Healios Kk · TSE:4593
Healios Kk — FY2016 2Q Earnings Presentation
59 slides from Healios Kk's earnings presentation, July 2016
Slide 1: FY2016 2Q Financial Results
“To be the change in an ever evolving world through enrichment of living” FY2016 2Q Financial Results
Slide 2: Contents
Contents 1 Financial Highlights P2 2 Updates in FY2016 2Q P5 Details of Somatic Stem Cell 3 P15 Regenerative Medicine 4 Details of iPSC Regenerative Medicine P23 5 Expansion to 3D Organ（Liver） P37 6 News Follow-Up P44 7 Company Overview P46 8 Appendix P51 1 Copyright © HEALIOS K.K. All rights reserv
Slide 3: 1. Financial Highlights
1. Financial Highlights 2
Slide 4: Statement of income
Statement of income （Million yen） FY 2016 2Q FY 2015 2Q YoY change Reasons for increase/decrease Sales 59 41 ▲18 Operating income ▲513 ▲2,602 ▲2,089 Research & Development Cost ▲2,030 Ordinary income ▲418 ▲2,647 ▲2,229 - Net income ▲417 ▲2,650 ▲2,233 - R&D 292 2,322 +2,030 Multistem License-in +1,80
Slide 5: Balance sheet
Balance sheet （Million yen / %） June 30, 2016 December 31,2015 Change Main reasons for increase/decrease Current assets 9,325 9,271 ▲54 (88.9%) (89.2%) Non-current 1,162 1,123 ▲38 assets (11.1%) (10.8%) Total assets 10,487 10,395 ▲92 (100.0%) (100.0%) Current 1,080 1,130 +50 liabilities (10.3%) (10.
Slide 6: 2. FY2016 2Q Updates
2. FY2016 2Q Updates 5
Slide 7: Highlights: FY2016 2Q Updates
Highlights：FY2016 2Q Updates Somatic - Advancement of preparation toward the start of a clinical study from the second stem cell regenerative MultiStem® half of this fiscal year after consulting with PMDA medicine - Consideration of construction of the sales system with alliance 2 - Optimization for
Slide 8: Collaborative research agreement with Universal Cells, Inc. in June 2016
Collaborative research agreement with Universal Cells, Inc. in June 2016 １ RPEs in Japan Healios entered a collaborative research and exclusive option iPSC Regenerative agreement with Universal Cells, Inc. regarding application of their medicine RPEs genome editing technology to process iPS cells. W
Slide 9: Announcement of a new clinical research by RIKEN in June 2016
Announcement of a new clinical research by RIKEN in June, 2016 Announcement of “Clinical research of transplantation 2 iPSC Regenerative RPEs in of induced pluripotent stem cells – derived retinal Japan pigment epithelium (RPE) cells for exudative age- medicine related macular degeneration (AMD)” Ou
Slide 10: Joint R&D Related to Pre-Transplant Immune Reaction Testing Method
Joint R&D Related to Pre-Transplant Immune Reaction Testing Method in June 2016 2 iPSC RPEs in -Joint R&D to develop a new testing method to confirm prior Regenerative to transplantation whether cells are suitable for transplant, Japan medicine including the possibility to cause immunorejection. Det
Slide 11: Results of a Phase III investigator initiated trial published
Results of a Phase III investigator initiated trial were published in an academic journal in June, 2016 3 Preparation for marketing approval in Japan Compounds BBG Advances towards GMP production at the contract manufacturers of the drug substance/product Journal of Japanese Ophthalmological Society
Slide 12: Pipeline
Pipeline Pre- Development Phase Phase Phase Apply- On the Filed Indication Market clinical Progress Status Code I Trial II Trial III Trial approve Market test Somatic stem Ischemic cell Planning to start Clinical Trial in HLCM051 Stroke Japan Regenerative the second half of FY2016 Medicine Start of
Slide 13: Transition to iPSC Regenerative Medicine
Transition to iPSC Regenerative Medicine Technological development from low molecular drug to antibody drug to iPSC regenerative medicine, etc. (Technological development） Low molecular drug Antibody drug iPSC regenerative medicine (Time) 12 Copyright © HEALIOS K.K. All rights reserved.
Slide 14: Portfolio
Portfolio Image of Development of Regenerative medicine STROKE i P S C i P S C Holy Grail O r g a n b u d RPE Immune Barrier Immunosuppressant LIVER LIVER KIDNEY KIDNEY Somatic stem cell iPS Cell(Mono) iPS Cell(3D) 13 Copyright © HEALIOS K.K. All rights reserved.
