.502.000 20 40 60 80 100120Absorbanceat475nmµg/mL MelaninPigmentation matter...
GMP process for differentiation and purification of RPE– Virtually unlimited supply from stem cell source– Optimized for m...
Phase I - Clinical Trial Design17SMD and dry AMD Trials approved in U.S., SMD Trial approved in U.K.12 Patients / trialasc...
18Participation by the leadingretinal surgeons in the worldJules Stein(UCLA)MassEye & EarInfirmaryWills EyeInstituteBascom...
Surgical Overview19Procedure:• 25 Gauge Pars Plana Vitrectomy• Posterior Vitreous Separation• Subretinal hESC-derived RPE ...
Preliminary Results20No Adverse EventsNo signs of hyperproliferation,abnormal growth, rejection or retinaldetachment.Persi...
Preliminary Results21Persistence of cellsAnatomical evidence of hESC-RPEsurvival and engraftment.Increased pigmentation wi...
Preliminary Results22BaselineInjection siteMonth 1 Month 2Increased pigmentation within the bedof the transplant.Persisten...
Preliminary Results23Recorded functional visualimprovements in both patients.• SMD Patient: Best corrected visualacuity im...
Preliminary Results24Varying degrees of improvement in visual acuityacross patients Some patients have pronounced gains i...
Halfway Point25Based on safety and functional data from first 18 patients,FDA and MHRA have approved new 4 patient cohorts...
Current Safety Profile2615 SMD Patients Treated6 patients (50K cells cohort) treated – US&UK Trials > Cohort Complete6 pat...
Phase II/III Projected Timeline27Completion of Phase I : 2013-2014Design of Phase II/III studies is an ongoing process, bu...
Expanding Clinical Programs28Myopia creates a higher risk of permanent vision loss dueto Myopic Macular Degeneration (MMD)...
Price Justification29Unmet Therapeutic NeedEfficacyPatient PrevalencePharmacoeconomicsPatient AdvocacyPricing Justificatio...
Price Justification30We understand that reimbursement challenges areincreasingly becoming a central focus for new treatmen...
Reimbursement Strategy31Conducting a reimbursement analysis and formulating a strategy.Working to secure meetings with CMS...
Implementation32Activity ObjectivesDocument UnmetNeeds and ClinicalValueEngaging our KOLs to help us to develop robust arg...
Implementation33Activity ObjectivesContinue discussionswith providersWe will identify and address hurdles that may remain ...
Implementation34Activity ObjectivesSupport approval andreimbursementProvide on-line, phone and in the field support for pa...
Intellectual Property – RPE ProgramDominant Patent Position for Treating Retinal Degeneration• Broad Coverage for Manufact...
RPE Program - Investment Thesis36Dry AMD: More than 50 million patients in major markets.1% market penetrationmay represen...
ACT Corporate Overview
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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Act corporate-presentation---may-2013---wscrm---print-version

  1. 1. This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced Cell Technology Inc”, or “the Company”) salient businesscharacteristics.The information herein contains “forward-looking statements” as defined under the federal securities laws. Actual results could vary materially.Factors that could cause actual results to 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 from time to time with the Securities and ExchangeCommission. The risks identified therein, as well as others not identified by the Company, could cause the Company’s actual results to differ materiallyfrom those expressed in any forward-looking statements. Ropes GrayCautionary Statement Concerning Forward-Looking Statements1LEADINGREGENERATIVEMEDICINEMay 2013
  2. 2. David versus Goliathis the story of everyemerging regenerativemedicine company inthis room….
  3. 3. 3Structure of RetinaThe Retina the light-sensitivetissue lining the inner surface ofthe eyeRetina
  4. 4. 4Life 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.
