Act april13regen-med-investor-day


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Act april13regen-med-investor-day

  1. 1. 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 Cautionary Statement Concerning Forward-Looking Statements 1 LEADING REGENERATIVE MEDICINE
  2. 2. 0 50 100 150 200 250 Per1000population Arthritis/ other musculoskeletal Heart/ other circulatory Vision Exemplary Chronic Health Conditions Source: U.S. Census Bureau Publication “65+ in the United States”, P23-209 2 18-44 45-64 65-74 75+ Age Groups
  3. 3. 133.7 142.2 173.4 202.7 207.4 205.5 37.4 52.9 64.8 81.9 105.3 121 214.6 274.6 395.9 568.5 752.2 870.4 34.4 53.2 80.1 121 198.5 305.3 2010 2020 2030 2040 20502000 2010 2020 2030 2040 20502000 80 and over 65 to 79 Population Aged 65 and Older for Developed and Developing Countries (in millions) Developed Countries Developing Countries Source: U.S. Census Bureau Publication “65+ in the United States”, P23-209 3
  4. 4. 4 Structure of Retina The Retina the light-sensitive tissue lining the inner surface of the eye Retina
  5. 5. 5 Life Support to Photoreceptors Provides nutrients and growth factors • photoreceptors see no blood Recycles Vitamin A • maintains photoreceptor excitability Detoxifies photoreceptor layer Maintains Bruch’s Membrane • natural antiangiogenic barrier • immune privilege of retina Absorbs stray light / protects from UV RPE Layer has multiple critical roles in the health and function of photoreceptors and the retina as a whole.
  6. 6. 6 Life Support to Photoreceptors Failure of RPE cells results in many degenerative diseases Stargardt’s disease Myopic Macular Dystrophy Age-related macular degeneration (AMD)
  7. 7. 7 RPE Therapy- Rationale • Massive unmet medical need • Easy to identify – aids manufacturing • Small dosage size – less than 200K cells • Immune-privileged site - minimal immunosuppression • Ease of administration - no separate device approval • Unique measuring and observation environment
  8. 8. Preclinical Models 8 Injected human RPE cells repair monolayer structure in eye Transplanted cells engraft and form correct anatomical structure Mouse model for macular degeneration
  9. 9. Preclinical Models 9 untreated treated Photoreceptor layer photoreceptor layer is lost Transplanted RPE cells protect photoreceptors and prevent loss of vision Rat model for macular degeneration •Untreated animals go blind •Treated animals maintain 70-80% of normal vision
  10. 10. 10 Participation by the leading retinal surgeons in the world Jules Stein (UCLA) Mass Eye & Ear Infirmary Wills Eye Institute Bascom Palmer Eye Institute Moorfields Eye Hospital Edinburgh Royal Infirmary
  11. 11. Surgical Overview 11 Procedure: • 25 Gauge Pars Plana Vitrectomy • Posterior Vitreous Separation • Subretinal hESC-derived RPE cells injection • Bleb Confirmation • Day Surgery/Sedation
  12. 12. Preliminary Results 12 No Adverse Events No signs of hyperproliferation, abnormal growth, rejection or retinal detachment. Persistence of cells Anatomical evidence of hESC-RPE survival and engraftment. Increased pigmentation within the bed of the transplant. Impact on Acuity Recorded functional visual improvements in both patients.