Slide 15: Healios' approach for Technology for Immune Reaction
Healios’ approach for Technology for Immune Reaction (Cost） Immune Barrier Immunosuppressant ｃ ｃ - HLA matched iPSC - Pre-transplant immune reaction testing - Genome editing technology - Pluripotent cells manufactured to avoid transplant rejection Autologous iPSC Allogeneic iPSC Bank Universal iPSC
Slide 16: 3. Details of Somatic Stem Cell Regenerative Medicine
3. Details of Somatic Stem Cell Regenerative Medicine 15
Slide 17: Licensing Contract Concluded between Athersys and Healios
Licensing Contract Concluded between Athersys and Healios Healios and Athersys, Inc. (“Athersys”) entered into a license agreement over products for regenerative medicine marketed in Japan, making use of the stem cell product MultiStem® and related technology, whose patent right and patent licensing
Slide 18: Outline of Ischemic Stroke in Japan
Outline of Ischemic Stroke in Japan Ischemic stroke Ischemic stroke, which represents the most common form of stroke (70–75％ in Japan), is caused by a blockage of blood flow in the brain that cuts off the supply of oxygen and nutrients and results in tissue loss It is estimated that 37.9％ of bedridd
Slide 19: Annual number of new patients with ischemic stroke in Japan
Annual number of new patients with ischemic stroke in Japan Target population Japan Note Annual medical costs for Number of patients (year) 230,000 – 330,000 ischemic stroke estimated at 1070.7 billion yen（2009) Severe patients （atherothrombotic and cardiogenic cerebral 130,000 infection) patients w
Slide 20: Mechanism of treatment using MultiStem
Mechanism of treatment using MultiStem Exhibits its therapeutic efficacy by suppressing inflammation and immune reactions and stimulating the formation of neuroprotective substances Following intravenous administration after acute neurological injury, MultiStem distributes to spleen, downregulatin
Slide 21: MultiStem Product Profile
MultiStem Product Profile （Source）Athersys Cell therapy product based on patented Consistent safety profile technology Promotes healing and tissue repair through Developing for “off-the-shelf” multiple mechanisms of action administration: no tissue matching needed Not a permanent transplan
Slide 22: Long-term (1 year) Results of Phase 2 clinical trial in the U.S. and U.K.
Long-term (1 year) Results of Phase 2 clinical trial in the U.S. and U.K. Percentage of Patients with Excellent Outcome 30% 29.0% MultiStem(18h-36h :n=31) 23.1% MultiStem(18h-48h :n=65) 16.1% 15.4% 8.2% Placebo(18h-48h :n=61) 6.6% 90 days 365 days (Source) Prepared by Healios based on the data provi
Slide 23: Strategic Background of Introduction of MultiStem
Strategic Background of Introduction of the Stem Cell Product, MultiStem Development in the field of stroke Cerebral infarction therapy with a new mechanism can be proposed and high market needs can be expected. Deliver it promptly to Japanese patients as a regenerative therapy product. Technica
Slide 24: 4. Details of iPSC Regenerative Medicine
4. Details of iPSC Regenerative Medicine Step 1 Treat diseases with iPS Cells Step 2 About AMD and Profitability Step 3 Strategy for Placing RPE Cell Medicine on the Market 23
Slide 25: Concept of iPSC Regenerative Medicine
Concept of iPSC Regenerative Medicine Make “Regular Cells” from iPS Cells nerve cell myocardial cell Cell Transplantation Induction Treatment iPS cells pancreatic cell hepatocyte cell 24 Copyright © HEALIOS K.K. All rights reserved.
