Leading Regenerative Medicine September 2011
Cautionary Statement Concerning Forward-Looking Statements This presentation is intended to present a summary of ACT’s (“ACT”, or “Advanced Cell Technology Inc”, or “the Company”) salient business characteristics. 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 Exchange Commission. The risks identified therein, as well as others not identified by the Company, could cause the Company’s actual results to differ materially from those expressed in any forward-looking statements. Ropes Gray 2
At the Forefront of Regenerative Medicine • Patented Technology for Producing 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 stromal cells products • Myoblast program for heart failure approved for Phase II 3
ACT Ocular Programs
Retinal Pigment Epithelial Cells - Rationale The RPE layer is critical to the function and health of • photoreceptors and the retina as a whole. RPE cells secrete trophic factors and impact on the chemical environment of the subretinal space. recycle photopigments – deliver, metabolize and store vitamin A – transport iron and small molecules between retina and choroid – – maintain Bruch’s membrane RPE malfunction may lead to photoreceptor loss and eventually blindness Discrete differentiated cell population Failure of RPE results in disease progression 5
Retinal Pigment Epithelial Cells - Rationale • Pigmented RPE cells are easy to identify (no need for further staining) • Small dosage vs. other therapies • The eye is generally immune-privileged site, thus minimal immunosuppression required, which may be topical. • 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 doctors RPE cell therapy may impact over 200 retinal diseases 6
GMP Manufacturing • Established GMP-compliant process for the Reproducible Differentiation and 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 controls and demonstrates the full transition from the hESC state. Ideal Cell Therapy Product • Centralized Manufacturing • Small Doses that can be Frozen and Shipped • Ease-of-Handling by Doctor 7
RPE Engraftment – Mouse Model Human RPE cells engraft and align with mouse RPE cells in mouse eye For each set: Panel (C) is a bright field image and Panel (D) shows immunofluorescence with anti- human bestrophin (green) and anti-human mitochondria (red) merged and overlayed on the bright field image. Magnification 400x 8
RPE Engraft and Function in Animal Studies RPE treatment in animal model of retinal dystrophy has slowed the natural progression of the disease by promoting photoreceptor survival. control treated Photoreceptor layer RPE cells rescued photoreceptors and slowed decline in visual acuity 9
RPE Program Summary • 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 46 year old female with baseline best corrected visual acuity • of hand motion that corresponded to 0 letters in the ETDRS chart. July 12, 2011: First Patients in each trial • Dry AMD were treated by Dr. Steven Schwartz, M.D at Jules Stein Eye Institute (UCLA) 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, that • corresponded to 21 letters in the ETDRS chart. 10
Stargardt’s Macular Dystophy • 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. – Orphan Drug Opportunity with Reimbursement 80,000-100,000 patients in North America and Europe 11
Age-Related Macular Degeneration • 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 Potential for – Estimated $20-30 Billion market Blockbuster Status 12
Phase I - Clinical Trial Design • 12 Patients for each trial, ascending dosages of 50K, 100K, 150K and 200K cells. For each cohort, 1 st patient treatment followed by 6 week DMSB review before remainder of cohort. – Patients will be monitored weekly - including high definition imaging of retina • High Definition Spectral Domain Optical Coherence Tomography (SD-OCT) Permit comparison of RPE and photoreceptor activity before Retinal Autofluorescence and after treatment Adaptive Optics Scanning Laser Ophthalmoscopy (AOSLO) Patient 1 Patients 2/3 50K Cells 100K Cells 150K Cells 200K Cells DSMB Review DSMB Review Engraftment and photoreceptor activity data available early in Phase I study. 13
Phase I - Clinical Trial Update • Prospective clinical studies to determine the safety and tolerability of sub-retinal transplantation of hESC-derived RPE cells. Straight forward surgical approach Vitrectomy including surgical induction of posterior vitreous • separation from the optic nerve was carried out • Submacular injection of 50,000 hESC-derived RPE cells in a volume of 150µl was delivered into a pre-selected area of the Drs. Steven Schwartz and Robert Lanza pericentral macula • Patients are monitored for systemic safety signals. More to come…. • Pre- and weekly postoperative ophthalmic examinations. Early clinical and laboratory Visual acuity, fluorescein angiography, optical coherence tomography findings with respect to safety, (OCT), autofluorescence imaging and visual field testing tolerability and engraftment to be made available • DSMB Review Underway 14
Next Ocular Program – Corneal Endothelium More than 10 million people with corneal blindness • The cornea is the most transplanted organ (1/3 of all • transplants performed due to endothelial failure) • Solutions include the transplantation of whole cornea “Penetrating Keratoplasty” (PKP) • More popular: Transplantation of just corneal endothelium & Descemet’s membrane (DSAEK). hESC-derived corneal endothelium resembles normal human corneal endothelium 15
ACT Hemangioblast Program
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 Korea – and 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 venture's proprietary hemangioblast cell technology. Products Opportunities include: • Universal Blood Components, such as Red Blood Cells and Platelets – Robust Meschenchymal Stem Cells – Product • Products for treating inflammatory diseases, promoting tolerance to grafts, repairing connective tissues, delivering therapeutic proteins, etc. Pipeline – Revascularization Therapies for treating ischemic injuries • September 13, 2011: U.S. Patent 8,017,393 broadly covers ACT’s proprietary method for deriving hemangioblast cells from embryonic stem cells. 17
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