Mid-Term Strategic Plan KWB 2.0
Full version
All for Kids, Kids for All

Kidswell Bio Corporation

Our vision
All for Kids, Kids for All

KIDS WELL, ALL WELL
～ For Comprehensive Healthcare System forChildren as well as Families, and Society ～
Focus
areas

Business
direction

Pediatric diseases
including juvenile
diseases,
intractable & rarediseaseTargeting diseases
which has lessaccessibility forsufficient medical
treatment

Families

Educational
Institutions

KIDS WELL

Friends

MedicalInstitutions

Society

Government

All for Kids, Kids for All• Under the declining birthrate and aging population, reducing theburden on children is a major social issue.

For Children• Provide new pharmaceuticals and therapeutics to patients
suffering from diseases and contribute to the realization of asociety where children and adults who support children live
happily and brightly.

Mid-term strategic plan KWB 2.0 to survive in the competitiveenvironment and promptly realize our vision2

Reasons for launching KWB 2.0- Changes in the biosimilar market•-

Launched GBS-007,our third biosimilar product, as the first biosimilar in the ophthalmology fieldin Dec. 2021.
 Sales continue to be better than expected and expecting further growth•

The entry of biosame (authorized biosimilar (AGS)) into the market is getting impacts
to the biosimilar market
Competitive Biosame
Name of productIndications

Medicinal effects

Development
Company

Approval
date

Impact on KWB’s
products

Eylea(Aflibercept)

Age-related
maculardegeneration

Anti-VEGF
antibody drugs

Bayer Yakuhin,
Ltd.

Feb. 2022

Competitive
product of GBS007

Nesp(Darbepoetin Alfa)

Renal anemia

Erythropoietin
Receptor
Activator

Kyowa Kirin Co.,
Ltd.

Aug. 2018

Competitive
product of GBS011

 As the number of biosame entering the market will increase, the competition formarket share with biosimilars will intensify (profitability will decline). As a result,potential partners of pharmaceutical companies will hesitate to launch new
biosimilar projects.
 Biosimilar projects that secure enough profitability will become less.
• Continue to maximize value for existing biosimilar pipelines• Need to change KWB’s profit structure that relies solely on biosimilar businesses.

Accelerating the creation of new medicines to increase corporate value by SHED
(cell therapy) development

3

Reasons for launching KWB 2.0–Strategies for an increase in stock prices -The stock market didn’t evaluate KWB’s business expansion by launching biosimilars.

(Yen)

2800
2600
2400
2200

Bio-brand
bubble

GBS-001(Filgrastim BS)

GBS-011(Darbepoetin alfa BS)

GBS-007(Ranibizumab BS)

Launched in Nov.
2012

Launched in Sept.
2019

Launched in Dec.
2021

2000
1800
1600
1400

Launched three biosimilar products with investment

1200
1000
800
600
400
200
0
(Period)

Stock price transition• The launch of biosimilars alone didn’t lead to stock price increase.
• Sticking to achieving target profitability with the biosimilar business alone won’t be reflected in ”big”
stock price increase.
4

KWB 2.0– Investment for breakthrough in SHED development -Active investment in the SHED (Cell Therapy) pipelines to promptly realize ourvision• Promote global business expansion and R&D of SHED(cell therapy)• Accelerate development in overseas while continuing business in Japan• Aiming to launch the world‘s first SHED medicine/ therapy products by FY 2030
Enhancing corporate• Continue biosimilar business and new biologics business
value

Aiming to be a one-of-a-kind company thatcreates cell and gene therapy products
utilizing SHED(Million yen)
1500

Revenue from SHED

1000

Revenue from BS
500

0

2011

2012

2013

2014

2015

2016

2017

2018

2019

2020

2021

（FY）

-500

-1000

-1500

Investment
in BS

Investment in
SHED
R&D

研究開発費
expenses

Sales
売上高
revenueGross

粗利revenue5

Business foundation for KWB 2.0
Step-ahead strengths among other bio ventures• Profit structure achieving sales of 3 billion yen and operating income of 1 billion
yen in FY 2025
• Well-knowledge and experienced human resources in biologics development• Well-accumulated know-how in the development of biosimilar products

Biosimilars Business Foundation
GBS-001(Filgrastim BS)

GBS-011(Darbepoetin alfa BS)

GBS-007(Ranibizumab BS)

Fourth
Product
Fifth Product...

