September 2015 Corporate Presentation Ocata Therapeutics - NASDAQ OCAT

Transcription

1 September 2015 Corporate Presentation Ocata Therapeutics - NASDAQ OCAT

2 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. 2

3 The Regenerative Ophthalmology Company Addressing Macular Degeneration with Groundbreaking RPE Therapy 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 - H Dry Age-related Macular Degeneration (AMD) Phase 2 - Q 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 3

4 2015 Milestones Demonstrate Execution Against Plan Milestone 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 Significance 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 Milestones in 1H 2015 Position Company for Success in Next Months 4

5 RPE Damage and Subsequent Photoreceptor Degeneration Leads to Loss of Central Visual Acuity 5

6 The First Evidence of Long-term Safety and Efficacy Signal Following Transplantation of RPE Cells 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 6

7 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 7

8 The Eye is Well-Suited for Cellular Transplantation Our Strategic Intent is to Own the Eye 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 Patients in the Ocata Studies with more than Three Years Post Transplant Experience Continue to Show Good Safety with Visual Acuity Gains 8

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

10 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 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 Multiple Opportunities for Product Development and Commercialization Preclinical IND Phase 1 Phase 2 SMD Dry AMD MMD Phase 2 Study for Dry AMD, Pivotal Trial for SMD (Orphan) Photoreceptors Ganglion Cells Opportunity for additional IND s Cornea Planned Commercial Launch for Stargardt s Macular Degeneration in

12 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 SMD, Dry AMD and MMD are Specialized Opportunities and Feasible for an Emerging Biotech 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 A Viable Standalone Plan Creates Synergy with Potential Partners 12

13 Clinical Programs RPE for SMD, Dry AMD and MMD 13

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 Loss of RPE Layer Results in Loss of Vision Ocata s Therapeutic Approach is to Transplant New RPE Layer and Help Restore Vision 14

15 Normal Physiology, RPE in Intimate Contact with Photoreceptors and Provide Vital Life Support 15

16 Macular Degeneration Leads to RPE Loss Photoreceptors incur Damage, Dormancy and eventual Death 16

17 Ocata s Therapeutic Approach is to Deliver Brand New Terminally Differentiated RPE Cells via Sub-Retinal Injection 17

18 Data Indicate Transplantation of New RPE Can Lead to Restoration of Anatomy & Function Neural signal restored 18

19 Macular Degeneration Long Term Data in SMD and Dry AMD 19

20 Phase 1/2 Studies Presented Evidence of Engraftment; Transplanted Cells Take Residence, as Intended Baseline* Month 6* 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 posttransplantation Anatomic Evidence of Successful Engraftment Illustrates that Transplant is Rebuilding the Support Structure in the Back of the Eye 20

21 Updated Status SMD and AMD Trials 31 Patients Reported with at least 12 Months Follow Up June 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 Safety of treatment continues to be positive and efficacy signals are persistent over the long term 21

22 Interim Data for AMD and SMD Lancet Study Showed that BCVA Improved and was Sustained Over the Long-Term Mean Change from Baseline (letters) Lancet publication: May 2014 Dry AMD Subjects with 12 Month Follow-up Days after transplant Mean Change from Baseline in BCVA (letters) Lancet publication: May 2014 SMD 5 Subjects with 12 Months Follow-up Days After Transplant Treated Eye Untreated Eye Treated Eye Untreated Eye Efficacy Signal and Safety Persists in Treated Eyes 22

23 Clinical Program Design Pivotal SMD Trial and Phase 2 Dry AMD Trial 23

24 SMD Pivotal Trial, Planned Initiation in the Second Half of 2015 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) Trial Designed to Enable Potential Approval in

25 AMD Phase 2 Safety & Proof-of-Concept, Planned Initiation - Q 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) Corporate First Cohort Data read out Q ( Anatomy and Efficacy Signal) 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 25

26 Pre-Clinical Pipeline Photoreceptor Progenitors 26

27 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 27

28 Systemic hesc-phrps Provide Neuroprotection in Mice and Rats Prevent photoreceptor degeneration in ELOVL4 mice Preserve outer segments of photoreceptor in RCS rats PhRPs rescue photoreceptors in RCS rats a-wave (cones & rods) PhRP, 2 mo PBS control missing OS (P90) Healthy ONL Healthy ONL PhRP, 1 mo PBS, 1 mo PBS, 2 mo Tail vein injection 60 ONL PhPR-treated (rod OS/rhodopsin) Missing ONL Missing ONL 10 0 PBS Ø Tx PBS No Injection RA 2 month Increased ONL thickness after 2 months Intravitreal injection 28

29 Operations and Corporate 29

30 Ocata Therapeutics DeltaCell Technology Manufacturing Process in cgmp Environment Step Master Cell Bank of hesc s Cryopreserved inexhaustible replicative capacity starting material, stored at Ocata and in remote location Expansion of cells ~20 fold Shift to Terminal Differentiation to RPE cells Purify RPE and re-passage to expand quantity Harvest bulk material and cryopreserve RPE inventory Induction of proliferation and cell culture expansion In-process assays to ensure morphology and sterility of cells The process of re-passaging the cells can produce >1,000 fold increase of quantity of RPE cells Available for patient dosing; one five month process typically yields ~1,000 doses 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 Cell Line to Patient Treatment Single Blastomere Derivation of hescs 1 Patent Family 4 Issued Patents 12 Pending Applications Expiry begins 2025 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 3 Patent Families 4 Issued Patents 26 Pending Applications Core Patents Improvements Pharmaceutical Preparations 5 Patent Families 2 Issued Patents 34 Pending Applications Core Patents Formulation Improvements Shipping Medium 2035 Product Release Assays 2 Patent Family 11 Pending Applications Expiry will begin 2031 &

32 Ocata Therapeutics Financial Overview 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 32

33 Future Milestones Bolster Position as The Leading Regenerative Ophthalmology Company Year Period Milestone 2015 Q3 Q4 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 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) Execution Against Strategic Plan is Our Focus 33

34 An Experienced Management Team Name Position Experience Paul K. Wotton, Ph.D. President & CEO PENWEST 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

35 Experienced Corporate & Scientific Boards Board of Directors Broad Experience in Life Science Sector World Class Scientific Advisory Board Name Experience Name Experience Michael Heffernan, (Chairman) CEO Collegium Pharmaceuticals 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) Robert Langer, Sc.D. Institute Professor Massachusetts Institute of Technology Constance Cepko, Ph.D. Harvard Medical School National Academy of Sciences (elected 2002); Alfred W. Bressler Prize in Vision Science (2011) Greg Perry CFO Agerion Pharmaceuticals 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) Alan C. Shapiro Finance Professor and Chairman, Department of Finance and Business Economics (retired) University of Southern California John Gearhart, Ph.D. University of Pennsylvania Director of the Institute for Regenerative Medicine; Science, Board of Reviewing Editors Stanford University Zohar Loshitzer Paul K. Wotton, Ph.D. President Presbia, Inc., & Principal at Orchard Capital President and CEO, Ocata Therapeutics Michael Longaker, M.D. Joseph Vacanti, M.D. Director, Institute of Stem Cell Biology and Regenerative Medicine; Director, Children's Surgical Research Massachusetts General Hospital Surgeon-in-Chief; Member, Institute of Medicine of the National Academy of Sciences

36 The World Leader in Regenerative Ophthalmology 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 36