Act corporate-presentationfeb13


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Act corporate-presentationfeb13

  2. 2. 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 2
  3. 3. ACT is a publicly-traded biotechnology company • Develop cellular therapies for the treatment of diseases and conditions that impact hundreds of millions of people worldwide • Premier scientific and research development team • Cell therapies are traditionally derided for being high cost, high touch and having lack of scalability. Not our approach at all >> Centralized, Scalable, Cost Effective Cell Therapy Products << About ACT: – Ticker – ACTC – Market Capitalization: $190 million – Principal lab and GMP facility: Marlborough, MA – Corporate HQ: Santa Monica, CA – Only hESC-derived tissue trials ongoing worldwide 3 Company Overview
  4. 4. Multiple Pluripotent Cell Platforms Single Blastomere-derived Embryonic Stem Cell Lines Generating hESC lines WITHOUT DESTRUCTION OF EMBRYO Utilizes a SINGLE CELL BIOPSY ACT never needs to make another hESC line again 4 Final Product Definition: hESC-derived products will be manufactured using a cell line made in 2005 from single cell isolated without the destruction of any embryos
  5. 5. Multiple Pluripotent Cell Platforms Induced Pluripotency Stem Cells (iPS cells) Generating pluripotent cell lines FROM ADULT TISSUE ….potential for patient specific stem cell lines • Early Innovator in Pluripotency (before iPS was even a term!) • Controlling Patent Filings (earliest priority date) to use of OCT4 for inducing pluripotency 5
  6. 6. RPE Clinical Program
  7. 7. 7 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.
  8. 8. 8 Life Support to Photoreceptors Loss of RPE cells Build up of toxic waste Loss of photoreceptors Dry AMD Bruch’s Mem. dehiscence Choroidal neovascularization Wet AMD
  9. 9. 9 RPE Therapy- Rationale Dry AMD represents more than 90% of all cases of AMD North America & Europe alone have more than 30 million dry AMD patients who should be eligible for our RPE cell therapy What if we could replace missing RPE cells?
  10. 10. RPE cell therapy may impact over 200 retinal diseases 10 RPE Therapy- Rationale • Massive unmet medical need • Unique measuring and observation environment • Easy to identify – aids manufacturing • Small dosage size – less than 200K cells • Immune-privileged site - minimal/no immunosuppression • Ease of administration - no separate device approval
  11. 11. GMP process for differentiation and purification of RPE – Virtually unlimited supply from stem cell source – Optimized for manufacturing Ideal Cell Therapy Product – Centralized Manufacturing – Small Doses – Easily Frozen and Shipped – Simple Handling by Doctor GMP Manufacturing 11 Product Cold Chain is Easily Scaled for Global Sales ACT Cleanroom Suite
  12. 12. Preclinical Models 12 Injected human RPE cells repair monolayer structure in eye Transplanted cells engraft and form correct anatomical structure Mouse model for macular degeneration
  13. 13. Preclinical Models 13 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
  14. 14. Phase I - Clinical Trial Design 14 SMD and dry AMD Trials approved in U.S., SMD Trial approved in U.K. 12 Patients / trial plus 4 in new cohort 2a – better vision ascending dosages of 50K, 100K, 150K and 200K cells. Regular Monitoring - including high definition imaging of retina 50K Cells 100K Cells 150K Cells 200K Cells Patient 1 Patients 2/3 DSMB Review DSMB Review
  15. 15. Participating Retinal Surgeons 15 Steven D. Schwartz, MD Chief, Retina Division Ahmanson Professor of Ophthalmology Director, Diabetic Eye Disease and Retinal Vascular Center Director, Ophthalmic Photography Clinical Laboratory Jules Stein Eye Institute Wills Eye Institute Carl D. Regillo, MD Director, Retina Service Professor of Ophthalmology, Jefferson Medical College Byron L. Lam, MD Director, Clinical Visual Physiology Professor of Ophthalmology Bascom Palmer Eye Institute
  16. 16. Participating Retinal Surgeons 16 Dean Eliott, MD Associate Director, Retina Service Professor, Harvard Medical School Massachusetts Eye and Ear Infirmary Moorfields Eye Hospital James Bainbridge, MA MB BChir PhD FRCOphth Professor of Retinal Studies, UCL Institute of Ophthalmology Philip J. Rosenfeld, MD PhD Professor of Ophthalmology Bascom Palmer Eye Institute Edinburgh Royal Infirmary Baljean Dhillon BMed Sci, BM BS, FRCS Surgeon, Edinburgh Royal Infirmary Surgeon, Lothian University Hospitals NHS Trust Professor of Ophthalmology, University of Edinburgh Professor of Visual Impairment Studies, Heriot Watt University
  17. 17. Surgical Overview 17 Procedure: • 25 Gauge Pars Plana Vitrectomy • Posterior Vitreous Separation (PVD Induction) • Subretinal hESC-derived RPE cells injection • Bleb Confirmation • Air Fluid Exchange
  18. 18. Preliminary Results 18 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.