  5. 5. 5Life Support to PhotoreceptorsFailure of RPE cellsresults in manydegenerative diseasesStargardt’s diseaseMyopic Macular DystrophyAge-related macular degeneration (AMD)
  6. 6. 6RPE Therapy- Rationale• Massive unmet medical need
  7. 7. 7RPE Therapy- Rationale• Massive unmet medical need
  8. 8. 8RPE Therapy- Rationale• Massive unmet medical need• Small dosage size– less than 200K cells• Immune-privileged site– minimal immunosuppression• Ease of administration– no separate device approval• Unique measuring and observation environment
  9. 9. Preclinical Models9Injected human RPE cellsrepair monolayerstructure in eyeTransplanted cellsengraft and formcorrect anatomicalstructureELOVL4 Mouse model for macular degeneration
  10. 10. Preclinical Models10untreated treatedPhotoreceptorlayerphotoreceptorlayer is lostTransplanted RPE cellsprotect photoreceptors andprevent loss of visionRCS Rat model formacular degeneration•Untreated animals go blind•Treated animals maintain70-80% of normal vision
  11. 11. GMP Process11Harvest forCryopreservationES CellsEBFormation RPE isolation P1 P20 5 weeks 12 weeks 15 weeks 18 weeksTEST CASE:• Spiked with 10 percent hESC• No hESC’s in harvested cellsLimit of Detection for hESC0.00008%3 weeksEBOutgrowth30 WeeksDifferentiation Media is NotPermissive for hESCs
  12. 12. GMP Process12Extensive Safety Studies ShowsLack of Tumorigenicity
  13. 13. GMP Process13Normal female (46 XX) karyotypeof the clinical RPE lot.Regular Marker and KaryotypeConfirmation
  14. 14. GMP Process14Cell potency of each lot is assessed by phagocytosis(critical function in vivo) of fluorogenic bioparticles.Flow cytometry histogram showingphagocytosis of pHrodo bioparticles4°C 37°CDeveloped Quantitative CellPotency Assays
  15. 15. GMP Process15y = 0.0141x + 0.00070.000.501.001.502.000 20 40 60 80 100120Absorbanceat475nmµg/mL MelaninPigmentation matters - optimizetime to harvest and cryopreserve
  16. 16. 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 Process16Product Cold Chain is Easily Scaled for Global SalesACT Cleanroom Suite
  17. 17. Phase I - Clinical Trial Design17SMD 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 CellsBest Vision Inclusion Criteria – First HalfSMD Trial: Hand Motion only, modified to 20/400Dry AMD Trial: 20/800, modified to 20/400
  18. 18. 18Participation by the leadingretinal surgeons in the worldJules Stein(UCLA)MassEye & EarInfirmaryWills EyeInstituteBascomPalmer EyeInstituteMoorfieldsEyeHospitalEdinburghRoyalInfirmary
  19. 19. Surgical Overview19Procedure:• 25 Gauge Pars Plana Vitrectomy• Posterior Vitreous Separation• Subretinal hESC-derived RPE cellsinjection• Bleb Confirmation• Day Surgery/Sedation only
  20. 20. Preliminary Results20No 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.
  21. 21. Preliminary Results21Persistence 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
  22. 22. Preliminary Results22BaselineInjection siteMonth 1 Month 2Increased pigmentation within the bedof the transplant.Persistence of cells
  23. 23. Preliminary Results23Recorded functional visualimprovements in both patients.• SMD Patient: Best corrected visualacuity improved from handmotions to 20/800 and improvedfrom 0 to 5 letters on the ETDRSvisual acuity chart in the study eye.• Dry AMD Patient: Vision improvedin the patient with dry age-relatedmacular degeneration (21 ETDRSletters to 28).SMDPatient
  24. 24. Preliminary Results24Varying degrees of improvement in visual acuityacross patients Some patients have pronounced gains in VA Observed persistence of cell engraftment andVA gains- 22 months now for initial 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 “rescue-able” photoreceptors
  25. 25. Halfway Point25Based on safety and functional data from first 18 patients,FDA and MHRA have 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 and MHRA Approved “Cohort 2a”Inclusion Criteria: vision 20/100+
  26. 26. Current Safety Profile2615 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 (150K cells cohort) treated – UK1 patient (“Cohort 2a”) treated – US8 dry AMD Patients Treated3 patients (50K cells cohort) treated > Cohort Complete3 patient (100K cells cohort) treated > Cohort Complete1 patient (150K cells cohort) treated – US1 patient (“Cohort 2a”) treated – US
  27. 27. Phase II/III Projected Timeline27Completion of Phase I : 2013-2014Design of Phase II/III studies is an ongoing process, butwill become more concrete during 2H2013• Design of future studies dependent upon informationgathered throughout PI/II study― Efficacy― Multiple Injections― Further evaluation of I/E criteria― Potentially less immunosuppressionPhase II/III study commencement 2014-2015Working with ourexperts/investigators indesign of studies
  28. 28. Expanding Clinical Programs28Myopia 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
  29. 29. Price Justification29Unmet Therapeutic NeedEfficacyPatient PrevalencePharmacoeconomicsPatient AdvocacyPricing Justificationacross all categoriesof consideration…this is what webelieve, now how willwe support this position.