Medium- to long-term cash flow + Experts for proceeding biosimilar
pipeline + Well-accumulated know-how

Expand and strengthen profits through biosimilar
products

6

Growth strategies for breakthrough in SHED
Commercialization

Launching regenerative
medicine products of the
1st generation of SHED

Technology

For Launching regenerative medicine products of the
1st generation of SHED

Invention in SHED and next
generation technology

For commercialization of the 2nd generation of SHED• Establishment of SHED Supply system• Introduction of next-generation technologies togenerate synergies• Selection of target diseases throughcollaborative researches• Combination with synergistic devices• Promoting technology adoption through alliances
and acquisitions• Establishment of clinical development
systemFocusing on R&D of SHED pipelines andaccelerating R&D in Japan and overseas
Fund-raising

Human resources andorganizational structure

Financing for strategic
execution

Strengthening SHED
development
structure• Financing from overseas market• Establishment of a SHED delivery system tooverseas• Large-scale financing to realize SHED
commercialization•• Equity financing specialized for SHED
development

Establishing office in overseas
 Fostering networks with international medical
institutions and academia Strengthening cooperation between Japanand overseas• Staff recruiting for global expansion

7

Breakthrough in SHED

- Prompt commercialization of the first generation of SHED -Selecting target diseases throughjoint research with academia

Accelerating clinical development inJapan and overseas

Based on the evidence through our R&D,
further joint research will be promoted
and maximize SHED potential.

Through Real World Data (RWD), Virtual
Clinical Trials (DCT), aiming for shortened
clinical trial period and earlycommercialization

Academia

Nonclinical

Clinical

Europ
e

U.S
Japan

SHED

Manufacturing（SHED Source Supply Service）

A one-stop service to providing highly reliable and domestically
produced SHED as an intermediate product for regenerative medicine
products through business collaboration8

Breakthrough in SHED - Investment schedule Focusing on SHED development and accelerating investment forincrease in corporate value
Estimation
Non-clinical study to ～ Corporate clinical trial(Expansion of indications）

Launching
2 to 5 billion yen

Japan

Recruiting, development of human resource andorganizational framework
2022

Over
seas

Some hundreds
million yen

2030

Feasibility study ・ Collaborative research ～ Corporate clinicaltrial• Office in overseas, recruiting & fostering global staff, the developmentof organizational structure• Introduction of next-generation technologies with synergies(Corporate alliances, M&A)
• Promoting the development of the next generation of SHED

Required investment in SHED development(year)

2 to 5 billion yen

Over 5 billion yen

Over 10 billion
yen

Making investments for launching SHED regenerative medicine/ cell therapy
products with equity financing
9Strategic Investment in human resources andorganizational structure• Contributing to society by the development of human resources andorganizational structure that leads to an increase in KWB’s corporate value• Pursuing contributions to society (S in sustainable development goals) throughthe creation of new medical treatments
Experts• Recruiting human resources with knowledge and experience in theregenerative medicine (cell therapy) field• Recruiting human resources with knowledge of next-generation modality• Recruiting global human resources for overseas business activities

Challenging work environment• Talent management to realize human resources strategy• Fostering a corporate culture with diversity (gender, age,nationality, values)• Flexible and appropriate personnel allocation

Organizational culture

with plenty of creativity
and innovation• Execution of career development plan (human resources
development plan)

Respect for each employees' work style• Flexible work system under emergency and pandemic• Establishing work infrastructure for flexible work styles• Fostering a corporate culture for understanding eachworking style
10Roadmap
Accelerating our R&D activities to realize our vision• Accelerate SHED R&D activities to realize ourvision and increase corporate value.

