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Corporate Presentation 
November 2014
Cautionary Statement Concerni ng Forward-Looking Statements 
This presentation is intended to present a summary of Ocata’s (“Ocata”, or “Ocata 
Therapeutics, 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. 
2
Ophthalmology Opportunities are Attractive 
$12 Billion US Market and Growing 
! Ocata's Regenerative Ophthalmology™ Research is Poised to Deliver 
Transformative New Therapies for Macular Degeneration and Other High 
Medical Need Diseases of the Eye 
! Stargardt’s Macular Degeneration (SMD) is an orphan disease with no 
available treatment 
– Leading form of inherited juvenile onset macular degeneration that leads to blindness 
! Approximately 15m people in the US have age related macular degeneration 
(AMD); ~90% have dry AMD 
– There is no current treatment to prevent, effectively treat or cure dry AMD 
! Myopic Macular Degeneration (MMD) 
– Prevalent condition especially in Asia affecting at least 18m in China and Japan 
3
Ocata Therapeutics Provides the First Long-term Evidence of Safety and Signs 
of Improvement in Vision with Transplantation of RPE Cells 
4 
“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
The Regenerative Ophthalmology™ Company 
Addressing Macular Degeneration with Groundbreaking RPE Therapy 
5 
Safety observed reassuring, 
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 
Initiating Phase 2 studies 
soon: 
• Stargardt’s Macular 
Degeneration (SMD) 
- Q4 2014 
• Dry Age-related Macular 
Degeneration (AMD) 
- Q2 2015 
Extensive proprietary position 
in major markets protecting 
the entire value chain of the 
cell therapy – from the origin 
of the stem cell to the delivery 
into patients’ eyes
Stem Cells are the Starting Material but 
Fully Differentiated Cells are the Treatment 
6 
Pluripotent 
Stem Cells 
Starting Source 
hESC 
iPSC 
Corneal 
Retinal Ganglion 
Photoreceptor 
Terminally 
Differentiated 
Cells For 
Transplantation 
Retinal Pigment Epithelium 
World Leaders in Terminal Differentiation of Pluripotent to Target Cells 
Induce pluripotent stem cells into terminally differentiated cells to become the target tissue, such as RPE
RPE Damage and Subsequent Photoreceptor Degeneration Leads 
to Loss of Central Visual Acuity 
Patients 
suffering from 
dry AMD 
and SMD 
have no effective 
treatment 
options 
7
Retinal Pigment Epithelium is Vital for Photoreceptor Health 
Failure of RPE Causes Macular Degeneration 
Disease Pathophysiology Prevalence 
Stargardt’s Macular 
Degeneration (SMD) 
Genetic 
(e.g. ABCA4 gene mutation) US/EU: 80k-100k 
Dry 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: 4.1m-16.4m 
Asia: 18m-47m 
Ocata's Proprietary RPE Transplant Therapy Addresses These Forms of Macular Degeneration 
Targeting Orphan Disease (SMD) and Potential Blockbuster Markets (e.g. Dry AMD and MMD) 
8
Ophthalmic Development Pipeline Includes SMD, Dry AMD and MMD 
Multiple Opportunities for Product Development and Commercialization 
Pre-clinical 
IND Phase 1 Phase 2 
SMD 
Dry AMD 
MMD 
Photoreceptors 
Ganglion Cells 
Cornea 
Phase 2 interim 
data, mid-2016 for 
RPE Programs 
Opportunity for 
additional IND’s 
Potential European launch for Stargardt’s Macular Degeneration in 2018/2019 
9
The Eye is Well-Suited for Cellular Transplantation 
! 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 
10
RPE Market Dynamics for Stargardt’s Macular Degeneration 
Orphan Disease with an Estimated Prevalence of 1:10,000 (~31,600 in the US) 
Three main 
subtypes of 
patients that 
physicians 
observe 
Fast deterioration 
Slow deterioration 
Rod and cone dystrophy 
Stargardt’s patients are 
primarily diagnosed and 
managed by retinal specialists 
60-70% of total Stargardt’s population: symptoms present 
early on and progress from 20/40 to 20/200 (5-10 years) 
5%-10% of total Stargardt's population: could potentially 
benefit from aggressive treatment 
Pipeline limited to a few 
product candidates in early 
stages of development: 
• StarGen (Oxford Biomedica) 
• ALK-001 (Alkeus) 
• Emixustat (Acucela) 
There are no approved 
therapies that slow or stop the 
progression of Stargardt’s 
disease; almost all patients 
develop 20/200 vision or worse 
and are managed currently 
with supportive therapies 
11
Market Dynamics Age-related Macular Degeneration 
~1.8m Dry AMD Patients Diagnosed Every Year in the USA 
In the late form of dry AMD, patients can progress to geographic atrophy (GA) – GA progresses slowly and 
leads to the death of the photoreceptors. 