  13. 13. Preliminary Results 13 Persistence of cells Anatomical evidence of hESC-RPE survival and engraftment. Increased pigmentation within the bed of the transplant. Engraftment and Survival: SD-OCT image collected at month 3 show survival and engraftment of RPE
  14. 14. Preliminary Results 14 Baseline Injection site Month 1 Month 2 Increased pigmentation within the bed of the transplant. Persistence of cells
  15. 15. Preliminary Results 15 • Varying degrees of improvement in visual acuity across patients Persistence for 18+ months in first patients • Increased letters on ETDRS Charts • Color perception • Contrast • Low light vision These are very late stage patients with a high degree of heterogeneity in degree of “rescueable” photoreceptors Impact on Visual Acuity
  16. 16. Halfway Point 16 Based on safety and functional data from first 18 patients, FDA has approved new 4 patient cohorts in each trial. Best Vision Inclusion Criteria for new Cohort 2a includes patients with vision as good as 20/100. 50K Cells 100K Cells 150K Cells 200K Cells 100K CellsFDA Approved “Cohort 2a” Inclusion Criteria: vision 20/100+
  17. 17. Current Safety Profile 17 14 SMD Patients Treated 6 patients (50K cells cohort) treated – US&UK Trials > Cohort Complete 6 patient (100K cells cohort) treated – US&UK Trials > Cohort Complete 1 patient (150K cells cohort) treated – US 1 patient (“Cohort 2a”) treated – US 7 dry AMD Patients Treated 3 patients (50K cells cohort) treated > Cohort Complete 3 patient (100K cells cohort) treated > Cohort Complete 1 patient (150K cells cohort) treated – US
  18. 18. RPE Program Milestone Objectives 18 Key upcoming milestones • Continue to treat and review patient data • Treat earlier stage disease to determine curative power of dissociated cell injections • Define efficacy endpoints and targeted patient visual criteria • Simplify shipping and cell-prep to enhance scaled distribution platform
  19. 19. Expanding Clinical Programs 19 Myopia creates a higher risk of permanent vision loss due to Myopic Macular Degeneration (MMD) • Severe near-sightedness causes elongation of the eyeball -- which can cause fissures in RPE layer. January 2013 - FDA Approved MMD Phase I/II study Jules Stein Eye Institute (UCLA) and ACT
  20. 20. Intellectual Property – RPE Program Dominant Patent Position for Treating Retinal Degeneration Broad Coverage for Manufacturing RPE Cells Broad protection of pharmaceutical preparations • RPE cell suspensions • scaffolded RPE layers. RPE Cells derived from other pluripotent stem cells Vigilant filing on improvements 20
  21. 21. Therapeutic Pipeline 21 Retinal Neural Progenitor cells & Isolated Protective Factors Photoreceptor Loss, Modulation of Müller Cells Protection of Retinal Ganglion cells (Glaucoma) Corneal Endothelium Corneal Disease Hemangioblast cells Ischemic retinopathy – diabetic retinopathy, vascular occlusions Mesenchymal Stromal Cells Ocular - Glaucoma, Uveitis, Retinitis Pigmentosa Autoimmune Diseases Inflammatory Diseases or disorders
  22. 22. Generation of Blood Products 22 Hemangioblasts RBCsHemangioblasts Enucleated RBC’s Process generates large quantities of functional red blood cells and megakaryocytes & platelets
  23. 23. ACT Corporate Overview
  24. 24. Financial Update – Strong Balance Sheet 24 • Company ended 2012 Q4 with $40 million in cash or availability of cash through financing commitments • $16 million annual cash-burn rate (funded through early 2015) • Settled nearly all litigation hangover from previous management
  25. 25. ACT Management Team Highly Experienced and Tightly Integrated Management Team Gary Rabin – Chairman & CEO Dr. Robert Lanza, M.D. – Chief Scientific Officer Edmund Mickunas – Vice President of Regulatory Affairs Dr. Irina Klimanskaya, Ph.D. – Director of Stem Cell Biology Dr. Shi-Jiang (John) Lu, Ph.D. – Senior Director of Research Dr. Roger Gay, Ph.D. - Senior Director of Manufacturing Kathy Singh - Controller Rita Parker – Director of Operations Dr. Matthew Vincent, Ph.D. – Director of Business Development Bill Douglass – Dir. of Corporate Communications & Social Media 25
  26. 26. Dr. Ronald M. Green: Chairman Dr. Judith Bernstein Dr. Jeremy B.A. Green Dr. Robert Kauffman Dr. Carol A. Tauer ACT Leadership Gary Rabin: Chairman & CEO Dr. Robert S. Langer, ScD: Prolific medical inventor; Chair – ACT SAB Gregory S. Perry: EVP – Immunogen Michael Heffernan: CEO – Collegium Pharma Zohar Loshitzer: CEO Presbia; Founder LifeAlert Medical Dr. Alan C. Shapiro: Renowned business school professor 26 World Class Board of Directors Highly-regarded Ethics Advisory Board
  27. 27. Thank you For more information, visit