KIDS WELL, ALL WELL

• Continue stable revenue from biosimilarbusiness and reduce manufacturing costs• Determine when to achieve profitability byprioritizing accelerated investment

Launching cellular medicine/cell therapy products

Accelerating cell therapy
products development
Focusing on SHED development

Establishment of revenue base
Establishment of biosimilar
development technology• Establishment of SHED platform• Active investment in human resources
and capital• Diverse personnel structure, including
experts of cell medicine development
and human resources with knowledge of
new modalities• Stable revenue from three BS products• Started development of the 4th BS
product• New BS pipeline development

Founded (2001)
to FY 2021

• Steady development progress in Japanand overseas• Accelerating development in overseas.
in addition to domestic development• Accelerating R&D by fund-raising fromoverseas investors• Acquisition of biopharmaceuticaldevelopment know-how

Aiming to launch the world's
first SHED medicine/ therapy
by FY2030• Strengthening SHED business activities

SHED + Human Resource Development
Maintaining stable revenue of biosimilar business

FY 2022

FY 2025 （Turning profitable）（3 billion yen of sales and 1 billion yenof operating profit ）FY 2030 onward
11Our Role in Society
Two Virtuous Cycles through “KidsCentricity”“

MedicalExpenses Reduction &improvement ofmedial access

New Medical
Treatment

Creating regenerative medicine
with SHED for all generations

More people will overcome diseases andthey will play an active role in our society.

Drugdiscovery

Patentapplication

Combining individual technologies andknow-how for building up
efficient development
systemProduct
manufacturing

Clinicaldevelopment

Integrating technologies and know-how in Japan andoverseas and building up efficient R&D system

12

Cell Therapy Business(Regenerative Medicine)13Market Trends of Drug and Healthcare• Trends of diseases have shifted from the number of patients to subdivided diseases.
• From blockbuster products* to individual products• Global drug market size will exceed 100 trillion yen.
* Products with global annual sales of more than 100 billion yen
Vitamin pills, Antibiotics
Drug for peptic ulcer, Lifestyle-related disease drugs
Autoimmune disease, Immunomodulator
Anticancer drugs, Central nervous system drugs
Intractable & rare disease
Pediatric diseases & juvenile
diseases
1960

1980

Global Drug
Market size

（Estimation）

Diseases with many patients
Blockbuster products

2000

About
20 trnyen

About 50
trn yen2020
About
100 trn yen

2040

Some
hundreds
trn yen!!

Subdivided diseases
Individual medical treatment• Increase importance of individual medical care
• R&D of intractable, rare and pediatric diseases, which tend to behind, will be
more focused.
14

SHED
(Stem Cells from Human
Exfoliated Deciduous teeth)

15What are stem cells? (For regenerative medicines)• Muscles, bones, blood, organs, skin, nerves, etc. are all made of cells.
• Stem cells are the source of all cells.
Stem
cell

Muscle• Stem cells can repair and regenerate cells and tissues that can’t
perform their original functions due to illness.

CardiacmuscleBones,
cartilage

Blood
cell

Skin

nervesOthers(Organs、Adipose,etc.)

Kinds of stem cells
Stem cells

Pluripotent

ES cells

Obtain from cells by culture of fertilized eggs

iPS cells

Stem cells that are artificially induced
multipotencies

stem cells

Human
Somatic stem
cells

Mesenchymal
stem cell (MSC)

Stem cells inside the human body(Spinal cord, adipose, cord blood, dental pulp,
etc.)

Focus on stem cells of deciduous teeth
16

What’s SHED?
SHED:Stem cells from Human Exfoliated Deciduous teeth• One type of dental pulp stem cells that extracts from adental pulp cavity• SHEDs have extremely high proliferating ability, repair
and regenerative capabilities.
• Possible to extract from the deciduous teeth, which has
many sampling timings, and less burden on the donor.
• A new stem cell with a short research history in the
world

Donorderived
risks

(From
Umbilical cord)

SHED(Fromdeciduous
teeth)BMSC(From bonemarrow)

