2013 Aegis Capital Healthcare Conference, Sept 26th 2013


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2013 Aegis Capital Healthcare Conference, Sept 26th 2013

  2. 2. 2 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
  3. 3. 3 Three clinical trials aimed at very large unmet medical needs • Dry Age related Macular Degeneration and Stargardt’s Disease affect nearly 30 million people in the US and EU, expected to grow by 50% over next 10 years World-class cell biology capabilities • ACT scientific team continues to develop best-in-class, and only-in-class cell lines which create several preclinical opportunities across many disease areas Strong Intellectual Property Estate • 190 patents filed, 37 issued which provide ACT with sustainable competitive advantage for decades to come Active partnering program to monetize non-core assets • ACT management is dedicated to monetizing non-core pre-clinical assets and continues to optimize portfolio of opportunities to fuel future clinical programs Several Key Inflection Points Provide Near-term and Long-term Growth Opportunities
  4. 4. 4 Pre-clinical/ in vitro POC – Animal Studies IND Approved Phase I Phase II Phase III Approval Dry AMD SMD MMD Photo- receptors Ganglion Neurons Cornea Platelets Mesenchymal Stem Cells Robust Development Pipeline Provides Multiple Opportunities to Commercialize and Partner Potential Gov’t Funding First Priority Based On Current Funding Advance into Phase I and Partner Based on POC results, pursue appropriate funding and collaborations OphthalmologyPrograms
  5. 5. The Retina the light-sensitive tissue lining the inner surface of the eye Retina Structure of the Retina 5
  6. 6. 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. Life Support for Photoreceptors 6
  7. 7. Failure of RPE cells results in many degenerative diseases Stargardt’s disease Myopic Macular Dystrophy Age-related macular degeneration (AMD) Life Support for Photoreceptors 7
  8. 8. 8 Prevalence of AMD Increases Exponentially with Age Data from http://www.nei.nih.gov/eyedata/ and U.S. Census Bureau Publication “65+ in the United States”, P23-209 Exponential rise in prevalence and incidence rates with age, with prevalence rates of late AMD quadrupling per decade 133.7 142.2 173.4 202.7 207.4 205.5 37.4 52.9 64.8 81.9 105.3 121 80+ 65-79 2000 2010 2020 2030 2040 2050 Developed Countries “macular degeneration will soon take on aspects of an epidemic” - former Director of the National Eye Institute Dr Carl Kupfer 60% 50% 40% 30% 20% 10% 50-5940-49 60-69 70-79 80+ Age Intermediate AMD Late AMD %Prevalence(U.S.)
  9. 9. 9 Prevalence of AMD Increases Exponentially with Age Exponential rise in prevalence and incidence rates with age, with prevalence rates of late AMD quadrupling per decade The currently 30 Million American and European AMD patients are projected to become 45 Million patients by 2025
  10. 10. RPE 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 10
  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 only
  12. 12. Jules Stein (UCLA) Mass Eye & Ear Infirmary Wills Eye Institute Bascom Palmer Eye Institute Moorfields Eye Hospital Edinburgh Royal Infirmary World renowned leadership to help us navigate the clinical path and ultimately support market launch Clinical Trials being led by World Leaders in Ophthalmology 12
  13. 13. • No Adverse Events • Persistence of cells • Impact on Acuity Recorded functional visual improvements in majority of patients. • Increased letters on ETDRS Charts • Color perception • Contrast • Low light vision Phase I Trials Exceeding Expectations – no adverse events and persistence of cells 13
  14. 14. IND Approved 50% Patient Enrollment 100% Patient Enrollment U.S. – Dry AMD U.S. – SMD U.K. – SMD U.S. – MMD 12/16 patients treated 10/16 patients treated 8/12 patients treated Enrolling – 12 patients total 2 years since the first patients were treated Measurable Improvements in Visual Acuity for Majority of Treated Patients Active Clinical Programs in AMD and SMD Indicate Encouraging Results 14
  15. 15. January 2013: FDA approved additional 4 patient “better vision” cohorts in each trial. For Cohort 2a – can enroll patients with vision as good as 20/100. Cohort 1 50K Cells Cohort 2 100K Cells Cohort 3 150K Cells Cohort 4 200K Cells Cohort 2a 100K Cells First Treatments informed a more aggressive strategy to treat “better vision” cohort, could lead to broader label and/or earlier approval 15
  16. 16. Several Important clinical milestones 2H 2013, 1H 2014 150K 200K Cohort 2a 100K Jan 2014 Jan 2015 Dry AMD & SMD Trials Patient follow-up PII design Patient TreatmentPhase II Patient TreatmentMMD Trial 16 Sep 2013 July 2014 Phase I Phase I
  17. 17. Expanding Clinical Programs 17 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
  18. 18. 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 18
  19. 19. Therapeutic Pipeline 19 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
  20. 20. 20 BY PRODUCING THE HIGHEST DEGREE OF QUALITY VETERINARY THERAPEUTICS WE INTEND TO BE A LEADER IN VETERINARY REGENERATIVE MEDICINE September 19, 2013 – “ACT Files Investigational New Animal Drug (INAD) Application with FDA to Treat 10 Different Disease Indications Using Pluripotent Stem Cells”
  21. 21. ACT Corporate Overview 21
  22. 22. Gary Rabin – Chairman and CEO Edward Myles – CFO and EVP of Corp Development Dr. Matthew Vincent, Ph.D. – Director of Business Development Dr. Robert Lanza, MD – Chief Scientific Officer Dr. Irina Klimanskaya, Ph.D. – Director of Stem Cell Biology Dr. Shi-Jiang (John) Lu, Ph.D. – Senior Director of Research Edmund Mickunas – Vice President of Regulatory Affairs Dr. Roger Gay, Ph.D. - Senior Director of Manufacturing Proven business leaders who can develop and implement corporate strategy and monetize assets to maximize shareholder value World-renowned scientific thought leaders pushing the cutting edge of science to develop important therapies Deep experience navigating the complex regulatory landscape from development to market GMP manufacturing to ensure the highest quality products are delivered to our patients An Experienced and Dedicated Management Team 22
  23. 23. Michael Heffernan CEO – Collegium Pharmaceuticals Robert S. Langer, Sc.D. Institute Professor, MIT Zohar Loshitzer CEO – Presbia, Inc., & Principal in Ochard Capital Greg Perry EVP & CFO - Immunogen Alan C. Shapiro Finance Professor and Chairman of the Department of Finance and Business Economics (retired) – University of Southern California Gary Rabin Chairman and CEO – Advanced Cell Technology A World-Class Board of Directors 23
  24. 24. Thank you For more information, visit www.advancedcell.com