2. Ocata Therapeutics Inc. (âOcataâ or âthe Companyâ) has filed a registration statement (including a
prospectus and a preliminary prospectus supplement) with the Securities and Exchange Commission
(âSECâ) for the offering to which this presentation relates. Before you invest you should read the
prospectus and the preliminary prospectus supplement in that registration statement and other
documents the Company has filed with the SEC for more complete information about the Company and
the offering. You may get these documents for free on the SECâs website at http://www.sec.gov
These slides and the accompanying oral presentation contain statements that are not historical facts and
are considered forward-looking information. In some cases you can identify these statements by
forward-looking words such as âanticipate,â âbelieve,â âcould,â âcontinue,â âestimate,â âexpect,â âintend,â
âmay,â âshould,â âwillâ, âwould,â âplan,â âprojected,â or the negative of such words or other similar words
or phrases. Investors are cautioned not to unduly rely on forward-looking statements because they
involve risks and uncertainties and statements related to future events or our future financial
performance, and involve known and unknown risks, uncertainties and other factors that may cause our
actual results, levels of activity, performance or achievements to be materially different from any future
results, levels of activity, performance or achievements expressed or implied by these forward-looking
statements. These statements are also subject to a number of material risks and uncertainties that are
described more fully in the prospectus and the preliminary prospectus supplement filed with the SEC,
including without limitation our most recently filed Report on Form 10-Q, as filed with the SEC. These
forward-looking statements speak only as of the date on which the statements were made and are not
guarantees of future performance. Except as may be required by applicable law, we do note undertake or
indent to update any forward-looking statements contained herein or in our public filings with the SEC.
Cautionary Statement Concerning Forward-Looking
Statements
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3. Addressing Macular Degeneration with Groundbreaking RPE Therapy
The Regenerative Ophthalmology⢠Company
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Safety observed, in
addition to anatomical and
functional evidence of
repair and restoration in
Phase 1 trials for dry AMD
and SMD
⢠Data published in The
Lancet, October 14, 2014
Phase 2 study initiated:
⢠Dry Age-related Macular
Degeneration (AMD)
- Q4 2015
Extensive proprietary
position in major markets
protecting the entire value
chain of the cell therapy â
from the origin of the stem
cell to the delivery into
patientsâ eyes
4. Age-Related Macular Degeneration (AMD)
â Approximately 15M people in the US
â ~85-90% have dry AMD
â No treatment available currently to prevent, effectively treat or cure dry AMD
Stargardtâs Macular Degeneration (SMD)
â Leading form of inherited juvenile onset macular degeneration
â Orphan disease indication with no available treatment
Myopic Macular Degeneration (MMD)
â Prevalent condition especially in Asia affecting up to 13M in China and Japan
Ophthalmology: A Large, Growing and Underserved Market
4
Ocata's Regenerative Ophthalmology⢠R&D programs target the major forms of
macular degenerative disease with potentially transformative therapies
5. The First Evidence of Long-term Safety and Efficacy Signal
Following Transplantation of RPE Cells
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âWhat we did is transplant the cells into patients who
have a disease where those particular cells are dying;
and we replaced those dying tissues with new tissue
that's derived from these stem cells. In a way it's a
retinal transplant.
- Steven Schwartz, MD, Professor of Ophthalmology, UCLA
6. Further Evidence of Long-term Safety and Efficacy Signal
in Asian Patients with SMD and AMD â April 2015
6
Four Korean patients: two with dry AMD and two with
SMD
Follow up at 1 year:
â No evidence of adverse proliferation, tumorigenicity,
ectopic tissue formation, or other serious safety
issues related to the transplanted cells.
â Visual acuity improved 9â19 letters in three
patients and remained stable (+1 letter) in one
patient.
Total of forty four patients safely treated worldwide with RPE Therapy.