AMSC

Pulp cell

5 to 12 yrs old

Dental Pulp
(SHEDs)Bone
Marrow

AdiposeUmbilical
cord

Type of Bank

PrivatePublicPrivatePublic/PrivateAge of Donor

5 to 12 yrs
old

20 to 55
yrs old

Mostly over
20 yrs old

0 yr old

Timing ofExtraction

Usually 20
times atextraction of
deciduous
teeth

When
transplant
ation

When
surgery

Time of birthBurden on
Donor

Low

High

High

Very low

Proliferating
Ability

Very high

High

High

Research
stage

(From
adipose)DPSC(From permanent
teeth pulp)

0 yrs old

Gum

Cellularsenescence
Excellent balance of donor
age and donor-derived risks

UMSC

EnamelDentineDentalpulp

20 to 55 yrs old(Donor age)17

Features
Pulp stem cells from neural crests are expected to be particularly applicable to diseases of
the nervous and muscle/bone systems.

SHED: High gene expression related to neurogenesis,
angiogenesis, and cell migration ※Features of SHED

Application for regenerative medicine

High proliferative ability※1,2Sufficient cells in a short period oftime

Expression of neurogenesisrelated genes※1, 3, high secretion of
nervous system growth factor※1, 4
and high nerve regenerative
ability※1, 5

Diseases related to nerve
regeneration(Ex. Spinal cord injury, brain
infarction, cerebral palsy, etc.)

Diseases required boneregeneration
出所:
※1,6
High
bone regenerative(Ex. Non-union
fracture,※1:
自社/共同研究データ,※2: Miura et al. ability
PNAS. 2003, ※3: Terunuma
et al. J Stem Cell Regen
Med. 2019,※4: Mead et al. PLoS One. 2014, ※5: Sakai et al. J Clin Invest. 2012, ※6: Nakajima et al. BBRC. 2018
osteonecrosis of the femoral head,
etc.)

SHED

BMMSC

ATMSC

1.2

Relative Amount

SHED: Young cell and highly proliferative ※

1
0.8
0.6
0.4
0.2
0

BDNF

NGF

VEGF

HGF

SHED: High secretion capacity ofneurotrophic factor (BDNF, NGF)※※ KWB internal data18

Boosting licensing-out activities
SHED R&D Projects as our growth driver
Development
Product

Symptom

Existing
Treatment

Eating and speechdisorderLip arthroplasty+ iliac bonegraft

Maxilla boneregeneration

ORTHOREBIRTH

2,000 patients per
year

15 out of 10,000
newborns

Enterectomy,
colostomy

Ganglion
regeneration

Mochida Pharmaceutical

100 patients

ー

None

Nerve protection,
activation andregeneration

Tokyo Metropolitan Instituteof Medical Science, Nagoya
University, Tokyo Medical and
Dental University

2,000 patients per
year,
30,000 patients in
total

100,000 patients
per year, 1.7
millions patients in
total

Spinal cord injury

Loss of motor
function andsensation

None

Nerve protection,
activation andregeneration

Nagoya University

5,000 patients per
year,
100,000 patientsin total

25,000 patients
per year, 500,000
patients in total(US, EU andJapan)

Non-union
fractures

Chronic pain, gait
disturbance

SurgeryBoneregeneration

Hokkaido University andSpinal Injuries Center

100,000 patients
per yearー

Peripheral nerve
palsyMotor function
and sensation
disorderNerve
reconstruction(Autologous
nervetransplantation)

Peripheral nerve
regeneration

Oita University

8,000 surgeries
per yearー

Bone-relateddiseases※※※

Showa University School of
Medicine※※

Ophthalmologic
disease※※※

Gifu Pharmaceutical
University

※

※

Under
consideration

※

※

※

NanoCarrier, BioMimetics
Sympathies※※

Target disease
Pediatric disease

Cleft lip and palate
Pediatric disease

Congenital Isolated
Hypoganglionosis
Pediatric disease

Cerebral palsy

Including Pediatricdisease1st
generation
SHED

2nd
generation
SHED

Intestinal
obstruction

Quadriplegia andPosture disorder

Therapeutictarget※ Details not disclosed

Partners

Number of patients Number of patients(Domestic)(Global)19Collaborative Researches with academia
Progress in collaborative researches with academia for the application of
diseases related to the nervous and muscle/bone systems

