Financial Results for the 2Nd Quarter of the Fiscal Year Ending January 31, 2020

（TSE Stock Code:4592）

Financial Results for the 2nd Quarter of the Fiscal Year ending January 31, 2020

SanBio Company Limited

Representative Director and President Keita Mori

September 19, 2019

©2019 SanBio Company Limited 0 Today’s Schedule 1. Overall Update Representative Director and President Keita Mori 2. Business Update in Japan Corporate Officer, Business Head (Japan/Asia) Hiroshi Yamamoto 3. Global Clinical Update Corporate Officer, Chief Medical Officer and Head of Research Bijan Nejadnik 4. Becoming a Global Leader Representative Director and President Keita Mori

©2019 SanBio Company Limited 1 Today’s Schedule 1.Overall Update Representative Director and President Keita Mori 2. Business Update in Japan Corporate Officer, Business Head (Japan/Asia) Hiroshi Yamamoto 3. Global Clinical Update Corporate Officer, Chief Medical Officer and Head of Research Bijan Nejadnik 4. Becoming a Global Leader Representative Director and President Keita Mori

Solid business progress (despite timing-related variances)

(Unit：Million yen) Factors of 2Q for FY2020.1 Forecast(A) Actual(B) (B)-(A) Difference

Revenue Portion of development (Development support 284 427 +143 support fee was recorded fees, etc.) earlier

Research and development R&D cost 3,478 2,013 -1,465 and manufacturing expense were recorded later Operating expenses 3,730 2,812 -918

Operating income -3,445 -2,385 +1,060

Net income -3,197 -2,048 +1,149

Yen/US$ exchange rate 110.00 109.93 -

Financial base stabilized through equity financing

(Unit：Million yen) As of Jan 31, 2019 As of Jul 31, 2019 (B)-(A) (A) (B) Cash & cash equivalent 12,453 16,732 +4,279 Current assets 13,058 17,584 +4,526 Non-current assets 917 752 -165 Total assets 13,975 18,337 +4,362 Current liabilities 1,066 466 -600 Non-current liabilities 4,000 4,000 - Total liabilities 5,066 4,466 -600 Net assets 8,909 13,870 +4,961 Total assets and liabilities 13,975 18,337 +4,362

* (off-balance) The amount of Commitment line: JP¥5.3 billion

～2020 ～2025

2020 ✓ TBI domestic Becoming a self-marketing Pharmaceutical Company 2025 Becoming a ✓ Maximize the value of SB623 Global Execute a global launch Leader Establish a global product supply system ✓ Strengthen the pipeline (In-licensing, M&A)

*TBI: Traumatic Brain Injury ©2019 SanBio Company Limited 5 Today’s Schedule 1. Overall Update Representative Director and President Keita Mori 2.Business Update in Japan Corporate Officer, Business Head (Japan/Asia) Hiroshi Yamamoto 3. Global Clinical Update Corporate Officer, Chief Medical Officer and Head of Research Bijan Nejadnik 4. Becoming a Global Leader Representative Director and President Keita Mori

R-SAT® Patient tracking system Regenerative Medicine Safety Accurate Traceability

Patient Product Follow-up after * registration traceability administration Input PMS Patient’s ID

Product lot Temperature management Able to link logger / GPS

【Scope of joint development】

*PMS: Post Marketing Surveillance

Distribution center Manufacturing- approved area Final product SB623 （GCTP*）

Cell drug Under ~150℃ Pharmaceutic Cell drug Under ~150℃ market to (Before shipment factory from Immediate shipment al wholesaler

judgment) Lot: XXXXX (if necessary)

Shipment judgment judgment Shipment judgment Shipment Same timing

Medical RT SB623 medication set box institution

Syringe & cannula (A / B) RT SanBio and Suzuken have started talks with a view to concluding a logistics outsourcing RT agreement covering transportation and delivery Washing suspension Immediate shipment of the products from manufacturing facility to Lot: XXXXX medical facilities, and manufacturing license to *GCTP: Good Gene, Cellular, and Tissue-based Products Manufacturing Practice make shipment decisions.

