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LEADING REGENERATIVE
MEDICINE
2013 Annual Global
Investment Conference
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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
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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
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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
Oph
thal
mol
ogy
Prog
ram
s
The Retina the light-sensitive tissue lining the inner surface of the eye
Retina
Structure of the Retina
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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
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Failure of RPE cells results in many degenerative diseases Stargardt’s disease Myopic Macular Dystrophy Age-related macular degeneration (AMD)
Life Support for Photoreceptors
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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%
50-59 40-49 60-69 70-79 80+ Age
Intermediate AMD Late AMD
% P
reva
lenc
e (U
.S.)
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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
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
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Procedure: • 25 Gauge Pars Plana
Vitrectomy • Posterior Vitreous Separation
(PVD Induction) • Subretinal hESC-derived RPE
cells injection • Bleb Confirmation • Air Fluid Exchange
Procedure takes ~90 secs, out-patient setting
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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
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• 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
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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
7/12 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
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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
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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 Treatment Phase II
Patient Treatment MMD Trial
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Sep 2013 July 2014
Phase I
Phase I
Therapeutic Pipeline - Other Programs
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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 Stromal Cells Glaucoma, Uveitis Retinitis Pigmentosa Management of Ocular Surfaces
Retina
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Mesenchymal Stem Cell Program
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Mesenchymal Stem Cells in Therapy
Self External Autoimmune Diseases Rheumatoid Arthritis, Psoriasis,
Multiple Sclerosis, Crohn’s disease, Type I Diabetes, Lupus
Allergic Reaction Asthma, eczema,
sinusitis
Immune Over-reaction
Balanced Immune System
Autoimmune Disease Prevalence • At least 80 disease affecting every organ system • Americans spend over $100B each year in total
healthcare costs associated with autoimmune disease • In the U.S., 14.7-23.5M people (5%-8%) (for comparison: heart disease (22M), Cancer (9M)
A rapidly growing health issue (% growth)
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Suppressing Immune Responses gives rise to Therapy
Promising therapeutic potential for treating autoimmune and inflammatory diseases.
However, adult-derived MSCs are limited by replicative capacity
Mesenchymal stem cells (MSCs) suppress disease-causing immune responses
ACT Proprietary Process • Manufacture MSC’s from hES and iPS Cell Banks
• Virtually inexhaustible source of starting material • Use Single Master Cell Bank • Less labor-intensive
A further differentiating feature…
Our MSC’s are substantially more potent than current sources of cells
ACT’s Breakthrough – Inexhaustible Supply of Very Potent MSC’s
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ACT Blood Components Program
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Hemangioblasts RBCs Hemangioblasts Enucleated RBC’s
Proprietary Process generates large quantities of functional red blood cells and
megakaryocytes & platelets
Generation of Blood Products
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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
Platelets are the blood product most difficult to maintain – cannot be refrigerated or frozen
The Case for Platelets
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ACT Corporate Overview
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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
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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
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Thank you For more information, visit www.advancedcell.com
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