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Presentation at Stem Cells USA & Regenerative medicine Congress 2011 in Boston, Sept. 2011
 

Presentation at Stem Cells USA & Regenerative medicine Congress 2011 in Boston, Sept. 2011

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9/15/2011

9/15/2011

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    Presentation at Stem Cells USA & Regenerative medicine Congress 2011 in Boston, Sept. 2011 Presentation at Stem Cells USA & Regenerative medicine Congress 2011 in Boston, Sept. 2011 Presentation Transcript

    • Leading Regenerative MedicineSeptember 2011
    • 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
    • At the Forefront of Regenerative Medicine• Patented Technology for Producing human Embryonic Stem Cells (“hESCs”)without Harm to Embryo• Working with Roslin Cells to create GMP-compliant hESC bank• 2 Human Clinical Trials utilizing hESC-derived Retinal Pigment Epithelial Cells• First Patients Treated on July 12, 2011• Stargardt’s Disease, aka Stargardt’s Macular Dystrophy (SMD)• Dry AMD – (Dry Age-Related Macular Degeneration)• Expecting Preliminary Safety and Engraftment Data by Year-End• Commencing European Trials – estimated first half 2012• Front-of-the-eye programs: Generating hESC-derived corneal tissues• Finalizing preclinical work for blood product IND from hemangioblast program• Generation of off-the-shelf hESC-derived mesenchymal stem cells (MSC) products• Myoblast program for heart failure approved for Phase II3
    • ACT Ocular Programs
    • The RPE layer is critical to the function and health of photoreceptors and theretina as a whole.– RPE cells secrete trophic factors and impact on the chemical environment of thesubretinal space.recycle photopigmentsdeliver, metabolize and store vitamin Atransport iron and small molecules between retina and choroidmaintain Bruch’s membrane– RPE loss may lead to photoreceptor loss and eventually blindness, such as dry-AMD– Loss of RPE layer and Bruch’s membrane may be part of mechanism underlyingprogression from dry-AMD to wet-AMD• Discrete differentiated cell population as target• Failure of target cells results in disease progression5Retinal Pigment Epithelial Cells - RationaleRPE cell as Target
    • • Pigmented RPE cells are easy to identify (no needfor further staining) – impacts manufacturing• Small dosage vs. other therapies• The eye is generally immune-privileged site, thusminimal immunosuppression required, which may betopical.• Ease of administration– Doesn’t require separate approval by the FDA (universal applicator)– Procedure is already used by eye surgeons; no new skill set required for doctorsRPE cell therapy may impact over200 retinal diseases6Retinal Pigment Epithelial Cells - Rationale
    • • Established GMP-compliant process for the Reproducible Differentiationand Purification of RPE cells.– Virtually unlimited supply of cells– Can be derived under GMP conditions pathogen-free– Can be produced with minimal batch-to-batch variation– Can be thoroughly characterized to ensure optimal performance– Molecular characterization studies reveal similar expression of RPE-specific genes to controlsand demonstrates the full transition from the hESC state.GMP ManufacturingIdeal Cell Therapy Product• Centralized Manufacturing• Small Doses that can be Frozen and Shipped• Ease-of-Handling by Doctor7
    • Differentiation Media is Not Permissive for hESCsHarvest forCryopreservationES CellsEBFormation RPE isolation P1 P20 5 weeks 12 weeks 15 weeks 18 weeksUp to 10 percent hESC spikedNo hESC’s in harvested cellsLimit of Detection for hESC1:20,000,000 cellsRPE differentiation is the default pathway underACT’s Patented Culture Conditions3 weeksEBOutgrowth30 Weeks8
    • RPE Animal Safety StudiesExtensive Safety Studies ShowsLack of TumorigenicityLong-term data(spanning the life ofthe animals) revealedno evidence ofteratoma formationafter subretinal hESC-RPE transplantation.9
    • RPE Engraftment – Mouse ModelFor each set: Panel (C) is a bright field image andPanel (D) shows immunofluorescence with anti-human bestrophin (green) and anti-humanmitochondria (red) merged and overlayed on thebright field image. Magnification 400xHuman RPE cells engraftand align with mouse RPEcells in mouse eye10
    • RPE Engraft and Function in Animal StudiesRPE treatment in animal model of retinal dystrophy has slowed thenatural progression of the disease by promoting photoreceptorsurvival.RPE cells rescued photoreceptors andslowed decline in visual acuitytreated controlPhotoreceptorlayer11
    • • Stargardt’s (SMD) Disease• IND approved in November 2010• European CTA filed• Orphan Drug Designation granted in U.S. and Europe• The SMD patient is a 26 year old female with baseline best corrected visual acuityof hand motion that corresponded to 0 letters in the ETDRS chart.• Dry AMD• IND approved in December 2010• European CTA in preparation• The dry AMD patient is a 77 year old female with baseline BCVA of 20/500, thatcorresponded to 21 letters in the ETDRS chart.RPE Program SummaryJuly 12, 2011: First Patients in each trialwere treated by Dr. Steven Schwartz, M.Dat Jules Stein Eye Institute (UCLA)12