Distribution Center Medical Institution

Cell storage tank Transport tank (001b）

Cell Cell drug drug Receiving Storage Patients Cell Delivery Cell Cell drug drug drug 001a 001a ＝ Cell drug (one or more) moved Transport tank＋Cell drug ④Status management to transport tank ＝Final Product (001c) from delivery to storage

③Final product ②Manufacturing registration / / shipping shipment ①Patient registration / instructions ordering Database

Company ⑤Delivery report, sales and delivery slip

Medical Medical institution Doctor

Confirm date of administration

Registrant ⇒ Approver Registrant ⇒ Approver Pharmaceutical companies Email / Register Check and adjust Register Phone schedule the schedule schedule

Register new Notification of Register new １ patient schedule corrections via patient schedule Main registration information email / phone Register correction ・ Estimated date of each if any checkpoint Manufacturing Check and adjust company the schedule

S.D.COLLABO Check and adjust CO.,LTD. the schedule S.D.Logi CO., LTD. Suzuken

R-SAT Email for completion Email for correction system of patient registration of patient schedule

【Advantages for patient, doctor, hospital】【Advantages for wholesalers and manufacturers】

✓ Able to check where the product is now ✓ Confirms shipment records and ensures and how it will be delivered to the product traceability. hospital. ✓ Confirms operation date and follow-up ✓ Clarifies the date of operation and by medical team. (Relieves anxiety on clarifies the division of roles such as the hospital side when first operating room, cell adjustment room, administering to a patient.) person in charge. ✓ Return system reduces the number of ✓ Reminder email for post-marketing discarded products to the minimum surveys for doctors. (Includes reminder possible. of the date of patients’ revisit; follow-up system is carefully designed). ✓ Prevents the omission of patient follow- up after administration and ensures ✓ Products that have not been opened execution of post-marketing (products and cells that are not used) surveillance. can be returned. ✓ Builds a complete regenerative (Note) Patient’s personal information is not obtained. medicine platform.

SanBio makes it easy to access the Japanese market

R-SAT Medical Foreign company system team

Pharma- PMS ceutical Japan market attracting affairs worldwide attention due to Domestic and overseas the Regenerative Medicine bio-ventures Safety Assurance Law and the revised Pharmaceutical Affairs Law (November 2014)

Strong positive reception at key conferences

Date Area Conference name Place Presenter

April 2019 American Association of San Dr. David O. US 2019 Neurological Surgeons Diego Okonkwo

The 14th Korea-Japan Joint April Hokka Dr. Masahito Japan Conference on Surgery for Cerebral 2019 ido Kawabori Stroke

August The 20th Annual Meeting of the Japan Dr. Yasuaki Japan Tokyo 2019 Society of Molecular Neurosurgery Karasawa

August Military Health System Research Kissim Dr. David O. US 2019 Symposium 2019 mee Okonkwo

September 2019 World Federation of Neurosurgical Dr. Takao China Beijing 2019 Societies Special World Congress Yasuhara

April 2018 Nov 2018 April 2019 By Jan 2020 By Jan 2021 File Publish Saki for LPI Approval Market Result gake Appro val

Prepare for Prepare for filing Evaluation Global Phase 2 marketing

Publish Result Filing ・Statistically significant improvement ・Aiming to submit an application for manufacturing and marketing approval in Japan during the fiscal year ending ・SB623 8.3 points, Control 2.3 points* January 31, 2020 (February 2019–January 2020), using the conditional and term-limited authorization system Sakigake Market ・Received Sakigake designation ・Aiming to market by January 2021 ・Results in improved positioning for approval *Reflects final score after updates

©2019 SanBio Company Limited 15 Today’s Schedule 1. Overall Update Representative Director and President Keita Mori 2. Business Update in Japan Corporate Officer, Business Head (Japan/Asia) Hiroshi Yamamoto 3.Global Clinical Update Corporate Officer, Chief Medical Officer and Head of Research Bijan Nejadnik 4. Becoming a Global Leader Representative Director and President Keita Mori

Bijan Nejadnik, M.D., Leading figure in clinical development with diverse experience

• Former career ✓ At Johnson and Johnson: early and late stage registrational trials in Alzheimer’s disease, immunology, autoimmune disorders, hematology and oncology. Several drug approvals. ✓ At Jazz Pharmaceuticals: as Executive Director, supervised biologic license applications (BLA) in ALL and BMT (VOD), receiving US FDA approval. ✓ Dr. Nejadnik was Chief Medical Officer at Galena Biopharma and later at Eureka Therapeutics, where he was responsible for all clinical development programs, regulatory, cell manufacturing and medical affairs.

• What do you think about SB623? ✓ The potential for impacting healthcare is tremendous. ✓ We have a great chance to achieve one of the most important objectives of medicine – to reverse biological injury. Using engineered stem cells, we have an opportunity to restore motor and sensory functions by inducing or promoting the innate, natural regenerative processes of patients’ physical functions.