  30. 30. Price Justification30We understand that reimbursement challenges areincreasingly becoming a central focus for new treatments• We are working to integrate reimbursement planning early in ourRPE product life cycle• Addressing reimbursement after market clearance doesn’t work anymore• Design the right studies the first time…consequences are significant• Understand payer requirements early; anticipate changes• Evidence of value is critical; not new…just more pressure• Value vs. alternatives• Value to certain patient subpopulations & stakeholders• Value vs. overall affordability (society, payer, employer & individual)
  31. 31. Reimbursement Strategy31Conducting a reimbursement analysis and formulating a strategy.Working to secure meetings with CMS (Medicare) and privateinsurers.Phase II and III: Planning to gather health economics andcomparative clinical effectiveness data.• Our design of later phases of our trials will include ability to collect andassess pharmaco-economic data to demonstrate the costeffectiveness and clinical effectiveness of our RPE product (relative toexisting treatments, if any approved).Building the case for adequate reimbursementbased on a therapy’s clinical and socialbenefits as part of the clinical trials is crucial.
  32. 32. Implementation32Activity ObjectivesDocument UnmetNeeds and ClinicalValueEngaging our KOLs to help us to develop robust arguments that ourRPE cellular therapeutic approach addresses a critical need and is“good medicine”.Collect data oneconomics oftreatmentAnalyzing in detail events, activities, length of time for treatment,timing and any other associated costs under alternative treatmentscenarios for macular degenerative diseases, particularly dry AMD.Watching progress of clinical studies using antibodies.Prepare stakeholderpresentations andproposalsDocumenting disease characteristics, unmet need, current diseasemanagement practices, and measurements of economic impactInitiate discussions withstakeholdersWill pursue parallel discussions with CMS, key private payers, and keytreatment centers to build awareness and solicit feedbackOur Strategy during clinical trials….
  33. 33. Implementation33Activity ObjectivesContinue discussionswith providersWe will identify and address hurdles that may remain regardinginstitutional review processes and procedures in key treatmentcenters – will use our KOLs as necessary.Engage withFoundation and PatientStakeholdersDevelop programs to refer patients, educate patients and families,and provide reimbursement support, as necessary.Our Prelaunch Strategy….
  34. 34. Implementation34Activity ObjectivesSupport approval andreimbursementProvide on-line, phone and in the field support for patient-by-patientproduct use and insurance coverageOur Strategy Post-Launch….We have some great rolemodels to follow already…
  35. 35. Intellectual Property – RPE ProgramDominant Patent Position for Treating Retinal Degeneration• Broad Coverage for Manufacturing RPE Cells• Broad protection of pharmaceutical preparationsCovers both RPE cell suspensions and scaffolded RPE layers.• RPE Cells derived from other pluripotent stem cells – e.g., iPS cellso Careful Consideration of Literal Scopeo Preservation of Doctrine of Equivalentso Constantly Mining Existing Filingso Vigilantly Filing on Improvements35Keeping ourIP Lawyerson their toes
  36. 36. RPE Program - Investment Thesis36Dry 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
  37. 37. ACT Corporate Overview
  38. 38. 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 Media38
  39. 39. 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 professor39World Class Board of DirectorsHighly-regarded Ethics Advisory Board
  40. 40. Thank youFor more information, visit www.advancedcell.com

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