    • • Also referred to as “Juvenile Macular Degeneration”– Causes progressive vision loss beginning in childhood.– Stargardt’s Disease is the most common hereditary macular dystrophy.– Prevalence rate of about 1-in-10,000.– Usually diagnosed in individuals under the age of twenty.• ACT has obtained Orphan Drug Designation in United States and Europe– 7 - 10 Years of Market Exclusivity for using RPE cells to treat Stargardt’s Disease.Stargardt’s Macular DystophyOrphan Drug Opportunity with Reimbursement80,000-100,000 patients in North America and Europe13
    • • AMD - estimate over 30 Million patients in North America and Europe– The prevalence of AMD in North America in the population aged 40 to 79 years is 8.8%– The prevalence of AMD in China in the population aged 40 to 79 years is 6.8%• Approximately 10% of people ages 66 to 74 have symptoms of macular degeneration• Prevalence increases to 30% in patients 75 to 85 years of age.Dry AMD (non-exudative)– The most common form of AMD (estimates as high as 90 percent)– No Effective Therapy Currently Available– Estimated $20-30 Billion marketAge-Related Macular DegenerationPotential forBlockbuster Status14
    • • 12 Patients for each trial, ascending dosages of 50K, 100K, 150K and 200K cells.– For each cohort, 1st patient treatment followed by 6 week DMSB review before remainder of cohort.• Patients will be monitored weekly - including high definition imaging of retinaHigh Definition Spectral Domain Optical Coherence Tomography (SD-OCT)Retinal AutofluorescenceAdaptive Optics Scanning Laser Ophthalmoscopy (AOSLO)Phase I - Clinical Trial Design50K Cells 100K Cells 150K Cells 200K CellsPatient 1 Patients 2/3DSMB Review DSMB ReviewEngraftment and photoreceptor activity dataavailable early in Phase I study.Permit comparison of RPE andphotoreceptor activity beforeand after treatment15
    • Phase I - Clinical Trial Update• Prospective clinical studies to determine the safety andtolerability of sub-retinal transplantation of hESC-derived RPEcells.• Vitrectomy including surgical induction of posterior vitreousseparation from the optic nerve was carried out• Submacular injection of 50,000 hESC-derived RPE cells in avolume of 150µl was delivered into a pre-selected area of thepericentral macula• Patients are monitored for systemic safety signals.• Pre- and weekly postoperative ophthalmic examinations.Visual acuity, fluorescein angiography, optical coherence tomography(OCT), autofluorescence imaging and visual field testing• DSMB Review UnderwayMore to come….Early clinical and laboratoryfindings with respect to safety,tolerability and engraftmentto be made availableDrs. Steven Schwartz and Robert LanzaStraight forward surgical approach16
    • Next Ocular Program – Corneal Endothelium• More than 10 million people with corneal blindness• The cornea is the most transplanted organ (1/3 of alltransplants performed due to endothelial failure)• Solutions include the transplantation of whole cornea“Penetrating Keratoplasty” (PKP)• More popular: Transplantation of just cornealendothelium & Descemet’s membrane (DSAEK).hESC-derived cornealendothelium resemblesnormal human cornealendothelium17
    • ACT HemangioblastProgram
    • Hemangioblast Program – JV Update• Stem Cell & Regenerative Medicine International (SCRMI).– ACT and CHA agree to restructure their joint venture.– SCRMI exclusively licensed the rights to hemangioblast program to ACT for North America and to CHA Biotech for Koreaand Japan.– SCRMI scientists reassigned to ACT to continue research and product development efforts as ACT employees– Both companies will work to develop clinical therapies based on the joint ventures proprietary hemangioblast celltechnology.• Products Opportunities include:– Universal Blood Components, such as Red Blood Cells and Platelets– Meschenchymal Stem Cells• Products for treating inflammatory diseases, promoting tolerance to grafts,repairing connective tissues, delivering therapeutic proteins, etc.– Revascularization Therapies for treating ischemic injuries• September 13, 2011: U.S. Patent 8,017,393 broadly covers ACT’s proprietary method forderiving hemangioblast cells from embryonic stem cells.19RobustProductPipeline
    • Hemangioblast Program: OverviewThe Hemangioblast cell is a multipotent cell, and acommon precursor to hematopoietic and endothelial cells.Hemangioblast cells can be used toproduce all cell types in the circulatoryand vascular systems• Hemangioblast cells can:• Self-renew.• Differentiate into both hematopoietic andendothelial cell lineages – and newlydiscovered by ACT, mesenchymal stemcells (MSC).• Be used to generate pathogen-free bloodcomponents• Be used to achieve vascular repair20Revascularization Blood Products
    • Hemangioblasts RBCsGeneration of Blood Products - RBCHemangioblasts EnucleatedRBC’sCapable of generating large quantities ofenucleated red blood cells.21- Blood Replacement Products- Triage and battlefield applications- Systemic Delivery System- Blood cells are great for transportingmaterials through the body as theentire circulatory system evolved tofacilitate their movement- Examples: Delivery of drugs orimaging agents
