Ocata Therapeutics Corporate Presentation June 2015

1. Ocata Therapeutics Corporate Presentation
June 2015

2. Cautionary Statement Concerning Forward-Looking Statements
Ocata Therapeutics Inc. (“Ocata” or “the Company”) has filed a registration statement (including a prospectus and a preliminary
prospectus supplement) with the Securities and Exchange Commission (“SEC”) 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 SEC’s 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 “anticipate,” “believe,”
“could,” “continue,” “estimate,” “expect,” “intend,” “may,” “should,” “will”, “would,” ”plan,” ”projected,” 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 Annual Report on Form 10-K, as amended, and our most recently filed Quarterly 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 wit the SEC.
2

3. Addressing Macular Degeneration with Groundbreaking RPE Therapy
The Regenerative Ophthalmology™ Company
Safety observed, in addition
to anatomical and functional
evidence of repair and
restoration in Phase 1 studies
for dry AMD and SMD
• Data published in The Lancet,
October 14, 2014
• Data in Asian patients published
in Stem Cells, April 30, 2015
Initiating Pivotal and Phase 2
studies:
• Stargardt’s Macular
Degeneration (SMD) – Pivotal
- H2 2015
• Dry Age-related Macular
Degeneration (AMD) – Phase 2
- Q3 2015
Addressing Macular
Degeneration with two
product candidates:
• An orphan indication in
SMD, followed by a
• Potential blockbuster in
AMD, based on patient
data in the US and EU
• ATMP status in Europe
Organ Transplantation is Well-Established, Our RPE Products are Simple Micro Transplants of
Support Cells required for Functioning Infrastructure at the Back of the Eye
3

4. Recent Milestones Demonstrate Continued Execution Against Plan
Milestone Significance
• Completion of P1/2 studies at highest dose (200k cells)
• SMD pivotal trial protocol agreed with EMA
• Up-listing of company stock to NASDAQ
• Publication of data in Asian patients (SMD & AMD)
• Expanded technology to include IPSCs via Allele Deal
• Three US patents issued covering RPE Program
• Safety in 44 patients, some with >3 years of follow up
• Confidence in commercial timing, 2019
• Increased liquidity, index funds and broader investor audience
• Supportive of US/UK trials, independently managed trial
• Bolsters pre-clinical pipeline and diversifies platform
• Potential to block all current competitors
Milestones in 1H 2015 Position Company for Success in Next 12 – 18 Months…
4

5. Future Milestones to Bolster Position as
The Leading Regenerative Ophthalmology Company
Year Period Goal
2015
Q3
• Dry AMD Phase 2 Study: First subject enrolled
• Special Protocol Assessment meeting with FDA
• SMD Pivotal Study: First subject enrolled
• Further strengthening of IP portfolio
Q4
• Publication of data on pre-clinical photoreceptor studies
• Partnership of non-core asset (e.g. platelet program)
2016
Q2 • Dry AMD P2 Study: First Cohort with 3 month data
Q3
• Dry AMD P2 Study: Second Cohort with 3 month data
• Partnership of core program in ex-US region (e.g. Asia, South America)
As Milestones are Achieved, Ocata will Continue to Broaden Market Awareness
5

6. RPE Damage and Subsequent Photoreceptor Degeneration Leads
to Loss of Central Visual Acuity
6

7. The First Evidence of Long-term Safety and Efficacy Signal Following
Transplantation of RPE Cells – October 2014
“What 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, eye specialist, UCLA
7

8. Further Evidence of Long-term Safety and Efficacy Signal in Asian Patients with
SMD and AMD – April 2015
Four Korean patients: two with dry AMD and two with SMD
Patients were followed for 1 year
– No evidence of adverse proliferation, tumorigenicity, ectopic tissue
formation, or other serious safety issues related to the transplanted cells.
– Visual acuity improved 9–19 letters in three patients and remained
stable (+1 letter) in one patient.
Total of 44 patients safely treated worldwide with RPE Therapy.
Independent trial in Asian population supports previously reported safety and efficacy signals
8

9. World Leaders in Terminal Differentiation of Pluripotent to Target Cells with
Proprietary DeltaCellTM Technology
Pluripotent
Stem Cells
Starting Source
Stem Cells are the Starting Material but Fully Differentiated Cells are the Treatment
hESC
iPSC
Corneal
Retinal Ganglion
Photoreceptor
Retinal Pigment Epithelium
Terminally
Differentiated
Cells For
Transplantation
9

