financial results for the 2nd quarter of the fiscal year ...€¦ · as of jan 31, 2019 (a) as of...

©2019 SanBio Company Limited 0

（TSE Stock Code:4592）

September 19, 2019

SanBio Company LimitedRepresentative Director and President

Keita Mori

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


Today’s Schedule

1.Overall Update

2.Business Update in Japan

3.Global Clinical Update

4.Becoming a Global Leader

Representative Director and PresidentKeita Mori

Corporate Officer, Business Head (Japan/Asia)Hiroshi Yamamoto

Corporate Officer, Chief Medical Officer and Head of ResearchBijan Nejadnik



Today’s Schedule

1.Overall Update









Consolidated Income Statement

Solid business progress (despite timing-related variances)(Unit：Million yen)

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

Revenue(Development support fees, etc.)

284 427 +143Portion of development support fee was recorded earlier

R&D cost 3,478 2,013 -1,465Research and development 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 -


Consolidated Balance Sheet

Financial base stabilized through equity financing

As of Jan 31, 2019(A)

As of Jul 31, 2019(B)

(B)-(A)

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

(Unit：Million yen)


Business targets

*TBI: Traumatic Brain Injury

～2020 ～2025

✓ TBI domestic self-marketing

✓ Maximize the value of SB623

Execute a global launch

Establish a global product supply system

✓ Strengthen the pipeline (In-licensing, M&A)

2020Becoming a

PharmaceuticalCompany 2025

Becoming aGlobalLeader


Today’s Schedule

1.Overall Update









Distribution - Suzuken

×


About the R-SAT system

R-SAT® Patient tracking system

Regenerative MedicineSafetyAccurateTraceability

Patient registration

Product traceability

【Scope of joint development】

Follow-up after administration Input

Patient’s ID

Temperature logger / GPSAble to link

Product lot management

PMS*

*PMS: Post Marketing Surveillance


Logistics concept

Medical institution

Cell drug(Before shipment

judgment)

Syringe & cannula(A / B)

Washing suspension

Cell drug Under ~150℃

RT

RT

Same timing

RT

Under ~150℃

Manufacturing-approved area

（GCTP*）

Ship

ment

judgm

ent

from

facto

ry

Ship

ment

judgm

ent

to m

ark

et

Immediate shipmentLot: XXXXX

Distribution center

Pharmaceutical wholesaler (if necessary)

SB623 medication set box

SB623

SanBio and Suzuken have started talks with a view to concluding a logistics outsourcing agreement covering transportation and delivery of the products from manufacturing facility to medical facilities, and manufacturing license to make shipment decisions.*GCTP: Good Gene, Cellular, and Tissue-based Products Manufacturing Practice

Immediate shipmentLot: XXXXX

Final product


Information flow

Distribution Center

Cell storage tank

Celldrug

Celldrug

Celldrug

Celldrug

001a

Celldrug

001a

Transport tank (001b）

Cell drug (one or more) moved to transport tank

＝

Medical Institution

Receiving Storage PatientsDelivery

④Status management from delivery to storage

①Patient registration / ordering

Transport tank＋Cell drug＝Final Product (001c)

③Final product registration /

shipment②Manufacturing

/ shipping instructions

Company⑤Delivery report, sales and delivery slip

Database


Partial view of R-SAT systemMedical institution

Pharmaceuticalcompanies

Manufacturing company

S.D.COLLABO CO.,LTD.

Suzuken

R-SATsystem

Medical Doctor

Confirm date of

administration

Register new patient schedule

Registrant ⇒ Approver

１

Email / Phone

Register schedule

Main registration information・ Estimated date of each checkpoint

Email for completion of patient registration

Check and adjust the schedule



Register new patient schedule

Register schedule

Register correction if any

Notification of corrections via email / phone

Email for correction of patient schedule

S.D.Logi CO., LTD.

Registrant ⇒ Approver


Features of R-SAT system

✓ Able to check where the product is now and how it will be delivered to the hospital.

✓ Clarifies the date of operation and clarifies the division of roles such as operating room, cell adjustment room, person in charge.

✓ Reminder email for post-marketing surveys for doctors. (Includes reminder of the date of patients’ revisit; follow-up system is carefully designed).

✓ Products that have not been opened (products and cells that are not used) can be returned.

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

✓ Confirms shipment records and ensures product traceability.

✓ Confirms operation date and follow-up by medical team. (Relieves anxiety on the hospital side when first administering to a patient.)