  19. 19. 19 Engraftment and Survival: SD-OCT image collected at month 3 show survival and engraftment of RPE SMD US01 3mo post-op Preliminary Results – Structural
  20. 20. 20 Baseline Injection site Month 1 Month 2 SMD PATIENT
  21. 21. 21 Baseline 2 months DRY AMD PATIENT
  22. 22. Current Safety Profile 22 12 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 6 dry AMD Patients Treated 3 patients (50K cells cohort) treated > Cohort Complete 3 patient (100K cells cohort) treated > Cohort Complete No reports of any adverse events or complications due to cells • No evidence of inflammation or infiltration • No evidence of ectopic tissue formation • No evidence of retinal detachment
  23. 23. Expanding Clinical Programs 23 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 causes fissures in RPE layer. • MMD can occur at ages as young as 30 years old. FDA Approved IND – MMD Phase I/II study Jules Stein Eye Institute (UCLA) and ACT • Will use ACT’s RPE cells to treat MMD • Primary focus will be to evaluate the safety • Dr. Stephen Schwartz is the investigator
  24. 24. RPE Program Milestone Objectives 24 Key upcoming milestones • Continue to treat and review patient data FDA has recently approved vision as good as 20/100 in treated eye for patient inclusion criteria • Define efficacy endpoints and targeted patient visual criteria • Treat earlier stage disease to determine curative power of dissociated cell injections • Simplify shipping and cell-prep to enhance scaled distribution platform
  25. 25. Intellectual Property – RPE Program Treatment Dominant Patent Position for Treating Retinal Degeneration Manufacturing Broad Coverage for Manufacturing RPE Cells from hESC Preparations Claims directed to pharmaceutical preparations of RPE Cells from hESC, including both cell suspensions and scaffolded RPE layers. Sources Issued patents cover RPE Cells derived from other pluripotent stem cells (including iPS cells) Vigilance Regularly Filing on Improvements • Extend patent life cycle, with significance to commercialization • Include composition-of-matter claims (cell preparations, pharmaceutical preparations, etc.) 25
  26. 26. RPE Program - Investment Thesis 26 • Immense unmet medical need • Small Doses • Immunoprivileged – permits central (allogeneic) source of cells • Noninvasive monitoring of retina Market potential: More than 50 million patients in major markets. 1% market penetration may represent $5-10B market opportunity. Orphan indications are meaningful: Estimating a 10% market penetration with reoccurring treatments every 3-5 years, Stargardt’s disease can be a $100+ million/year product.
