ocata corp-presentation-september-2015-2-4

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September 2015 Corporate Presentation Ocata Therapeutics - NASDAQ OCAT

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Page 1: Ocata corp-presentation-september-2015-2-4

September 2015 Corporate Presentation Ocata Therapeutics - NASDAQ OCAT

Page 2: Ocata corp-presentation-september-2015-2-4

Cautionary Statement Concerning Forward-Looking Statements

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 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.

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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 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

• 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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2015 Milestones Demonstrate Execution Against Plan

Milestone Significance

• Completion of P1/2 Studies at highest dose (200k cells) • SMD Pivotal Trial 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 • Four new US patents issued covering RPE Program

• Completed $30 Million Financing

• Entered Russell 2000 Index

• Safety in 44 patients, some with up to 4 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 cell source

• Potential to block all current competitors

• Funded through next inflexion point

• Increased Visibility

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Milestones in 1H 2015 Position Company for Success in Next 12 – 18 Months…

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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

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“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 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 forty four patients safely treated worldwide with RPE Therapy. Independent trial in Asian population supports previously reported safety and efficacy signals

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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 Our Strategic Intent is to “Own the Eye”

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Patients in the Ocata Studies with more than Three Years Post Transplant Experience Continue to Show Good Safety with Visual Acuity Gains

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World Leaders in Terminal Differentiation of Pluripotent to Target Cells with Proprietary DeltaCell Technology

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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

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1 2

3 4

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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

Hemangioblast Mesenchymal Cells - HMC • 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 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

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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 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) 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 – 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

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A Viable Standalone Plan Creates Synergy 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

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Loss of RPE Layer Results in Loss of Vision Ocata’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

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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 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

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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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31 Patients Reported with at least 12 Months Follow Up – June 24’ 2015 26 Patients from US, 5 Patients from South Korea – Diverse Populations Some patients have up to 4 years follow up and safety profile is good No evidence of adverse proliferation, rejection, adverse ocular or systemic safety issues related to the transplanted RPE Cells Patches of increased and persistent sub-retinal pigmentation were observed indicating transplants have taken successfully Patients showed improved or stable vision over prolonged timeframe

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Updated Status – SMD and AMD Trials

Safety of treatment continues to be positive and efficacy signals are persistent over the long term

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Interim Data for AMD and SMD Lancet Study Showed that BCVA Improved and was Sustained Over the Long-Term

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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

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10.0

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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

7.8 9.0

9.8

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5.4 5.2 4.2 4.6

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5.0

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0 31 60 91 121 152 182 213 244 274 305 335Mea

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Days After Transplant

5 Subjects with 12 Months Follow-up

Treated Eye Untreated Eye

Lancet publication: May 2014 SMD

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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 included untreated masked control group, consistent with EMA and FDA guidance

Design Highlights – Double-masked study – 1:1 Randomization (N=100, 50 treated : 50 controls)

First patient treated in 2H 2015 following SPA meeting with FDA, full enrollment in 2017 (Anatomy and Efficacy)

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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, active : control) First Cohort Data read out Q1 2016 ( Anatomy and Efficacy Signal)

AMD Phase 2 Safety & Proof-of-Concept, Planned Initiation - Q3 2015

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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 Pipeline Photoreceptor Progenitors

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Pre-Clinical Pipeline is Maturing - Ocata’s Technology Extends Beyond hESC’s and includes Induced Pluripotent Cells (iPSC’s)

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 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

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™ 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

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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 Assays Pharmaceutical Preparations Methods-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

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• Nasdaq-listed – OCAT • Closed $30m equity offering in June

2015, cash on hand into late 2016 • ~41.6m shares and 2.75m warrants

outstanding – $10m debt facility with SVB – no preferred stock – ~2.8m options and RSU’s held by

executive team, employees and directors

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Ocata Therapeutics Financial Overview

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Future Milestones Bolster Position as The Leading Regenerative Ophthalmology Company

Year Period Milestone

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)

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Execution Against Strategic Plan is Our Focus

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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

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Experienced Corporate & Scientific Boards

Name Experience

Michael Heffernan, (Chairman) CEO – Collegium Pharmaceuticals

Robert Langer, Sc.D. Institute Professor – Massachusetts Institute of Technology

Greg Perry CFO – Agerion Pharmaceuticals

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

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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 Recently completed $30 Million financing, NASDAQ up-listing and Russell 2000 provides corporate visibility to a wider audience 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; Reported in credible peer reviewed publications. 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 Experienced management team, corporate and scientific boards

The World Leader in Regenerative Ophthalmology

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