act corporate-bioeuropemarch13
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LEADING REGENERATIVE
MEDICINE
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
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There is a tremendous
UNMET MEDICAL NEED for treatments of dry AMD & other forms of macular degeneration
RPE Program - Investment Thesis
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Dry AMD: More than 50 million patients in major markets.
1% market penetration may represent $5-10B market opportunity.
Orphan indications: 10% market penetration of SMD alone may be a $100+ million/year product. Orphan status provides options for early authorization.
Immense Unmet Medical Needs
Small Doses & Globally Scalable Cold
Chain
Immune Privileged Injection Site
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Structure of Retina The Retina the light-sensitive tissue lining the inner surface of the eye
Retina
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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.
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Life Support to Photoreceptors
Failure of RPE cells results in many degenerative diseases Age-related macular degeneration (AMD) Myopic Macular Dystrophy Stargardt’s disease
RPE cell therapy may impact over 200 retinal diseases
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RPE Therapy- Rationale
• Massive unmet medical need • 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
• Unique measuring and observation environment
Preclinical Models
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Injected human RPE cells repair monolayer structure in eye
Transplanted cells engraft and form
correct anatomical structure
Mouse model for macular degeneration
Preclinical Models
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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
Phase I - Clinical Trial Design
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SMD and dry AMD Trials approved in U.S., SMD Trial approved in U.K.
12 Patients / trial 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
100K Cells FDA Approved “Cohort 2a” Inclusion Criteria: vision 20/100+
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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
Surgical Overview
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Procedure: • 25 Gauge Pars Plana Vitrectomy • Posterior Vitreous Separation (PVD
Induction) • Subretinal hESC-derived RPE cells
injection • Bleb Confirmation
Preliminary Results
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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.
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Engraftment and Survival: SD-OCT image collected at month 3 show survival and engraftment of RPE
SMD001 3mo post-op
Preliminary Results – Structural
Current Safety Profile
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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
RPE Program Milestone Objectives
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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
• 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
Expanding Clinical Programs
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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
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
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Generation of Blood Products
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Hemangioblasts RBCs Hemangioblasts Enucleated RBC’s
Process generates large quantities of functional red blood cells and
megakaryocytes & platelets
The Case for Platelets
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Platelets are key elements of hemostasis and thrombosis as well as
tissue regeneration after injury or surgery. • Wound repair and treatment of trauma • Thrombocytopenia • Reconstructive, plastic and joint replacement surgery • Current supply limits use: expanded use of platelets predicts a
market for several million more units of platelets yearly
Platelets are the blood product most difficult to maintain – cannot be refrigerated or frozen
Clinical Program Status
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• Animal model studies show proper in vivo function
• Achieved clinical dose scale manufacturing
• Completely feeder-free process (bioreactor capable!)
• Pre-IND meeting with FDA
SC-derived plts Incorporate into clot
Platelets 2.0
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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)
Therapeutic Pipeline
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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
Financial Update – Strong Balance Sheet
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• 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
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
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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
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World Class Board of Directors
Highly-regarded Ethics Advisory Board
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