  27. 27. Therapeutic Pipeline - Ocular Programs
  28. 28. Retinal Pigment Epithelial Cells  Macular Degeneration - dry AMD  Retinitis Pigmentosa  Photoreceptor protection Hemangioblast cells  Ischemic retinopathy – diabetic retinopathy, vascular occlusions Retinal Neural Progenitor cells Isolated Protective Factors  Photoreceptor Loss, Modulation of Müller Cells  Protection of Retinal Ganglion cells (Glaucoma) Corneal Endothelium, Corneal Epithelium, Descemet’s Membrane  Corneal Disease Mesenchymal Stem Cells  Glaucoma, Uveitis  Retinitis Pigmentosa  Management of Ocular Surfaces 28 Retina
  29. 29. Mesenchymal Stem Cell Program 29
  30. 30. Mesenchymal Stem Cells in Therapy 30 Mesenchymal stem cells (MSCs) regulate immune responses promising therapeutic potential for treating autoimmune and inflammatory diseases. • Allogeneic - without HLA matching. • Track Record - Adult-derived MSCs already in 200+ clinical trials. Derived from Bone Marrow and Adipose Tissue However, allogeneic adult-derived MSC’s are limited by replicative capacity and as it turns out, a relative lack of potency
  31. 31. A differentiated MSC 31 Unlimited Supply of Donor-less MSCs • Immense replicative capacity • hESC & iPS - inexhaustible sources of starting material Advantages for Manufacturing • Use Single Master Cell Bank • Simplifies FDA/regulatory process • No need for continually finding and qualifying donors • Less labor-intensive
  32. 32. A differentiated MSC 32 Our mesenchymal stem cells are substantially more potent than both bone marrow and adipose derived MSC
  33. 33. Potential applications 33 • >100 autoimmune diseases • Multiple Sclerosis • Osteoarthritis • Crohn’s Disease/IBS • Aplastic Anemia • Chronic Pain • Limb Ischemia • Heart Failure/MI • Stroke • Graft-versus-host Disease • Spinal Cord Injury • Parkinson’s Disease • Liver Cirrhosis • Emphysema/Pulmonary Diseases • Wound healing (ulcers/decubitus/burns) • HSC engraftment/irradiated cancer patients • Eye diseases (uveitis, retinal degeneration, glaucoma)
  34. 34. ACT Blood Components Program
  35. 35. Hemangioblast Program: Overview 35 Hemangioblast: common precursor to hematopoietic and endothelial cells. Multipotent cell produces all cell types in the circulatory and vascular systems
  36. 36. Generation of Blood Products 36 Hemangioblasts RBCsHemangioblasts Enucleated RBC’s Process generates large quantities of functional red blood cells and megakaryocytes & platelets
  37. 37. The Case for Platelets 37 Platelets are key elements of hemostasis and thrombosis as well as tissue regeneration after injury or surgery. • Maintain vascular integrity and play vital roles in wound repair • Thrombocytopenia is a major cause of morbidity in sepsis, cancer and preeclampsia • Used regularly in soft- and hard-tissue applications in most fields of surgery. . Beyond Donor Supply: Estimate demand for additional 1-2M units/year
  38. 38. The Case for Platelets 38 Platelets are the blood product most difficult to maintain without unnecessary wastage. Platelets do not tolerate refrigeration. Storage at room temperature is limited to 5–7 days Need for new strategies to generate platelets for infusion therapy.
  39. 39. Platelets 2.0 39 Ability to control platelet manufacturing provides opportunities to improve storage, fine tune platelets for use in wound healing applications, as well as to engineer new roles for platelets beyond traditional involvement in wound healing. • Improve cryopreservation of platelets • Make lyophilization of platelets tractable solution • Utilize platelets for drug delivery • Utilize platelets in imaging (load with contrast agents) CONFIDENTIAL - BUSINESS PROPRIETARY
  40. 40. Achieving Function and Scale 40 • Ultrastructural and morphological features of SC-derived platelets are indistinguishable from normal blood platelets. • SC-derived platelets respond to thrombin stimulation, form microaggregates, and facilitate clot formation and retraction. • SC-derived platelets contribute to developing thrombi at sites of laser-induced vascular injury in mice - first evidence for in vivo functionality of SC-derived platelets.
  41. 41. Clinical Program Status 41 • Animal model studies show proper in vivo function • Achieved clinical dose scale manufacturing • Completely feeder-free process (bioreactor capable!) • Pre-IND meeting requested with FDA
  42. 42. Sample 10 year plan 42 Achieve 100 plt/mk Achieve 500 plt/mk Achieve 1011 scale Achieve 1012 scale Achieve 1014 scale Phase 0 Study Prepare and File IND Phase I/II Study Phase II File Exploratory IND EOP I meeting Achieve 1 Million Dose Scale 1yr 2yr 3yr 4yr 5yr 10yr Achieve 2000-5000 plt/mk
  43. 43. ACT Corporate Overview
  44. 44. Financial Update – Strong Balance Sheet 44 • Company ended 2012 Q3 with $42 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
  45. 45. 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 45
  46. 46. 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 46 World Class Board of Directors Highly-regarded Ethics Advisory Board
  47. 47. Thank you For more information, visit