Independent trial in Asian population supports previously reported safety and efficacy signals
7. Core Technology: Terminal Differentiation of Pluripotent
to Target Cells
7
Pluripotent
Stem Cells
Starting Source
hESC
iPSC
Corneal
Retinal Ganglion
Photoreceptor
Retinal Pigment Epithelium
Terminally
Differentiated Cells
For Transplantation
Stem Cells are the Starting Material but fully Differentiated Cells are the Treatment
8. 1
2
3 4
8
Additional Cell Types in Development to Address a
Spectrum of Ocular Disorders
Photoreceptor Progenitor
⢠Macular Degeneration -
dry AMD, SMD, MMD
⢠Retinitis Pigmentosa
Retinal Ganglion Progenitor
⢠Glaucoma
Mesenchymal Stem Cells
⢠Uveitis
⢠Management of Ocular Surfaces
Corneal Endothelial Cells
⢠Corneal Disease
1
2
3
4
Rich preclinical pipeline of regenerative ophthalmology product opportunities
each addressing large unmet medical needs
9. Immune privileged
â Less prone to rejection
Compact Structure
â Relatively small doses
required to treat
Straightforward delivery using
currently available technology
Validated tools for clinical
outcome assessment
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The Eye is Well-Suited for Cellular Transplantation
11. Required for vision and
maintenance of photoreceptor
health
Delivers and metabolizes Vitamin A
â Recycles photopigments
Phagocytosis of photoreceptor
outer segments
Transport of metabolic waste from
retina to choriocapillaris
Absorbs stray light for improved
image resolution
Secretes growth and survival
factors needed for photoreceptor
differentiation
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Retinal Pigment Epithelium: Vital for Photoreceptor Health
12. Failure of RPE Causes Macular Degeneration
Regardless of Etiology
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Treatment of macular degeneration with Ocata Therapeuticsâ terminally differentiated retinal
pigment epithelial cells uniquely poised to restore visual function
Disease Pathophysiology Prevalence
Stargardtâs Macular
Degeneration (SMD)
Genetic
(e.g. ABCA4 gene mutation)
US/EU: 80k-100k
Age-related Macular
Degeneration (AMD)
Environmental/Genetic
(e.g. smoking, obesity) and
genetic (e.g. Y402H mutation in
complement factor H)
US/EU
Wet: 7.2m-11m
Dry: 61m-65m
Myopic Macular
Degeneration (MMD)
Environmental/Genetic
(e.g. family history and race)
US/EU: 0.7m-1.3m
Asia: 7.2-13.2m
16. Thesis of RPE Transplantation: Restoration of Anatomy &
Function
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Neural signal restored
RPE transplatation
17. D. Black dashed circle outlining area of subretinal transplantation
E. Green rectangle overlying white dashed arrow demonstrating optical coherence tomographic section (OCT)
at baseline and at 6 months following subretinal MA09-hRPE injection
F. White arrows demonstrating persistence of subretinal pigment epithelial cells 12 months post-transplantation
Phase 1/2 Trials: Evidence of Engraftment (Lancet 2014)
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Baseline* Month 6*
Anatomic Evidence of Successful Engraftment in 13/18 Subjects
21. SMD: BCVA Improved and Sustained At 1 Year
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Signal of efficacy in treated eyes & lack of improvement in untreated eyes
*3 subjects excluded due to cataract formation; 1 subject
excluded due to <6 months follow-up
December 2014May 2014 - The Lancet publication
*5 subjects excluded due to cataract formation; 1 subject
excluded due to <6 months follow-up
0.0 0.4
2.6
4.8 5.2
0.0
5.0
10.0
15.0
20.0
0 31 60 91 121 152 182 213 244 274 305 335
MeanChange(+/-SEM)from
BaselineinBCVA(letters)
Days After Transplant
Treated Eye Untreated Eye Difference Between Eyes
0.0
2.0
3.5 3.5
6.0 6.5
0.0
5.0
10.0
15.0
20.0
0 30 60 90 120 150 180 210 240 270 300 330 360
MeanChange(+/-SEM)from
BaselineinBCVA(letters)
Days After Transplant
4 Subjects with 12 Months Follow-up*
Treated Eye Fellow Eye Difference Between Eyes
5 Subjects with 12 Months Follow-up*
22. 38 subjects treated to date, 6 with more than 3 years of exposure
Adverse Events
â Systemic: observed most frequently in AMD population and in apparent association with
systemic immunosuppressive therapy
â Ocular adverse events consistent with vitreoretinal surgery
â No evidence of adverse proliferation, serious adverse ocular events or systemic safety
issues related to the transplanted tissue
Visual acuity data indicate either stability or increased vision in most subjects;
loss of vision observed in one subject attributed to cataract development
Safety Summary: AMD & SMD Interim Data
Overall, subretinal transplantation with hRPE was tolerated well
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23. ď Primary objective: evaluate the safety of 3 regimens
of short-term, low dose, systemic immunosuppression
therapy (IMT) as rejection prophylaxis prior to and/or
following transplant of RPE cells
ď Eligible patients will have geographic atrophy (GA)
secondary to AMD
ď Overall study duration is anticipated to be
approximately 47 months (from enrollment of the first
subject to the last study visit in the extension phase)
ď An independent Data Safety Monitoring Board will
periodically monitor safety
AMD Phase 2: Overview of Study Design
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The study has a
planned enrollment of
60 subjects at up to 8
sites in North America
24. AMD Phase 2: Study Design Schematic
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Approximately
60 subjects to
be treated
Transplantation of 200,000
RPE cells in study eye
only 1 eye treated per
subject
(n=45)
TREATMENT GROUP
Sham transplantation
surgery
(n=15)
CONTROL GROUP
Cohort 1: severe vision loss (n=15)
Day
-5
Day
-21
Baseline
period
Day 0
Surgery
Day
-65
Day
-51
Screening
period
Day
1
Month
18
Post-surgical treatment and follow-up period
Cohort 2: moderate vision loss (n=15)
Cohort 3: moderate vision loss (n=15)
Cohort 1: severe vision loss (n=5)
Cohort 2: moderate vision loss (n=5)
Cohort 3: moderate vision loss (n=5)
25. Initiating Phase 2 with novel, potentially curative therapy in areas
where no approved products exist today
Phase 1
â Safety observed, in addition to anatomical evidence of engraftment
â Visual acuity data indicate either stability or increased vision in most subjects; loss
of vision observed in one subject attributed to cataract development
â No evidence of adverse proliferation, serious adverse ocular events or systemic
safety issues related to the transplanted tissue
â Systemic: observed most frequently in AMD population and in apparent association
with systemic immunosuppressive therapy
Data published in The Lancet, October 14, 2014
Established IP position in major markets protecting the life span of the
cell therapy â from the origin of the cell to the delivery into patientsâ
eyes
The World Leader in Regenerative Ophthalmology
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