10. 1
2
3 4
Addressing a Spectrum of Ocular Disorders with other Terminally Differentiated Cells
Photoreceptor Progenitor Cells
• Macular Degeneration -
dry AMD, SMD, MMD
• Retinitis Pigmentosa
Retinal Ganglion Progenitor Cells
• Glaucoma
Mesenchymal Stem Cells
• Uveitis
• Management of Ocular Surfaces
Corneal Endothelial Therapy
• Corneal Disease
1
2
3
4
Rich preclinical pipeline of Regenerative Ophthalmology product opportunities
each addressing large unmet medical needs
10

11. Ophthalmic Development Pipeline Includes SMD, Dry AMD and MMD
Pre-
clinical IND Phase 1 Phase 2
SMD
Dry AMD
MMD
Photoreceptors
Ganglion Cells
Cornea
Phase 2 Study
for Dry AMD,
Pivotal Trial for
SMD (Orphan)
Opportunity for
additional IND’s
Planned Commercial Launch for Stargardt’s Macular Degeneration in 2019
Multiple Opportunities for Product Development and Commercialization
11

12. Immune privileged
– Less prone to rejection
Compact Structure
– Relatively small doses required
to treat
Straightforward delivery using
currently available technology
Validated tools for clinical outcome
assessment
The Eye is Well-Suited for Cellular Transplantation
Patients in the Ocata Studies with more than Three Years Post Transplant
Experience, Continue to Show Positive Safety Signals with Visual Acuity Gains
12

13. Well-defined prescriber base
– Patients are referred to retinal specialists (~2,500 in the US of which ~1,500- 2,000 are vitreoretinal
surgeons) who diagnose and manage subsequent patient care
– Market penetration achievable with a small salesforce; ~50 reps in the US
Ease of administration: cellular transplantation performed with current technology
– Utilizes pars plana vitrectomy (more than one million procedures in the US already being completed, p.a.)
and subretinal injection (~90 seconds added to vitrectomy procedure) modalities scalable to surgeons
Small dosage requirement
– Commercial scalability of manufacturing and distribution in process
Significant unmet medical need – no approved treatments for dry AMD or SMD
– Approximately 1.8 million new dry-AMD patients diagnosed per year in the US
– Approximately 90,000 patients currently suffering from SMD in US and EU
SMD, Dry AMD and MMD are Specialized Opportunities
and Feasible for an Emerging Biotech
A Viable Standalone Plan Creates Leverage with Potential Partners
13

14. Clinical Programs
RPE for SMD, Dry AMD and MMD
14

15. Required for vision and maintenance of
photoreceptor health
Delivers and metabolizes Vitamin A
– 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
Retinal Pigment Epithelium: Vital for Photoreceptor Health
Loss of RPE Layer Results in Loss of Vision
Ocata’s Therapeutic Approach is to Transplant New RPE Layer and Help Restore Vision
15

16. Normal Physiology, RPE in Intimate Contact with Photoreceptors and
Provide Vital Life Support
16

17. Macular Degeneration Leads to RPE Loss
Photoreceptors incur Damage, Dormancy and eventual Death
17

18. Ocata’s Therapeutic Approach is to Deliver “Brand New” Terminally
Differentiated RPE Cells via Sub-Retinal Injection
18

19. Data Indicate Transplantation of New RPE Can Lead to Restoration of
Anatomy & Function
Neural signal restored
19

20. Macular Degeneration – Long Term
Data in SMD and Dry AMD
20

21. D. Black dashed circle outlining area of subretinal transplantation
E. Green rectangle overlying white dashed arrow demonstrating optical coherence tomographic section 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 Studies Presented Evidence of Engraftment;
Transplanted Cells Take Residence, as Intended
Baseline* Month 6*
Anatomic Evidence of Successful Engraftment Illustrates that Transplant is
Rebuilding the Support Structure in the Back of the Eye
21

22. Interim Data for AMD and SMD Lancet Study Showed that BCVA Improved
and was Sustained Over the Long-Term
Efficacy Signal and Safety Persists in Treated Eyes
Lancet publication: May 2014
Dry AMD
0.0
17.9
17.0 16.3
14.7
0.0
7.6 8.0
2.4
0.8
-5.0
0.0
5.0
10.0
15.0
20.0
25.0
0 31 60 91 121 152 182 213 244 274 305 335 366
MeanChangefromBaseline(letters)
Days after transplant
8 Subjects with 12 Month Follow-up
Treated Eye Untreated Eye
0.0
5.8
7.8
9.0
9.8
0.0
5.4 5.2
4.2 4.6
0.0
5.0
10.0
15.0
0 31 60 91 121 152 182 213 244 274 305 335
MeanChangefromBaselineinBCVA(letters)
Days After Transplant
5 Subjects with 12 Months Follow-up
Treated Eye Untreated Eye
Lancet publication: May 2014
SMD
22