下肢運動機能スコア（BBB score）

20
SpinalCord Injury

GFAP

Peripheral Nerve Palsy
SHED

✱✱✱✱

15

SHED
10

Control

Myelinatednerve axon
count

5

Control0
day1 1w

2w

3w

4w

5w

6w

Control SHED

Improvement of motor function and gliosis
with SHED administration

Missing nerves regeneration with SHEDtransplantation

Non-Union Fractures

Control

SHED

Defective bone regeneration by SHEDtransplantation

20

Market potential
Cerebral palsy

Spinal Cord Injury

Non-union fractures

Existing
treatment

None

None

Noe

Number ofpatients

10,000 patients per year in
U.S. (children)

12,500 patients per year in
U.S.

100,000 patients per year

Made by KWB from MyChild at CerebralPalsy.org and OSSGROW FOR NON-UNION FRACTURES, DOI: 10.1177/2151458517696680

25,000

Nervous system and muscle/ bone diseases targeted by SHED
will grow from 700 billion to 800 billion yen market by 2040.

20,000（100 million yen）

Estimation for market size in
2040 (100 million yen）
15,000Respiratory
Metabolic Disorders
Immunology

Autologous

Allogeneic stem

stem…

cells: 20,193

10,000Genetic Disorders
Gastrointestinal
Dermatology
Central Nervous System
Cardiovascular

5,000

0

※ Including Japan, theU.S. Europe, China andIndia in global market(Targeted 10% of the
population in China and2022 2023 2024 2025 2026 2027 2028 2029 2030 2031 2032 2033 2034 2035 2036 2037 2038 2039 2040（年） India）

Estimation for market size of allogeneic stem cells
KWB’s research based on Regenerative medicine and gene therapy market research in 2019 Final report from the website of Japan Agency for Medical Research andDevelopment, Global Data from epidemiological literature and websites from rare diseases (Orphanet, NORD, Japan Intractable Diseases Information Center, ClinicalDevelopment Success Rates 2006-2015, BIO Industry Analysis」 and related documents from general meeting of Central Social Insurance Medical Council

21

2nd Generation SHED22

Therapeutic modalities for the society
Shift to development trends in domestic & overseas:
designer cells for radical treatments
Cell Therapy

1900～2000
Small
molecule medicine

×Enhancedtherapeutic effectImprovement ofdisease directivity

2000～2020
Antibody drugs＝

Designer Cells

2020～
What comes next?

Designer Cells
Regenerative
Medicine

Modality of Celltherapy

Nucleic acid medicine
Antibody drugs
Protein/Peptide
pharmaceutical
Small molecule medicine→

Protein/Peptide
pharmaceutical
Small molecule
medicine→

Antibody drugs→

Protein/Peptide
pharmaceutical

Modality ofmolecule

Small molecule→→
medicine
Diversification of modalities (types of treatment methods) by increasing the speed oftechnological innovation
Created by Kidswell Bio Corporation referring from strategic proposal “Designer Cell” by Japan Science and Technology Agency

23

Designer Cells for Next Generation Cell Therapy
Launched development of designer cells as reinforced SHEDs to achieve highmedical treatment goals
Development of new gene transfer
methods

Target diseases ofdevelopment※（Ex: NanoCarrier）

Reinforced
SHED

Development of new culture methods(Ex: BioMimetics Sympathies Inc.）

Verification of the effects ofvarious types of transgenes

Genetic diseases
Degenerative (Nerve)
Cancer, etc.
※ in addition to nervous andmuscle/bone diseases in the list oftarget diseases of 1st generation SHED

(Ex: Nagoya Univ., Hamamatsu
University School of Medicine ）Aim to develop reinforced SHED with enhanced therapeutic efficacy
Infection

Lifestyle related

Degenerative（Nerve・Organ）

Autoimmunity

Cancer

Genetic diseases

Pediatric diseases

Permanent
Cure
Treatment

2nd Generaiton “Designer Cells”
Vaccine(Prevention・Treatment)

1st Generation Cell
Therapy

Recombinant Cell Therapy (Stem Cell-Based)
Add/Enhance Functions by Genetic Engineering

Non-Recombinant Typeof Stem Cells etc.