Approximately 110,000-165,000 Cases of Advanced Dry AMD are diagnosed every year with central GA – our 
primary target indication. 
Severe Dry AMD is a precursor to Wet AMD – Wet AMD is a blockbuster drug category e.g. Lucentis (Roche/ 
Novartis) and Eylea (Regeneron) 
Dry AMD patients are 
primarily managed by general 
ophthalmologists who are 
referred to retinal specialists 
when there are risks for wet 
AMD or severe symptoms 
Pipeline indicative of unmet 
need and commercial 
opportunity includes: NT-501 
(Neurotech), emixustat 
(Acucela, Lampalizumab 
from (Roche) and MC-1101 
from MacuCLEAR 
There are no approved 
therapies that slow or stop the 
progression of AMD; almost all 
patients develop 20/100 or 
20/200 vision or worse and 
can only be managed with 
supportive therapies 
12
SMD, Dry AMD and MMD are Specialized Opportunities, 
and Feasible for an Emerging Biotech 
! Well-defined prescriber base 
– Patients are referred to retinal specialists (~2,500 in the US of which ~1,500- 2,000 are surgical RS) 
who diagnose and manage subsequent patient care 
– Vitreoretinal surgeons will be responsible for the majority of procedures 
– Physician outreach can be readily achieved with a relatively small but specialized technical 
salesforce supported by targeted marketing efforts 
! Ease of administration: the transplanted cells are delivered via pars plana vitrectomy 
– P1 trial being run by KOLs is scalable to all retinal surgeons 
! Small dosage required for treatment 
– Commercial scalability of manufacturing and distribution in process 
! Significant unmet medical need – no approved treatments for dry AMD or SMD 
– Opportunity to treat earlier stage disease 
13
Clinical Programs 
RPE for SMD, Dry AMD and MMD 
14
Retinal Pigment Epithelium: Vital for Photoreceptor Health 
! 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 
15
Anatomica l Overview 
16
Macular Degene rative Diseases 
17 
Normal 
Dry AMD SMD 
MMD
Visual Physio logy: Normal 
18
Visual Physiology: M acular Degeneration 
19
RPE Trans plantation 
20
Thesis of RPE Transplantation: R estoration of Anatomy & Function 
21 
Neural signal restored
Phase 1/2 Study Design: 
38 Patients Treated, Safety Well Characterized 
! Open-label, non-randomized, sequential 
multi-center trial 
! 4 dose cohorts 
– Poor Vision 
■ Initially HM -> not better than 20/400; given 
safety, FDA supportive of treating not better 
than 20/100 
– Sequential enrollment of dose groups 
– Independent DSMB review of safety 
■ First subject in cohort: 6 weeks after 
transplantation 
■ Subsequent review: 4 weeks after completion 
of 3-subject cohort prior to dose increase 
22
Phase 1 Trials: Evidence of Engraftment 
Patches of increasing 
subretinal, pigmented 
cellular tissue consistent 
with transplanted, 
integrated RPE were 
observed in most 
subjects 
Images of transplanted 
tissue (autofluorescence, 
optical coherence 
tomography) suggest 
physiologic polarization 
and function of 
transplanted RPE 
23 
*Subject 106 
Baseline* Month 6* 
Anatomic Evidence of Successful Engraftment in 13/18 Subjects
Demographics of 18 Patients St udied in The Lancet Publication 
24 
SMD Dry AMD 
N 9 9 
Age 
median (range) 50 (20-71) 70 (70-88) 
Race 8 Caucasian; 1 Black 9 Caucasian 
Female 
n (%) 5 (56%) 6 (67%) 
Baseline Ocular 
Characteristics Study Eye Fellow Eye Study Eye Fellow Eye 
BCVA (ETDRS Letters) 
median (range) 5 (0-22) 6 (0-30) 21 (4-29) 62 (22-96)
BCVA Improved in Both Patient Populations and was Sustained 
25 
SMD (N=5) 
0 
1 1 
5 
9 
0 31 91 182 366 
25 
20 
15 
10 
5 
0 
-5 
Dry AMD (N=8) 
0 
6.5 5.5 
11.5 13 
0 31 91 182 366 
Days After Transplant 
Treated Eye Untreated Eye Difference Between Eyes 
25 
20 
15 
10 
5 
0 
-5 
Success in Phase 1/2 Studies Supports Investment in Proof-of-Concept Trials 
Median Change in Best Corrected 
Visual Acuity (Letters)
Translating Phase 1 Success in to an Optimized Phase 2 Plan 
26 
SMD Phase 2 Dry AMD Phase 2 MMD Phase 1 
N 100 50 12 