23. Available Interim Data for AMD Study Showed that BCVA Improved
and was Sustained Over Two Years
Efficacy Signal and Safety Persists in Treated Eyes for Two Years
Now Moving to Controlled Phase 2 Study
March 2015
Dry AMD Data
0.0
6.2
5.4 5.2
6.0
7.2
8.2
0.0
1.6
5.2
4.4
-0.4
-3.2
-1.2
-5.0
0.0
5.0
10.0
15.0
20.0
25.0
0 60 121 182 244 305 366 425 486 547 609 670 731
MeanChangeinBCCVA(letters)
Days After Transplant
5 Subjects with 24 Month Follow-up
Treated Eye Untreated Eye
23

24. Clinical Program Design
Pivotal SMD Trial and Phase 2 Dry AMD Trial
24

25. Objectives
– Pivotal Controlled Study to demonstrate safety and efficacy of RPE cell transplant therapy
– Designed to confirm efficacy signal seen in prior studies
– Will include untreated masked control group, consistent with EMA and FDA guidance
Design Highlights
– Double-masked study
– 1:1 Randomization (N=100, 50 treated : 50 control)
First patient treatment expected in 2H 2015 following SPA meeting with FDA, full enrollment
expected in 2017 (Anatomy and Efficacy)
SMD Pivotal Trial, Planned Initiation in the Second Half of 2015
Trial Designed to Enable Potential Approval in 2019
25

26. Objectives
– Safety & tolerability of 3 different immunosuppressive regimens (13,7 and 1 week(s))
– Note - No evidence of any transplant rejection to date in patients treated up to four years
– Exploring efficacy signal seen in previous open studies
– Will include a “better vision” arm
Design Highlights
– Control group of untreated patients
– Three cohorts of 20 patients
– 3:1 Randomization (N=60, 45 treated : 15 control)
First Cohort Data read out expected Q2 2016 ( Anatomy and Efficacy Signal)
AMD Phase 2 Safety & Proof-of-Concept, Planned Initiation - Q3 2015
Corporate
Success in this Controlled Study will support SMD Program and Further RPE Therapy as a
Transformational Therapy that Could Provide Benefit for Patients with SMD and Dry AMD
26

27. Pre-Clinical Pipeline
Photoreceptor Progenitors
27

28. Pre-Clinical Pipeline is Maturing - Ocata’s Technology Extends Beyond hESCs
and includes Induced Pluripotent Cells (iPSCs)
Photoreceptor Progenitors were transplanted into mice with
severe retinal degeneration
After 3 weeks, mice showed significant improvement with the
number of surviving cells in each animal strongly correlated
to magnitude of visual improvement
Similar efficacy using either human ESC or iPS cells
processed using our DeltaCell™ Technology
Pre-clinical Data Expected H2 2015, this Product could Compliment RPE Therapy
as Viable Potential Solution for End-Stage Disease
28

29. Systemic hESC-PhRPs Provide Neuroprotection in Mice and Rats
a-wave
(cones & rods) PhRP, 2 mo
PhRP, 1 mo
PBS, 1 mo
PBS, 2 mo
Prevent photoreceptor
degeneration in ELOVL4 mice
ONL→
PBS control
Intravitreal injection
missing OS (P90)
PhPR-treated
(rod OS/rhodopsin)
Preserve outer segments of
photoreceptor in RCS rats
Tail vein injection
Healthy ONL
Missing ONL
PhRPs rescue photoreceptors in RCS rats
Healthy ONL
Missing ONL
0
10
20
30
40
50
60
PBS
NoInjection
RA2month
Increased ONL thickness after 2 months
PBS Ø Tx
29

30. Operations and Corporate
30

31. Ocata Therapeutics DeltaCell™ Technology
Manufacturing Process in cGMP Environment
Continued Investment and Advances in Manufacturing and Delivery Generates Expansion of IP Estate
Cryopreserved – inexhaustible replicative capacity starting
material, stored at Ocata and in remote location
Master Cell Bank of
hESC’s
Induction of proliferation and cell culture expansion
Expansion of cells
~20 fold
In-process assays to ensure morphology and
sterility of cells
Shift to Terminal
Differentiation
to RPE cells
The process of re-passaging the cells can produce >1,000
fold increase of quantity of RPE cells
Purify RPE and
re-passage to
expand quantity
Available for patient dosing; one five month process
typically yields ~1,000 doses
Harvest bulk material
and cryopreserve
RPE inventory
12 full-time employees
dedicated to manufacturing,
quality control, quality
assurance & assay
development
FDA review of CMC accepting
of release criteria and
processes
Step
31