Breakthrough Therapy Regenerative Medicine Advanced Designation Therapy (RMAT) Designation

A drug is intended to treat a serious A drug is a regenerative medicine therapy, condition, AND AND Qualifying preliminary clinical evidence indicates the drug is intended to treat, modify, reverse, that the drug may demonstrate or cure a serious condition, AND criteria substantial improvement on a clinically preliminary clinical evidence indicates that the significant endpoint(s) over available drug has the potential to address unmet therapies medical needs for such disease or condition

⚫ All fast track designation features, including • Actions to expedite ⚫ All breakthrough therapy designation development and review features, including early interactions to • Rolling review discuss any potential surrogate or intermediate endpoints Features ⚫ Intensive guidance on efficient drug development, beginning as early as ⚫ Statute addresses potential ways to Phase 1 support accelerated approval and satisfy post-approval requirements ⚫ Organizational commitment involving senior managers

✓ The FDA granted the RMAT designation based on clinical results for SB623, including the Phase 2 Study of Modified Stem Cells in Traumatic Brain Injury (STEMTRA) trial. ✓ The trial met its primary endpoint, demonstrating significantly improved motor function from baseline compared to the control group, with a well-tolerated safety profile. ✓ TBIs are one of the most common health conditions worldwide, and often cause long-term complications or death. RMAT allows us to work closely with the FDA on a potentially accelerated clinical development program to address this unmet medical need. ✓ RMAT constitutes major recognition by the FDA, providing a strong endorsement and increasing the potential for SB623 to have a successful path to approval. ✓ The RMAT designation augments the Sakigake Designation for innovative medical products from the Ministry of Health, Labour, and Welfare of Japan.

April November April September 2018 2018 2019 2019

Publish Publish LPI ATMP RMAT FPI LPI Result Result

Preparation Phase 3 Market Global Phase 2 for Phase 3

ATMP Phase 3 ・Granted Advanced Therapy Medicinal ・Plan to initiate a Phase 3 trial for TBI by Product Classification by European the end of the fiscal year ending Medicines Agency (EMA) January 31, 2021 RMAT ・Study design and development details are currently under consideration ・Granted Regenerative Medicine Advanced Therapy by Food and Drug Administration (FDA)

Continue development to aim for global expansion: clinical trial planning in US

FY2020.1 (2019/2~2020/1) FY2021.1~

Phase 2b* Clinical design Start study

*Detailed analysis Discussing US development with

©2019 SanBio Company Limited 22 Today’s Schedule 1. Overall Update Representative Director and President Keita Mori 2. Business Update in Japan Corporate Officer, Business Head (Japan/Asia) Hiroshi Yamamoto 3. Global Clinical Update Corporate Officer, Chief Medical Officer and Head of Research Bijan Nejadnik 4.Becoming a Global Leader Representative Director and President Keita Mori

TBI:2.5 million*7 TBI:11.0 million*5 *8 Stroke:3.7 million *6 Stroke:15.7 million TBI:5.3 million*1 Stroke: 5.7 million*2 TBI:40,000*3 Stroke:780,000*4

TBI:26,000*9 Stroke:420,000*10

*1:Traumatic Brain Injury In the United States: Epidemiology and Rehabilitation（US Centers for Disease Control and Prevention） *2:Heart Disease and Stroke Statistics, 2016 update *3:Japanese Ministry of Health, Labour and Welfare 2017 (The total number of cerebrovascular disease patients) *4:Japanese Ministry of Health, Labour and Welfare 2017 *7:Center-TBI HP (https://www.center-tbi.eu/) *5:Arch Neurol. 1986;43(6):570-572 (Wang et al.) *8:The burden of stroke in Europe (The stroke Alliance for Europe) *6:Circulation. 2017;135:759–771 (Wang et al.) *9:Australian Family Physician, Volume 43, No.11, November 2014 Pages 758-763 *10:Australia’s health 2018 (Australian Institute of Health and Welfare） ©2019 SanBio Company Limited 24 Competitors - TBI Competitive drugs are few and mostly for Acute

Agent Company Phase Mechanism Key Details

VAS-203 • Vasopharm’s European Phase III trial is testing VAS-203, III Nitric Oxide a neuroprotective agent, in acute TBI patients to improve (Ronopterin) Synthase Inhibitor clinical outcomes at 6 and 12 months

Autologous Bone • This cell-based product requires autologous harvesting, CEVA-101 II Marrow-derived isolation and processing, and is intravenously infused Mononuclear Cells within 48 hours of injury

NNZ-2566 • IV-trofinetide has received Fast Track Designation from II Neurotrophic the FDA for TBI, Fragile X, and Rett Syndrome and (Trofinetide) Peptide Analogue collaborates with the U.S. Army for TBI. Acute TBI.