Initiation December 2014 June 2015 April 2015 
Objective 
• Efficacy and safety 
• Assessment of relevant functional 
and anatomic parameters 
• Safety evaluation of systemic 
immunosuppressive regimens 
• Exploration of efficacy 
parameters 
• Safety and exploration of efficacy 
parameters 
Endpoint(s) 
• Change from month 1 to 12 in 
average best corrected visual 
acuity (BCVA) OR 
• Rate of change in area of atrophy 
from baseline to 18 months post-transplant 
• Change from month 1 to 12 in 
average best corrected visual 
acuity (BCVA) 
• Rate of change in area of atrophy 
from baseline to 18 months post-transplant 
• Safety and tolerability of 
transplantation of hRPE cells as 
therapy in subjects with MMD 
Design 
• Phase 2, open-label, fellow-eye 
controlled, dose-escalation, multi-center 
trial 
• Phase 2, open-label, fellow-eye 
controlled, multi-center trial 
• Phase 1, open-label, dose-escalation, 
single-center trial 
Duration 
• Enrollment: ~18 months 
• Pre-specified interim analysis 
planned at months 6 and 12 
• Enrollment: ~12 months 
• Ongoing analysis of data 
• Enrollment: ~12 months 
• Full readout by end of 2016
Cells are Transplanted via Common Surgical Technique 
! Vitrectomy: Commonly performed outpatient procedure (~300,000/annually) by 
approximately 2,000 surgeons in the US 
– Sub-retinal administration is a ~90 second “add-on” 
– Builds on commonly practiced eye procedures and referral patterns 
27
Additional Cell Types in Development to Address a Spectrum of Ocular Disorders 
1 2 
3 4 
28 
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
Operations, Finance and Corporate 
29
Ocata Therapeutics has Invested Heavily to Own Manufacturing Process 
in cGMP Environment 
Cryopreserved – inexhaustible replicative capacity starting 
material, stored at ACT and in remote location 
Step 
Master Cell Bank of 
hESC’s 
Induction of proliferation and Expansion of cells cell culture expansion 
~20 fold 
In-process assays to ensure morphology and 
sterility of cells 
Shift to Terminal 
Differentiation 
to RPE cells 
Each re-passage step 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 
Continued Investment and Advances in Manufacturing and Delivery Generates Expansion of IP Estate 
30
IP Coverage From Stem Ce ll Line to Patient Treatment 
Single Blastomere Derivation of hESCs Methods of Manufacturing hESC-derived RPE cells 
3 Patent Families 
• 13 Issued Patents 
• 26 Pending Applications 
Core Patent expiry – 2031 (with Patent Term Extension) 
Methods-of-Treatment Pharmaceutical Preparations Product Release Assays 
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 
31
• Trades on OTC – OCAT 
• September 30, 2014 cash balance - 
$7.8m 
• Currently funded from $30m equity line 
with Lincoln Park Capital, of which ~ 
$20m remains 
• ~35m shares outstanding (fully diluted) 
– no preferred stock 
– no debt 
• Active Dialogue with Nasdaq 
32 
Ocata Therapeutics Financial Overview
Potential Milestones: Next 18 – 24 Months 
Year Quarter Milestone 
2014 Q4 • SMD P2: First subject treated 
• Up-listing of company stock to Nasdaq 
2015 
Q1 • Advanced Therapy Medicinal Product Designation 
• Publication of data in Asian patients (SMD & AMD) 
Q2 
• AMD P2: First subject treated 
• MMD P1: First subject treated 
• Partnership of non-core asset (e.g. platelet program) 
2016 
Q2 • MMD P2: 50% of subjects with 6-month data 
Q3 • SMD P2: 50 subjects with 6-month data 
• AMD P2: 50 subjects with 6-month data 
Q4 • MMD P1: 100% with 12-month data 
33
The World Leader in Regenerative Ophthalmology 
! Initiating Phase 2 with novel, potentially curative therapy in areas where no 
approved products exist today (Dry AMD/SMD) 
! 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 14, 2014 
! Leading 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 
34

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Corporate presentation-20141120