32. IP Coverage From Stem Cell Line to Patient Treatment
Single Blastomere Derivation of hESCs Methods of Manufacturing hESC-derived RPE cells
Product Release AssaysPharmaceutical PreparationsMethods-of-Treatment
3 Patent Families
• 13 Issued Patents
• 26 Pending Applications
Core Patent expiry – 2031 (with Patent Term Extension)
5 Patent Families
• 2 Issued Patents
• 34 Pending Applications
Core Patents - 2031
Formulation Improvements - 2032
Shipping Medium –2035
3 Patent Families
• 4 Issued Patents
• 26 Pending Applications
Core Patents - 2031
Improvements - 2032
2 Patent Family
• 11 Pending Applications
Expiry will begin 2031 & 2032
1 Patent Family
• 4 Issued Patents
• 12 Pending Applications
Expiry begins 2025
32

33. • Trades on Nasdaq – OCAT
• March 31, 2015 cash balance - $3.5m
• Currently funded from $30m equity line
with Lincoln Park Capital, of which
~$8m remains
• ~35.6m shares outstanding
– no preferred stock
– no debt
– ~2.8m options and RSU’s held by
management and directors
Ocata Therapeutics Financial Overview
33

34. Future Milestones to Bolster Position as
The Leading Regenerative Ophthalmology Company
Year Period Goal
2015
Q3
• Dry AMD Phase 2 Study: First subject enrolled
• Special Protocol Assessment meeting with FDA
• SMD Pivotal Study: First subject enrolled
• Further strengthening of IP portfolio
Q4
• Publication of data on pre-clinical photoreceptor studies
• Partnership of non-core asset (e.g. platelet program)
2016
Q2 • Dry AMD P2 Study: First Cohort with 3 month data
Q3
• Dry AMD P2 Study: Second Cohort with 3 month data
• Partnership of core program in ex-US region (e.g. Asia, South America)
As Milestones are Achieved, Ocata will Continue to Broaden Market Awareness
34

35. An Experienced Management Team
Name Position Experience
Paul K. Wotton, Ph.D. President & CEO
Edward (Ted) Myles
Chief Operating Officer &
Chief Financial Officer
Robert Lanza, M.D. Chief Scientific Officer
Eddy Anglade, M.D. Chief Medical Officer
LeRoux Jooste Chief Commercial Officer
PENWEST

36. Experienced Corporate & Scientific Boards
Name Experience
Michael Heffernan,
(Chairman)
CEO – Collegium Pharmaceuticals
Robert Langer, Sc.D.
Institute Professor – Massachusetts Institute of
Technology
Greg Perry EVP & CFO – Eleven Biotherapeutics
Alan C. Shapiro
Finance Professor and Chairman, Department of
Finance and Business Economics (retired) –
University of Southern California
Zohar Loshitzer
President – Presbia, Inc., & Principal at Orchard
Capital
Paul K. Wotton, Ph.D. President and CEO, Ocata Therapeutics
World Class Scientific Advisory Board
Name Experience
Robert Langer, Sc.D.
(Chairman)
Massachusetts Institute of Technology
Queen Elisabeth Prize for Engineering (2015); Member
of all 3 National Academies; President’s National
Medal of Technology and Innovation (2013)
Constance Cepko, Ph.D.
Harvard Medical School
National Academy of Sciences (elected 2002); Alfred
W. Bressler Prize in Vision Science (2011)
George Daley, M.D., Ph.D.
Harvard Medical School
NIH Director’s Pioneer Award (2004); E. Mead Johnson
Award from the American Pediatric Society (2009)
John Gearhart, Ph.D.
University of Pennsylvania
Director of the Institute for Regenerative Medicine;
Science, Board of Reviewing Editors
Michael Longaker, M.D.
Stanford University
Director, Institute of Stem Cell Biology and
Regenerative Medicine; Director, Children's Surgical
Research
Joseph Vacanti, M.D.
Massachusetts General Hospital
Surgeon-in-Chief; Member, Institute of Medicine of the
National Academy of Sciences
Board of Directors – Broad Experience in Life Science Sector

37. Initiating Pivotal Trial for SMD and Phase 2 Study for dry AMD with novel, potentially
curative therapy in areas where no approved products exist today
Dry AMD is a potential blockbuster indication – a precursor to Wet AMD where
Treatments include Eylea (Regeneron) and Lucentis (Novartis/Roche)
Safety observed, in addition to anatomical and functional evidence of repair and
restoration; data published in The Lancet, October 14th, 2014 and data in Asian
Patients published on April 30th 2015 in Stem Cell Reports
Established and growing IP position in major markets protecting the life span of the
cell therapy – from the origin of the cell to the delivery into patients’ eyes
Novel and world leading pre-clinical pipeline
Ability to produce fully differentiated cell types (e.g. RPE, PhRP) with DeltaCell™
Technology
Experienced management team, corporate and scientific boards
The World Leader in Regenerative Ophthalmology
37