✓ Return system reduces the number of discarded products to the minimum possible.

✓ Prevents the omission of patient follow-up after administration and ensures execution of post-marketing surveillance.

✓ Builds a complete regenerative medicine platform.(Note) Patient’s personal information is not obtained.


Platform concept for regenerative medicine

SanBio makes it easy to access the Japanese market

Foreign company

Domestic and overseas

bio-ventures

R-SATsystem

Medicalteam

Pharma-ceuticalaffairs

PMS Japan market attracting worldwide attention due to the Regenerative Medicine

Safety Assurance Law and the revised Pharmaceutical Affairs

Law (November 2014)


Scientific conference presentations

Strong positive reception at key conferences

Date Area Conference name Place Presenter

April2019

US2019 American Association of Neurological Surgeons

San Diego

Dr. David O. Okonkwo

April 2019

JapanThe 14th Korea-Japan JointConference on Surgery for Cerebral Stroke

Hokkaido

Dr. Masahito Kawabori

August 2019

JapanThe 20th Annual Meeting of the JapanSociety of Molecular Neurosurgery

TokyoDr. Yasuaki Karasawa

August 2019

USMilitary Health System Research Symposium 2019

Kissimmee

Dr. David O. Okonkwo

September 2019

China2019 World Federation of Neurosurgical Societies Special World Congress

BeijingDr. Takao Yasuhara


・Statistically significant improvement・SB623 8.3 points, Control 2.3 points*

Aiming for rapid domestic approval

Schedule – TBI (Japan)

Global Phase 2

April 2018

LPIPublish

Result

Nov 2018

Saki gake

File for

Approval

Approval Market

Prepare for filing EvaluationPrepare for marketing

April 2019 By Jan 2020 By Jan 2021

Publish Result

・Aiming to submit an application for manufacturing and marketing approval in Japan during the fiscal year ending January 31, 2020 (February 2019–January 2020), using the conditional and term-limited authorization system

Filing

・Received Sakigake designation・Results in improved positioning for approval

Sakigake

・Aiming to market by January 2021

Market

*Reflects final score after updates


Today’s Schedule

1.Overall Update









In the beginning…

• 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.

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

• 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.


Received RMAT designation (Sept. 19, 2019)


What is RMAT?

Source: Expedited Programs for Regenerative Medicine Therapies for Serious Conditions (https://www.fda.gov/media/120267/download)

Breakthrough Therapy Designation

Regenerative Medicine Advanced Therapy (RMAT) Designation

Qualifyingcriteria

A drug is intended to treat a serious condition, ANDpreliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies

A drug is a regenerative medicine therapy, ANDthe drug is intended to treat, modify, reverse, or cure a serious condition, ANDpreliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition

Features

⚫ All fast track designation features,including

• Actions to expedite development and review

• Rolling review

⚫ Intensive guidance on efficient drug development, beginning as early as Phase 1

⚫ Organizational commitment involving senior managers

⚫ All breakthrough therapy designation features, including early interactions to discuss any potential surrogate or intermediate endpoints

⚫ Statute addresses potential ways to support accelerated approval and satisfy post-approval requirements


RMAT approval and impact

✓ 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.


Schedule – TBI (Global)

Global Phase 2

FPI LPIPublish

Result

Preparation for Phase 3

Phase 3 Market

・Plan to initiate a Phase 3 trial for TBI by the end of the fiscal year ending January 31, 2021

・Study design and development details are currently under consideration

Phase 3

・Granted Regenerative Medicine Advanced Therapy by Food and Drug Administration (FDA)

RMAT

Global development progress

RMAT

September 2019

April2018

LPIPublish

Result

November2018

・Granted Advanced Therapy Medicinal Product Classification by European Medicines Agency (EMA)

ATMP

ATMP

April2019


Schedule - Stroke

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

*Detailed analysisDiscussing US development with

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

Phase 2b* Clinical design Start study


Today’s Schedule

1.Overall Update









Market size

TBI:5.3 million*1

Stroke: 5.7 million*2TBI:40,000*3

Stroke:780,000*4

TBI:2.5 million*7

Stroke:3.7 million*8

*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 *5:Arch Neurol. 1986;43(6):570-572 (Wang et al.)*6:Circulation. 2017;135:759–771 (Wang et al.)

