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Act april13regen-med-investor-day
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
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
Structure of Retina
The Retina the light-sensitive
tissue lining the inner surface of
the eye
Retina
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
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
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. 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
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. Surgical Overview
11
Procedure:
• 25 Gauge Pars Plana Vitrectomy
• Posterior Vitreous Separation
• Subretinal hESC-derived RPE cells
injection
• Bleb Confirmation
• Day Surgery/Sedation
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. 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
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. 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+
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. 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. 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
22. Generation of Blood Products
22
Hemangioblasts RBCsHemangioblasts Enucleated
RBC’s
Process generates large
quantities of functional
red blood cells
and
megakaryocytes &
platelets
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. 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. 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