    • Generation of Blood Products - Platelets• Scalable: Generation of large quantities of Platelets from hESC and iPS sources• Off-the-shelf allogeneic products: Short in vivo half-life (7 days) and allotolerance for acute uses• Tractable regulatory approval process• Therapeutic products for accelerating soft and hard tissue healing.– Platelets improve the process of tissue repair – release agents involved in inflammation, angiogenesis and extracellularmatrix synthesis – all involved in wound repair• Product Opportunties: bone fractures and bony defects; laminectomy procedures; lateral epicondylitis (i.e., tenniselbow); total joint arthroplasty procedures (i.e., knee, hip, shoulder); plantar fasciitis; shoulder arthroscopy anddistal clavicle resection; and spinal fusion.• Extensive Cosmetic Uses– Wrinkle and Lift Procedures: Platelets are loaded with growth factors for skin and blood vessels and produce newcollagen without the wounds of a laser and the risk of scarring22 Efficiently generate functional megakaryocytes & platelets.• ES-derived platelets participate in clot formation.• ES-derived platelets incorporate into mouse thrombus at site of laser-induced arteriolar injury
    • Repair of Vascular DamageHemangioblasts were tested in animal models of diabetic retinopathy,heart disease and peripheral vascular damageResults from treatment withhemangioblast cells• Restoration of blood flow toischemic limbs.• Survival after myocardial infarction.• Revascularizes ischemic retinasPotential Impact on a Large Number of Vascular Diseasesmyocardial infarction, vascular ischemic damage, ischemia-reperfusioninjury, diabetic vascular disease and peripheral artery disease (PAD) that areleading causes of death and/or disability worldwide.Hemangioblasts promoted repair in peripheral vascular damage23
    • Mesenchymal Stem CellshESC-MSCs can beobtained using thehemangioblast method• hESC-MSCs are easy to derive andcan be expanded to large numbersin vitro• Quality controls are easier tomanage for a renewable cell source• Can serve as an “off the shelf”therapy, available for immediate use• Products for treating inflammatorydiseases, promoting tolerance tografts, repairing connective tissues,delivering therapeutic proteins, etc.24
    • Platform Technology for GeneratingRobust Human Embryonic Stem CellsWithout the Need to Destroy EmbryosSingle Blastomere Technology
    • First Proven Alternative hESC Method• Enables Derivation of new hESC Lines via single cell biopsymethod  Does not change the fate of the embryo from which thebiopsy was taken• Utilizes single cell biopsy similar to pre-implantation genetic diagnostics(PGD).• Roslin Cells and ACT plan to generate GMP-compliant bank of humanES Cells for research and commercial uses.• Head-to-head comparison with 24 NIH lines: Average 5X more efficientthan best NIH lines for producing cells from all three germ layers.26Single Blastomere Technology
    • Intellectual Property OverviewRetinal Pigment Epithelial Cells•Worldwide Patent Portfolio•Dominant Patent Position for Treating Retinal Degeneration• US Patent 7,794,704 broadly cover methods for treating retinal degeneration using human RPE cells differentiated from humanembryonic stem cells (hESCs).•Broad Coverage for Manufacturing RPE Cells from hESC• U.S. Patents 7,736,896 and 7,795,025 are broadly directed to the production of retinal pigment epithelial (RPE) cells from humanembryonic stem cells.Single Blastomere Technology•Worldwide Patent Filings•Broad Claims to use of Single Blastomeres• U.S. Patent 7,893,315 broadly covers ACT’s proprietary single-blastomere technology that provides a non-destructive alternative forderiving human embryonic stem cell (hESC) lines.Hemangioblast Technology•Worldwide Patent Filings•U.S. Patent 8,017,393 - Dominant Patent Position for deriving hemangioblast cells from embryonic stem cells.Other Notables•Controlling Filings (earliest priority date) to use of OCT4 relating to induced pluripotency (iPS).•Pending and issued patent filings directed to significant protocols for transdifferentiation.27
    • Financial Update – Strong Balance Sheet28Most Stable Financial Situation In Company History• The Company ended 2011 Q2 with $16.1 million cash on hand• $17 million more equity available• Virtually debt-free• Able to self-fund both U.S. clinical trials and EP clinical trial• Significantly deepened management team (and on-going)• Gary Rabin appointed CEO (change from interim status)• Exciting new Board of Director members to announce• Unqualified audit opinionEntering clinical trials with astrong balance sheet
    • ACT Management TeamWorld Class Scientific TeamSeasoned Management TeamDr. Robert Lanza, M.D. – Chief Scientific OfficerDr. Irina Klimanskaya, Ph.D. – Director of Stem Cell BiologyDr. Matthew Vincent, Ph.D. – Director of Business DevelopmentEdmund Mickunas – Vice President of Regulatory AffairsBill Douglass – Director of Corporate Communications & Social MediaStephen Price – Interim SVP – Corporate DevelopmentGary Rabin – Chairman and CEOKathy Singh - ControllerRita Parker – Director of Operations29
    • Thank you for Attending.For more information, visit www.advancedcell.com