Healios Kk · TSE:4593
Healios Kk — FY2016 3Q Earnings Presentation
48 slides from Healios Kk's earnings presentation, November 2016
Slide 1: FY2016 3Q Financial Results
“To be the change in an ever evolving world through enrichment of living” FY2016 3Q Financial Results
Slide 2: Contents
Contents 1 Financial Highlights P2 2 Updates in FY2016 3Q P5 Details of Somatic Stem Cell 3 P12 Regenerative Medicine 4 Details of iPSC Regenerative Medicine P18 5 Expansion to 3D Organ（Liver） P32 6 News Follow-Up P37 7 Company Overview P39 8 Appendix P43 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 3Q FY 2015 3Q YoY change Reasons for increase/decrease Sales 76 57 ▲19 Operating income ▲705 ▲3,053 ▲2,347 Research & Development Cost ▲2,269 Ordinary income ▲611 ▲3,099 ▲2,488 - Net income ▲582 ▲3,103 ▲2,521 - R&D 375 2,644 +2,269 Multistem License-in +1,80
Slide 5: Balance sheet
Balance sheet （Million yen / %） September 30, 2016 December 31,2015 Change Main reasons for increase/decrease Current assets 9,325 8,665 ▲660 (88.9%) (88.4%) Non-current 1,162 1,138 ▲24 assets (11.1%) (11.6%) Total assets 10,487 9,803 ▲684 (100.0%) (100.0%) Current 1,080 1,052 ▲28 liabilities (10.3%
Slide 6: 2. FY2016 3Q Updates
2. FY2016 3Q Updates 5
Slide 7: HLCM051 to Clinical Stage
HLCM051 to Clinical Stage Clinical trial protocol notification was submitted to PMDA. A 30-day review Somatic stem cell period was completed for the clinical study initiation. HLCM051 regenerative MultiStem® medicine “Placebo-Controlled, Double-Blind, Phase 2/3 Efficacy and Safety Trial of HLCM051 (
Slide 8: Study Design Based on the Result of the Phase II Study by Athersys
Study Design Based on the Result of the Phase II Study by Athersys in Europe and America Somatic The proportion of patients who achieved Excellent Outcome was statistically stem cell HLCM051 significant (compared with the placebo group) both at Day 90 and Day 365 in regenerative MultiStem® the group
Slide 9: Pipeline
Pipeline Pre- Development Phase Phase Phase Apply- On the Field Indication Market clinical Progress Status Code I Trial II Trial III Trial approve Market test Somatic stem Ischemic cell Regenerative HLCM051 Stroke Japan To Clinical stage Medicine Start of clinical studies originally HLCR011 Wet AMD
Slide 10: 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) 9 Copyright © HEALIOS K.K. All rights reserved.
Slide 11: Image of the Transition of Cellular Medicine Products
Image of the Transition of Cellular Medicine Products Somatic Stem Cell Holy Grail Immune Barrier Immunosuppressant E S C i P S C i P S C O r g a n b u d 10 Copyright © HEALIOS K.K. All rights reserved.
Slide 12: 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 13: 3. Details of Somatic Stem Cell Regenerative Medicine
3. Details of Somatic Stem Cell Regenerative Medicine 12
Slide 14: Introduced New Pipeline: HLCM051 in January 2016
Introduced New Pipeline: HLCM051 in January 2016 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 authorit
Slide 15: 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 16: 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 17: Mechanism of treatment using HLCM051
Mechanism of treatment using HLCM051 Exhibits its therapeutic efficacy by suppressing inflammation and immune reactions and stimulating the formation of neuroprotective substances Following intravenous administration after acute neurological injury, HLCM051 distributes to spleen, downregulating hy
Slide 18: HLCM051 Product Profile
HLCM051 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 transplant:
Slide 19: 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 18
Slide 20: 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 19 Copyright © HEALIOS K.K. All rights reserved.
Slide 21: Mechanism of iPSC Regenerative Medicine
Mechanism of iPSC 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 20 Copyri
Slide 22: 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 21
Slide 23: Explanation of AMD Pathological Condition
Explanation of AMD Pathological Condition AMD causes RPE Cells to degenerate, which will damage functions Developed Dry AMD Immunity Barrier Maintained → Visual Cell Degenerative Disease → Dry AMD Wet AMD Macula Destruction of Immunity Barrier → Invasion of Immunity Cells → Inflammation → Wet AMD Re
Slide 24: 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 25: 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 26: 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- 363.6 billion 294.7 billion 696.3 billion VEGF 2013 yen 38.0 billion yen yen yen Medicine/ Wet Restraint of New Blood 414.
Slide 27: 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 28: 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 29: 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 30: 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 29
Slide 31: Autologous and Allogeneic Transplantation
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 Mass Production Extraction Extraction High Profit Cultivation Cultivation Indu
Slide 32: Project System: Domestic Value Chain with Sumitomo Dainippon Pharma
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 6.8 billion yen by Collaborative Secured Pe
Slide 33: 5. Expansion to 3D Organs (Liver)
5. Expansion to 3D Organs（Liver） 32
Slide 34: 3D Organ Generation Mechanism
3D Organ Generation Mechanism Generating “Orgun bud” by co-culturing 3 types of cells iPS cell of a Human Vascular Consideration of endothelial cell Precursor cell MSC or MultiStem the availability of MultiStem Organ bud transplantation Consideration of therapy (origin of organ) the large supply of
Slide 35: 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 36: 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 37: Expansion of Marketability
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 38: 6. News Follow-Up
6. News Follow-Up 37
Slide 39: 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 Consideration of construction of the sales system RPEs in Preclinical Study Clinical Study Marketing Japan iP
Slide 40: 7. Company Overview
7. Company Overview 39
Slide 41: 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 5,387million yen(As of end of September, 2016) Research Institution Kobe
Slide 42: 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 Hardy TS Kagimoto, MD Al Reaves, Ph.D Responsible for innovative Succeeded in Developing BBG clinical pr
Slide 43: Company History and Social Trends
Company History and Social Trends Pioneer of iPSC regenerative medicine development Patent licensing agreement Collaborative entered with iPS Academia Joint research on organ bud research Retina Institute Japan Japan started with Yokohama City agreement entered established University with Universal
Slide 44: 8. Appendix
8. Appendix 43
Slide 45: Rapid Impact of Modified Regulations
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 46: Historical Alleviation of Regulations
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 47: 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 48: 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.