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Ocata Therapeutics Corporate Presentation June 2015
Cautionary Statement Concerning Forward-Looking StatementsOcata 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 mayget 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 mostrecently filed Annual Report on Form 10-K, as amended, and our most recently filed Quarterly Report on Form 10-Q, as filed withthe SEC. These forward-looking statements speak only as of the date on which the statements were made and are not guarantees offuture 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.
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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
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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…
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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)
2016Q2 • 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
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RPE Damage and Subsequent Photoreceptor Degeneration Leads to Loss of Central Visual Acuity
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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
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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 SMDPatients 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
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World Leaders in Terminal Differentiation of Pluripotent to Target Cells with Proprietary DeltaCellTM Technology
PluripotentStem 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
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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
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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 Studyfor 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
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Immune privileged– Less prone to rejectionCompact 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
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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 USEase 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 surgeonsSmall dosage requirement– Commercial scalability of manufacturing and distribution in processSignificant 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
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Clinical Programs RPE for SMD, Dry AMD and MMD
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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 VisionOcata’s Therapeutic Approach is to Transplant New RPE Layer and Help Restore Vision
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Normal Physiology, RPE in Intimate Contact with Photoreceptors and Provide Vital Life Support
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Macular Degeneration Leads to RPE Loss Photoreceptors incur Damage, Dormancy and eventual Death
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Ocata’s Therapeutic Approach is to Deliver “Brand New” Terminally Differentiated RPE Cells via Sub-Retinal Injection
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Data Indicate Transplantation of New RPE Can Lead to Restoration of Anatomy & Function
Neural signal restored
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Macular Degeneration – Long Term Data in SMD and Dry AMD
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D. Black dashed circle outlining area of subretinal transplantationE. Green rectangle overlying white dashed arrow demonstrating optical coherence tomographic section at baseline and at 6 months following subretinal MA09-hRPE injectionF. 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
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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 2014Dry AMD
0.0
17.9 17.0 16.314.7
0.0
7.6 8.0
2.40.8
-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
Mea
n C
hang
e fr
om B
asel
ine
(lette
rs)
Days after transplant
8 Subjects with 12 Month Follow-up
Treated Eye Untreated Eye
0.0
5.8
7.89.0
9.8
0.0
5.4 5.24.2 4.6
0.0
5.0
10.0
15.0
0 31 60 91 121 152 182 213 244 274 305 335Mea
n C
hang
e fro
m B
asel
ine
in B
CVA
(let
ters
)
Days After Transplant
5 Subjects with 12 Months Follow-up
Treated Eye Untreated Eye
Lancet publication: May 2014SMD
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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 YearsNow Moving to Controlled Phase 2 Study
March 2015 Dry AMD Data
0.0
6.2 5.4 5.2 6.07.2 8.2
0.01.6
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 731Mea
n C
hang
e in
BC
CVA
(let
ters
)
Days After Transplant
5 Subjects with 24 Month Follow-up
Treated Eye Untreated Eye
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Clinical Program Design Pivotal SMD Trial and Phase 2 Dry AMD Trial
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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
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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
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Pre-Clinical PipelinePhotoreceptor Progenitors
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Pre-Clinical Pipeline is Maturing - Ocata’s Technology Extends Beyond hESCsand 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
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Systemic hESC-PhRPs Provide Neuroprotection in Mice and Rats
a-wave(cones & rods) PhRP, 2 mo
PhRP, 1 moPBS, 1 moPBS, 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
No
Inje
ctio
nR
A 2
mon
th
Increased ONL thickness after 2 months
PBS Ø Tx
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Operations and Corporate
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Ocata Therapeutics DeltaCell™ TechnologyManufacturing 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 expansionExpansion 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
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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 - 2031Formulation Improvements - 2032Shipping Medium –2035
3 Patent Families• 4 Issued Patents• 26 Pending Applications
Core Patents - 2031Improvements - 2032
2 Patent Family• 11 Pending Applications
Expiry will begin 2031 & 2032
1 Patent Family• 4 Issued Patents• 12 Pending Applications
Expiry begins 2025
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• 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
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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)
2016Q2 • 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
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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
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. ShapiroFinance 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 TechnologyQueen 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 SchoolNational Academy of Sciences (elected 2002); Alfred W. Bressler Prize in Vision Science (2011)
George Daley, M.D., Ph.D.Harvard Medical SchoolNIH Director’s Pioneer Award (2004); E. Mead Johnson Award from the American Pediatric Society (2009)
John Gearhart, Ph.D.University of PennsylvaniaDirector of the Institute for Regenerative Medicine; Science, Board of Reviewing Editors
Michael Longaker, M.D.Stanford UniversityDirector, Institute of Stem Cell Biology and Regenerative Medicine; Director, Children's Surgical Research
Joseph Vacanti, M.D.Massachusetts General HospitalSurgeon-in-Chief; Member, Institute of Medicine of the National Academy of Sciences
Board of Directors – Broad Experience in Life Science Sector
Initiating Pivotal Trial for SMD and Phase 2 Study for dry AMD with novel, potentially curative therapy in areas where no approved products exist todayDry 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 ReportsEstablished 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’ eyesNovel and world leading pre-clinical pipelineAbility 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
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