NA-1 • NoNo Pharma’s lead candidate promotes cell survival and I PSD95 Inhibitor disrupts toxic cell signals following damage to neurons, Tat-NR2B9c in both TBI and ischemic stroke

• Tetra Discovery’s leading compound is in furthest BPN-14770 I PDE4D Modulator development for Fragile X Syndrome, and in Phase I for TBI and Alzheimer’s Disease

Allogeneic Bone • Compared to other MSCs, itMSCs secrete higher levels of PIIa Marrow-derived itMSCs growth factors usually associated with angiogenesis and IND ischemia-tolerant healing. mesenchymal stem cells

Clinical development by SanBio TBI: Clinical development by SanBio => Considering commercialization => Self-marketing or business alliance alternatives Stroke: Discussing with

East-Asia (China, Korea, Taiwan), Australia =>Southeast Asia, West Asia, Middle East

 Mr. Tsujimura amassed wide-ranging experience regarding overseas business administration with Nichimen Corporation (now Sojitz Corporation) and also garnered a wealth of pharmaceutical business experience at Santen Pharmaceutical Co., Ltd., where he concluded sales license agreements, conducted M&A, and managed overseas expansion. Corporate Officer, Business Head (Japan/Asia) Chief Medical Officer Hiroshi Yamamoto Bijan Nejadnik  Has wide business experience in Japan and Asia region  Has succeeded in securing approvals from

 Background: Eisai Co., Ltd.; AbbVie Inc.; BLA/NDA/MAA Santen Pharmaceutical Co., Ltd.  Background: Johnson & Johnson, Jazz Medical Director and Head of Pharmaceuticals, Galena Biopharma, Eureka Development / Japan Therapeutics Takehiko Kaneko  Has extensive experience in clinical Senior Director, Marketing development of pharmaceuticals Michael Gaither  Background: Bristol-Myers Squibb Company; Pfizer Inc. (US) and Pfizer Japan  Has over 25 years of experience in launching Inc.; Novartis Pharma K.K. and marketing products from major pharmaceutical companies and pioneers in Senior Advisor biotechnology Damien Bates  Background: GfK Health, Nexus Global, Impax Labs, Genentech USA  Has the distinction of leading the first and only successful regulatory approval of an allogeneic cell therapy product in the US.

Deploying human resources familiar with development and commercialization in each region

Efficacy and Safety Pharmaceutical affairs • Confirmation of efficacy in a double-blind study • FDA: RMAT • Confirmation of safety in over • PMDA: Sakigake 200 cases

Market size Competitors There are no competing • The number of patients is • products for the chronic stage comparable to Stroke (“blue ocean”)

Manufacturing Distribution • Stable supply system, • Smooth launch with partnering with Hitachi SanBio’s platform under Chemical and others construction

Grow by expanding into new indications and geographies

Alzheimer’s disease

Spinal cord injury

Parkinson’s disease

Retinitis Pigmentosa

AMD (dry)

Hemorrhagic Indication (chronic)

Stroke (chronic) Phase 2b →Detailed analysis

TBI Phase 2 Phase 2 (chronic) →Preparing to File →Phase 3

Asia JP US EU Oceania etc.

Deliver novel therapeutics to patients as rapidly as possible to maximize corporate value

This presentation material, including any comments made during or following the presentation, is provided solely for the purpose of reference to those investors who make their own evaluation of the company at their own risk. This material contains estimates, such as plans, strategies and judgments, that are forward-looking statements which are made based on management’s assumptions and beliefs in light of the information currently available to it and may contain risks and uncertainty. Therefore you should not place undue reliance on them in making investment decisions. SanBio cautions you that actual results may differ substantially from those discussed in this material due to various factors. We do not guarantee the accuracy or completeness of the information herein. Unless otherwise stated, estimates or forecasts are solely those of our company and subject to change without notice. We accept no liability whatsoever for any direct or consequential loss arising from any use of this report. SanBio Company Limited Management Administration Contact: [email protected]