  • 2. Cautionary Statement Concerni ng Forward-Looking Statements This presentation is intended to present a summary of Ocata’s (“Ocata”, or “Ocata Therapeutics, 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. 2
  • 3. Ophthalmology Opportunities are Attractive $12 Billion US Market and Growing ! Ocata's Regenerative Ophthalmology™ Research is Poised to Deliver Transformative New Therapies for Macular Degeneration and Other High Medical Need Diseases of the Eye ! Stargardt’s Macular Degeneration (SMD) is an orphan disease with no available treatment – Leading form of inherited juvenile onset macular degeneration that leads to blindness ! Approximately 15m people in the US have age related macular degeneration (AMD); ~90% have dry AMD – There is no current treatment to prevent, effectively treat or cure dry AMD ! Myopic Macular Degeneration (MMD) – Prevalent condition especially in Asia affecting at least 18m in China and Japan 3
  • 4. Ocata Therapeutics Provides the First Long-term Evidence of Safety and Signs of Improvement in Vision with Transplantation of RPE Cells 4 “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
  • 5. The Regenerative Ophthalmology™ Company Addressing Macular Degeneration with Groundbreaking RPE Therapy 5 Safety observed reassuring, 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 Initiating Phase 2 studies soon: • Stargardt’s Macular Degeneration (SMD) - Q4 2014 • Dry Age-related Macular Degeneration (AMD) - Q2 2015 Extensive proprietary position in major markets protecting the entire value chain of the cell therapy – from the origin of the stem cell to the delivery into patients’ eyes
  • 6. Stem Cells are the Starting Material but Fully Differentiated Cells are the Treatment 6 Pluripotent Stem Cells Starting Source hESC iPSC Corneal Retinal Ganglion Photoreceptor Terminally Differentiated Cells For Transplantation Retinal Pigment Epithelium World Leaders in Terminal Differentiation of Pluripotent to Target Cells Induce pluripotent stem cells into terminally differentiated cells to become the target tissue, such as RPE
  • 7. RPE Damage and Subsequent Photoreceptor Degeneration Leads to Loss of Central Visual Acuity Patients suffering from dry AMD and SMD have no effective treatment options 7
  • 8. Retinal Pigment Epithelium is Vital for Photoreceptor Health Failure of RPE Causes Macular Degeneration Disease Pathophysiology Prevalence Stargardt’s Macular Degeneration (SMD) Genetic (e.g. ABCA4 gene mutation) US/EU: 80k-100k Dry 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: 4.1m-16.4m Asia: 18m-47m Ocata's Proprietary RPE Transplant Therapy Addresses These Forms of Macular Degeneration Targeting Orphan Disease (SMD) and Potential Blockbuster Markets (e.g. Dry AMD and MMD) 8
  • 9. Ophthalmic Development Pipeline Includes SMD, Dry AMD and MMD Multiple Opportunities for Product Development and Commercialization Pre-clinical IND Phase 1 Phase 2 SMD Dry AMD MMD Photoreceptors Ganglion Cells Cornea Phase 2 interim data, mid-2016 for RPE Programs Opportunity for additional IND’s Potential European launch for Stargardt’s Macular Degeneration in 2018/2019 9
  • 10. The Eye is Well-Suited for Cellular Transplantation ! 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 10
  • 11. RPE Market Dynamics for Stargardt’s Macular Degeneration Orphan Disease with an Estimated Prevalence of 1:10,000 (~31,600 in the US) Three main subtypes of patients that physicians observe Fast deterioration Slow deterioration Rod and cone dystrophy Stargardt’s patients are primarily diagnosed and managed by retinal specialists 60-70% of total Stargardt’s population: symptoms present early on and progress from 20/40 to 20/200 (5-10 years) 5%-10% of total Stargardt's population: could potentially benefit from aggressive treatment Pipeline limited to a few product candidates in early stages of development: • StarGen (Oxford Biomedica) • ALK-001 (Alkeus) • Emixustat (Acucela) There are no approved therapies that slow or stop the progression of Stargardt’s disease; almost all patients develop 20/200 vision or worse and are managed currently with supportive therapies 11