TBI:11.0 million*5

Stroke:15.7 million*6

TBI:26,000*9

Stroke:420,000*10

*7:Center-TBI HP (https://www.center-tbi.eu/)*8:The burden of stroke in Europe (The stroke Alliance for Europe)*9:Australian Family Physician, Volume 43, No.11, November 2014 Pages 758-763*10:Australia’s health 2018 (Australian Institute of Health and Welfare）


Agent Key Details

VAS-203

(Ronopterin)

• Vasopharm’s European Phase III trial is testing VAS-203,

a neuroprotective agent, in acute TBI patients to improve

clinical outcomes at 6 and 12 months

• IV-trofinetide has received Fast Track Designation from

the FDA for TBI, Fragile X, and Rett Syndrome and

collaborates with the U.S. Army for TBI. Acute TBI.

CEVA-101

NNZ-2566

(Trofinetide)

• This cell-based product requires autologous harvesting,

isolation and processing, and is intravenously infused

within 48 hours of injury

• NoNo Pharma’s lead candidate promotes cell survival and

disrupts toxic cell signals following damage to neurons,

in both TBI and ischemic stroke

NA-1

Tat-NR2B9c

• Tetra Discovery’s leading compound is in furthest

development for Fragile X Syndrome, and in Phase I for

TBI and Alzheimer’s DiseaseBPN-14770

Company MechanismPhase

Nitric Oxide

Synthase InhibitorIII

Autologous Bone

Marrow-derived

Mononuclear CellsII

Neurotrophic

Peptide AnalogueII

PSD95 InhibitorI

PDE4D ModulatorI

• Compared to other MSCs, itMSCs secrete higher levels of

growth factors usually associated with angiogenesis and

healing.itMSCs

Allogeneic Bone

Marrow-derived

ischemia-tolerant

mesenchymal stem cells

PIIa

IND

Competitive drugs are few and mostly for Acute

Competitors - TBI


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

Clinical development by SanBio=> Considering commercialization

alternatives

Leverage business platform built on TBI

Global commercialization plans

TBI: Clinical development by SanBio=> Self-marketing or business alliance

Stroke: Discussing with


Management for global leadership

Chief Medical OfficerBijan Nejadnik

Has succeeded in securing approvals from BLA/NDA/MAA

Background: Johnson & Johnson, Jazz Pharmaceuticals, Galena Biopharma, Eureka Therapeutics

Senior Director, MarketingMichael Gaither

Has over 25 years of experience in launching and marketing products from major pharmaceutical companies and pioneers in biotechnology

Background: GfK Health, Nexus Global, Impax Labs, Genentech USA

Senior AdvisorDamien Bates

SanBio, Inc. CEOAkihiro Tsujimura 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)Hiroshi Yamamoto Has wide business experience in Japan

and Asia region Background: Eisai Co., Ltd.; AbbVie Inc.;

Santen Pharmaceutical Co., Ltd.

Medical Director and Head of Development / Japan Takehiko Kaneko Has extensive experience in clinical

development of pharmaceuticals Background: Bristol-Myers Squibb

Company; Pfizer Inc. (US) and Pfizer Japan Inc.; Novartis Pharma K.K.

Has the distinction of leading the first and only successful regulatory approval of an allogeneic cell therapy product in the US.

Background: Former SanBio, Inc. CMO; Baxter; Organogenesis; Allergan


Deploying human resources familiar with development and commercialization in each region

Strengthening the management team for global leadership


SB623: major potential in TBI

• The number of patients is comparable to Stroke

Market size

• Stable supply system, partnering with Hitachi Chemical and others

Manufacturing

• There are no competing products for the chronic stage (“blue ocean”)

Competitors

• Smooth launch with SanBio’s platform under construction

Distribution

• FDA: RMAT• PMDA: Sakigake

Pharmaceutical affairs• Confirmation of efficacy in a

double-blind study• Confirmation of safety in over

200 cases

Efficacy and Safety


Becoming a global leader

Grow by expanding into new indications and geographies

Ind

icati

on

Alzheimer’s disease

Spinal cord injury

Parkinson’s disease

Retinitis Pigmentosa

AMD (dry)

Hemorrhagic(chronic)

Stroke (chronic) Phase 2b→Detailed analysis

TBI(chronic)

Phase 2→Preparing to File

Phase 2→Phase 3

JP US EUAsia

Oceaniaetc.


Global leader in regenerative medicine

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


Disclaimer

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]

financial results for the 2nd quarter of the fiscal year ...€¦ · as of jan 31, 2019 (a) as of...

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