Slide 26: Mechanism of iPS Regenerative Medicine
Mechanism of iPS Regenerative Medicine Replace damaged cells with regular cells Tissue (cells) Damaged iPSC Regenerative Medicine Replace Damaged Tissue (cells) by Aging, Disease or Target Cells made from iPS cells with regular cells Accident Replacement Patient Recovery of Organ Function 25 Copyrig
Slide 27: 4. Details of iPSC Regenerative Medicine
4. Details of iPSC Regenerative Medicine Step 1 Treat diseases with iPS Cells Step 2 About AMD and Profitability Step 3 Strategy for Placing RPE Cell Medicine on the Market 26
Slide 28: Explanation of AMD Pathological Condition
Explanation of AMD Pathological Condition AMD causes RPE Cells to degenerate, which will damage functions Regular Macular Part Developed Dry AMD Immunity Barrier Maintained → Visual Cell Degenerative Disease → Dry AMD Macula Wet AMD Retinal Pigment Destruction of Immunity Barrier → Invasion of Immun
Slide 29: Estimated Number of AMD Future Patients in Serious Cases in US
Estimated Number of AMD Future Patients in Serious Cases in US Expected to Increase over Medium to Long Term as Society Ages （Thousand patients） 6,000 5.44 million 5,000 total caucasian black hispanic others 4,521 4,000 3.66 million 3,115 3,000 2.07 million 1,850 2,000 1,000 446 178 240 132 247 228
Slide 30: Number of AMD Patients
Number of AMD Patients Number of both Wet and Dry patients (including mild cases) （Thousand patients） America Japan Others The Number of AMD Patients 10,000 9,230 13,000 The Number of AMD Patients in Serious Cases 2,000 690 2,600-3,220 Wet-Patients in 1,000-1,500 630 1,300-1,950 serious cases Dry-Pa
Slide 31: Market Scale of AMD
Market Scale of AMD Annual Sales of Medicinal Treatments of Wet AMD: 831.6 billion yen. No Medicine for Dry AMD. Medicin Condition e/Effect Year Total America Japan Others Anti- VEGF 2013 363.6 billion yen 38.0 billion yen 294.7 billion yen 696.3 billion yen Medicine/ Wet Restraint of New Blood 2014
Slide 32: Treatment Subjects for Wet AMD
Treatment Subjects for Wet AMD Patients recurring within 1 Year, which is the case of approx. 92% of Wet AMD Patients, are the candidates for treatment. Recurrence frequency after receiving anti-VEGF medication No Relapse in 12 months 8% Present Treatment Subjects: Approx. 92％ Relapse in 12 months 3
Slide 33: Changes in Vision
Changes in Vision Good vision can be maintained with early treatment RPE transplantation (single) Vision） RPE transplantation (Prediction) Continuous administration of anti-VEGF medication Anti-VEGF medication Administration Not administered discontinued * The purpose of this illustration is to prom
Slide 34: Guide to Medicine Price – Approach from Medical Expense
Guide to Medicine Price – Approach from Medical Expense of Existing Health Care Anti-VEGF Medicine mostly Continues From the Beginning of the Treatment to Death Annual Medical Expense Annual Recommended Annual Medical Expense Unit Price of Anti-VEGF Medication Protocol 170,000 yen 1,020,000 yen 6 Ti
Slide 35: 4. Details of iPSC Regenerative Medicine
4. Details of iPSC Regenerative Medicine Step 1 Treat diseases with iPS Cells Step 2 About AMD and Profitability Step 3 Strategy for Placing RPE Cell Medicine on the Market 34
Slide 36: Autologous and Allogeneic Transplantation: Mass Production
Autologous and Allogeneic Transplantation We Handle Cross-Regenerative Medical Treatment , with High Profit Margins iPSC Regenerative iPSC Regenerative Medicine Medicine （Autologous） (Allogeneic) Patient A Donor Cell Cell Extraction Extraction Mass Production High Profit Cultivation Cultivation Indu
Slide 37: Project System
Project System Arranged Domestic Value Chain by Cooperation with Sumitomo Dainippon Pharma Co., Ltd Development Production Marketing Sales Can Build Up Developing, Sales and Manufacturing in Japan Institution (※) Medical Healios SighRegen SighRegen Healios Secured Personnel Structure for 6.8 billion
Slide 38: 5. Expansion to 3D Organs (Liver)
5. Expansion to 3D Organs（Liver） 37
Slide 39: Joint Research Agreement with Yokohama City University
Joint Research Agreement in Organ Regenerative Medicine with Department of Regenerative Medicine, Yokohama City University Graduate School of Medicine Leading authority of organ regeneration Prominent researcher of human organ construction Department of Regenerative Medicine, Yokohama City Departmen
Slide 40: Technical synergistic effects with iPSC regenerative medicine area
Technical synergistic effects with iPSC regenerative medicine area Consideration as a candidate for the one of the 3 types of cells that are necessary to create liver organ bud iPS cell of a Human Vascular Consideration of endothelial cell Precursor cell MSC or MultiStem the availability of MultiSte
Slide 41: Formation process of 3D Organ
Formation process of 3D Organ （Source）Japan Science and Technology Agency Science News “Diverse Approaches in Regenerative Medicine from Cell to Tissue/Organ” (Distributed October 3, 2013) https://sciencechannel.jst.go.jp/M130001/detail/M130001005.html 40 Copyright © HEALIOS K.K. All rights reserved
Slide 42: Survival Rate of Liver Failure Model Mouse