  • 12. Market Dynamics Age-related Macular Degeneration ~1.8m Dry AMD Patients Diagnosed Every Year in the USA In the late form of dry AMD, patients can progress to geographic atrophy (GA) – GA progresses slowly and leads to the death of the photoreceptors. Approximately 110,000-165,000 Cases of Advanced Dry AMD are diagnosed every year with central GA – our primary target indication. Severe Dry AMD is a precursor to Wet AMD – Wet AMD is a blockbuster drug category e.g. Lucentis (Roche/ Novartis) and Eylea (Regeneron) Dry AMD patients are primarily managed by general ophthalmologists who are referred to retinal specialists when there are risks for wet AMD or severe symptoms Pipeline indicative of unmet need and commercial opportunity includes: NT-501 (Neurotech), emixustat (Acucela, Lampalizumab from (Roche) and MC-1101 from MacuCLEAR There are no approved therapies that slow or stop the progression of AMD; almost all patients develop 20/100 or 20/200 vision or worse and can only be managed with supportive therapies 12
  • 13. SMD, Dry AMD and MMD are Specialized Opportunities, and Feasible for an Emerging Biotech ! Well-defined prescriber base – Patients are referred to retinal specialists (~2,500 in the US of which ~1,500- 2,000 are surgical RS) who diagnose and manage subsequent patient care – Vitreoretinal surgeons will be responsible for the majority of procedures – Physician outreach can be readily achieved with a relatively small but specialized technical salesforce supported by targeted marketing efforts ! Ease of administration: the transplanted cells are delivered via pars plana vitrectomy – P1 trial being run by KOLs is scalable to all retinal surgeons ! Small dosage required for treatment – Commercial scalability of manufacturing and distribution in process ! Significant unmet medical need – no approved treatments for dry AMD or SMD – Opportunity to treat earlier stage disease 13
  • 14. Clinical Programs RPE for SMD, Dry AMD and MMD 14
  • 15. Retinal Pigment Epithelium: Vital for Photoreceptor Health ! 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 15
  • 17. Macular Degene rative Diseases 17 Normal Dry AMD SMD MMD
  • 18. Visual Physio logy: Normal 18
  • 19. Visual Physiology: M acular Degeneration 19
  • 21. Thesis of RPE Transplantation: R estoration of Anatomy & Function 21 Neural signal restored
  • 22. Phase 1/2 Study Design: 38 Patients Treated, Safety Well Characterized ! Open-label, non-randomized, sequential multi-center trial ! 4 dose cohorts – Poor Vision ■ Initially HM -> not better than 20/400; given safety, FDA supportive of treating not better than 20/100 – Sequential enrollment of dose groups – Independent DSMB review of safety ■ First subject in cohort: 6 weeks after transplantation ■ Subsequent review: 4 weeks after completion of 3-subject cohort prior to dose increase 22
  • 23. Phase 1 Trials: Evidence of Engraftment Patches of increasing subretinal, pigmented cellular tissue consistent with transplanted, integrated RPE were observed in most subjects Images of transplanted tissue (autofluorescence, optical coherence tomography) suggest physiologic polarization and function of transplanted RPE 23 *Subject 106 Baseline* Month 6* Anatomic Evidence of Successful Engraftment in 13/18 Subjects
  • 24. Demographics of 18 Patients St udied in The Lancet Publication 24 SMD Dry AMD N 9 9 Age median (range) 50 (20-71) 70 (70-88) Race 8 Caucasian; 1 Black 9 Caucasian Female n (%) 5 (56%) 6 (67%) Baseline Ocular Characteristics Study Eye Fellow Eye Study Eye Fellow Eye BCVA (ETDRS Letters) median (range) 5 (0-22) 6 (0-30) 21 (4-29) 62 (22-96)
  • 25. BCVA Improved in Both Patient Populations and was Sustained 25 SMD (N=5) 0 1 1 5 9 0 31 91 182 366 25 20 15 10 5 0 -5 Dry AMD (N=8) 0 6.5 5.5 11.5 13 0 31 91 182 366 Days After Transplant Treated Eye Untreated Eye Difference Between Eyes 25 20 15 10 5 0 -5 Success in Phase 1/2 Studies Supports Investment in Proof-of-Concept Trials Median Change in Best Corrected Visual Acuity (Letters)
  • 26. Translating Phase 1 Success in to an Optimized Phase 2 Plan 26 SMD Phase 2 Dry AMD Phase 2 MMD Phase 1 N 100 50 12 Initiation December 2014 June 2015 April 2015 Objective • Efficacy and safety • Assessment of relevant functional and anatomic parameters • Safety evaluation of systemic immunosuppressive regimens • Exploration of efficacy parameters • Safety and exploration of efficacy parameters Endpoint(s) • Change from month 1 to 12 in average best corrected visual acuity (BCVA) OR • Rate of change in area of atrophy from baseline to 18 months post-transplant • Change from month 1 to 12 in average best corrected visual acuity (BCVA) • Rate of change in area of atrophy from baseline to 18 months post-transplant • Safety and tolerability of transplantation of hRPE cells as therapy in subjects with MMD Design • Phase 2, open-label, fellow-eye controlled, dose-escalation, multi-center trial • Phase 2, open-label, fellow-eye controlled, multi-center trial • Phase 1, open-label, dose-escalation, single-center trial Duration • Enrollment: ~18 months • Pre-specified interim analysis planned at months 6 and 12 • Enrollment: ~12 months • Ongoing analysis of data • Enrollment: ~12 months • Full readout by end of 2016
  • 27. Cells are Transplanted via Common Surgical Technique ! Vitrectomy: Commonly performed outpatient procedure (~300,000/annually) by approximately 2,000 surgeons in the US – Sub-retinal administration is a ~90 second “add-on” – Builds on commonly practiced eye procedures and referral patterns 27
  • 28. Additional Cell Types in Development to Address a Spectrum of Ocular Disorders 1 2 3 4 28 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
  • 29. Operations, Finance and Corporate 29
  • 30. Ocata Therapeutics has Invested Heavily to Own Manufacturing Process in cGMP Environment Cryopreserved – inexhaustible replicative capacity starting material, stored at ACT and in remote location Step Master Cell Bank of hESC’s Induction of proliferation and Expansion of cells cell culture expansion ~20 fold In-process assays to ensure morphology and sterility of cells Shift to Terminal Differentiation to RPE cells Each re-passage step 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 Continued Investment and Advances in Manufacturing and Delivery Generates Expansion of IP Estate 30
  • 31. IP Coverage From Stem Ce ll Line to Patient Treatment Single Blastomere Derivation of hESCs Methods of Manufacturing hESC-derived RPE cells 3 Patent Families • 13 Issued Patents • 26 Pending Applications Core Patent expiry – 2031 (with Patent Term Extension) Methods-of-Treatment Pharmaceutical Preparations Product Release Assays 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 31
  • 32. • Trades on OTC – OCAT • September 30, 2014 cash balance - $7.8m • Currently funded from $30m equity line with Lincoln Park Capital, of which ~ $20m remains • ~35m shares outstanding (fully diluted) – no preferred stock – no debt • Active Dialogue with Nasdaq 32 Ocata Therapeutics Financial Overview
  • 33. Potential Milestones: Next 18 – 24 Months Year Quarter Milestone 2014 Q4 • SMD P2: First subject treated • Up-listing of company stock to Nasdaq 2015 Q1 • Advanced Therapy Medicinal Product Designation • Publication of data in Asian patients (SMD & AMD) Q2 • AMD P2: First subject treated • MMD P1: First subject treated • Partnership of non-core asset (e.g. platelet program) 2016 Q2 • MMD P2: 50% of subjects with 6-month data Q3 • SMD P2: 50 subjects with 6-month data • AMD P2: 50 subjects with 6-month data Q4 • MMD P1: 100% with 12-month data 33
  • 34. The World Leader in Regenerative Ophthalmology ! Initiating Phase 2 with novel, potentially curative therapy in areas where no approved products exist today (Dry AMD/SMD) ! 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 14, 2014 ! Leading 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 34