Survival Rate of Liver Failure Model Mouse Survival rate improves significantly in transplantation experiments Treatment effects of liver organ bud Process in which organ formed from organ bud links transplantation using human iPS cells mouse ‘s vascular network autonomously 100 Liver organ bud tran
Slide 43: Metabolic Liver Disease in Newborns
Metabolic Liver Disease in Newborns Yokohama City University planning to start clinical study in 2019 Urea cycle defect Disease caused by congenital abnormality of enzyme functioning in metabolic pathway (urea cycle) which detoxifies ammonia in the liver and produces urea. Presently, the only defini
Slide 44: Prospecting R&D for alternative treatment for liver transplantation
Expansion of Marketability (Alternative Treatment for Replacing Liver transplantation) Prospecting R&D for alternative treatment for liver transplantation Liver transplantation Total US Japan Europe Number of patients undergoing Approx. 6,000 Approx. 400 Approx. 4,000 Approx. 10,000 treatment (Annua
Slide 45: 6. News Follow-Up
6. News Follow-Up 44
Slide 46: Summary of Main News Follow-Up
Summary of Main News Follow-Up Clinical Study Application/ Market Somatic License in Clinical study MultiStem preparation approval release stem cell （Ischemic regenerativ Stroke ） e medicine Business alliance Preclinical Study Clinical Study Marketing RPEs in Japan RIKEN：Clinical research re-start A
Slide 47: 7. Company Overview
7. Company Overview 46
Slide 48: Healios Overview
Healios Overview About Us Company Name HEALIOS K.K.（TSE: 4593） Representative Hardy TS Kagimoto,MD President and CEO World Trade Center Building 15F Head Office 2-4-1 Hamamatsucho Minato-ku, Tokyo Japan 105-6115 Paid in Capital 5380million yen(As of end of June, 2016) Research Institution Kobe and Y
Slide 49: Company Roots
Company Roots How it was founded Started business to establish treatments for age-related macular degeneration and determined to build up a pharmaceutical company which can provide treatments for intractable diseases. Founded Aqumen Biopharmaceuticals K.K. (later transferred business to Healios)
Slide 50: Our Experienced Team for Development
Our Experienced Team for Development Lineup to Develop in Japan, US, and Europe Ophthalmology Expert Expert in Age-related Macular Degeneration President and CEO Director Overseas Development Al Reaves, Ph.D Hardy TS Kagimoto, MD Responsible for innovative Succeeded in Developing BBG clinical pr
Slide 51: Company History and Social Trends
Company History and Social Trends Pioneer of iPSC regenerative medicine development Collaborative Patent licensing agreement research Retina Institute Japan entered with iPS Academia Japan Joint research on organ bud agreement entered established with Universal Cells started with Yokohama City (curr
Slide 52: 8. Appendix
8. Appendix 51
Slide 53: Summary of safety results for all patients
Phase 2 clinical trial in the U.S. and U.K. [Safety] Summary of safety results for all patients Safety results observed for MultiStem-treated vs placebo-treated patients MultiStem Placebo Diff. Parameter, at day 90 (n=65) (n=61) Baseline NIHSS(>24 hours) 13.4 13.3 n/a Mortality 6.2% 14.8% 8.6% Lif
Slide 54: Phase 2 clinical trial in the U.S. and U.K. [Efficacy]
Phase 2 clinical trial in the U.S. and U.K. [Efficacy] Summary of efficacy results for the subset of patients It showed significantly favorable recovery compared to placebo at 90 days according to the second analysis of patients administered MultiStem within 36 h after onset MultiStem Placebo Diff
Slide 55: Long-term (1 year) Results of Phase 2 clinical trial
Long-term (1 year) Results of Phase 2 clinical trial in the U.S. and U.K. Between-group Differences in Excellent Outcome at 90 and 365 Days 90days 365days Subjects MultiStem (n=65) within 48 hours of stroke 15.4% 23.1% Placebo (n=61) within 48 hours of stroke 6.6% 8.2% Between-group difference 8.8%
Slide 56: Approval of regenerative medicine products, September 2015
Rapid Impact of Modified Regulations Approval of regenerative medicine products, September 2015 Product Name Temcell® HS Injection Heart Sheet Company JCR Pharmaceuticals Terumo Acute GVHD after hematopoietic Severe heart failure due to ischemic heart Indications stem cell transplantation disease Pr
Slide 57: Japanese Government Revises Regulations to Put Japan at the Forefront
Historical Alleviation of Regulations Japanese Government Revises Regulations to Put Japan at the Forefront Process of the Development So Far Clinical Research Clinical Trial Approval Sales Confirm Effectiveness and Safety Development Process of Introducing Early Approval System After-sales Effectiv
Slide 58: Important Note on Future Events, etc.
Important Note on Future Events, etc. Descriptions on future events, etc. in this document include Healios’ assumptions, prospects, etc. based on information which could be acquired at the time this document was presented. For this reason, actual performance, development progress, etc. may differ fr
Slide 59: Contact information
＜Contact information＞ Phone: +81-(0)3-5777-8308 Corporate Communication Group For the press: [email protected] Personnel and Administration Department For the investors: [email protected] HEALIOS K.K.