Symptomatic
Treatment

Small, Mid
Molecule Drug

DTx

Antibody Drug

Nucleic
Acid
Medicine* Created by Kidswell Bio Corporation referring from Strategic Proposal of Designer Cell by Japan Science and Technology Agency. 24

S-Quatre: SHED Supply Source Service

25

SHED Source Supply Service
Manufacture SHED MCB in compliance with GMPSHED MCB Manufacturing
Process

Deciduous teeth
donation

Isolation andCultivation (MCB)

Tooth extraction

Development of
final products

Kidswell Bio Corporation
Organiza
tion

Role

ChiVo Net

Univ. of Tokyo Hospital
Showa Univ. Dental Hospital

Secure stableregistration ofdonor
candidates

Confirmation of the generalcondition of the donor and tooth
extraction at the dentist and oral
surgery• Explanation and acquisition of consent• Gather and provide donor information• Donor screening (examination &inspection）• Collection and provision of extracted
teeth• Management of information related toresearch implementation• Management of Personal Information
and research IDs

Nikon CeLL innovation(Cell development・Manufacturing)

Development Partners

Manufacturing MCB incompliance with GMP

Supply MCB todevelopment partners
to develop final
products• Receive extracted teeth• Isolate and culture SHED
from pulp tissue• Manufacture MCB incompliance with GMP• Manufacture final products
such as cell therapies, exvivo gene therapies, andexosome therapeutics withMCB
• Possible to consign
manufacturing final
products.

Establishment of SHED
intermediate product supply（SHED Source Supply Service）
service26

Biosimilar Business27

Outline of Development
Challenge the development of new biosimilars with our experience of thelaunch of three BS products and cost competitiveness through highly efficientmanufacturing technology（Goal）High quality &reasonable BS

Launch 4th BS product by
2025
FY2022BS development to contributeprofits after FY2030
F2025

FY2030【New BS pipelines】• Promote the development of new biosimilars with high-cost competitiveness, although theenvironment for the biosimilar market is severe due to the impact of biosame.
• Aiming to create biosimilars that significantly reduce the financial burden on patients against
biopharmaceuticals with high annual drug costs.

【Experience of launch of BS products】
Accumulated track record: Knowledge and experience inthe development and the launch of BS products from thestart of the biosimilar business in 2007
Project

GBS-001(Filgrastim BS)

GBS-011(Darbepoetin alfa
BS)

GBS-007(Ranibizumab
BS)

OutlineG-CSF製剤(顆粒球コロニー形成
刺激因子製剤)

持続型赤血球造血刺激因子製剤

Anti-VEGF
antibody drug

Therapeutic
area

Neutropenia

Chronic Kidney
Disease,
Renal anemia

Age-related
maculardegeneration

Partners

Fuji Pharma Co.,
Ltd., MochidaPharmaceuticalCo., Ltd.

Sanwa Kagaku
Kenkyusho Co., Ltd.

Senju
PharmaceuticalCo., Ltd.

【High-Yield Protein Producing Technology】
Promoting collaborative researches with chromocenter(artificial chromosome technology) and SOLA Biosciences(Tapboost® technology* ) to dramatically reduce
manufacturing costs and ensure cost competitiveness andprofitability(* Technology to correctly arrange the three-dimensional structure of the target
protein in the production cell line ）Chromocenter

SOLA Bioscience

Increase in number ofgene copies by artificial
chromosome
technology＝ Mass production ofmRNAs

Increased rewind
efficiency by Tapboost®
＝＞Mass production ofproteins
28

Biosimilar pipeline
Clinical trial（Clinical study）
Project

Therapeutic
area

Basic researchPhase I

PhaseⅢ

Appli
catio
n/
Mark
eting

Appr
oval
/
Laun
ch

Partners

Fuji Pharma Co., Ltd.
MochidaPharmaceutical Co.,Ltd.

GBS-001
Filgrastim

Oncology

GBS-004
Bevacizumab

Oncology

GBS-005
Adalimumab

Immunologicaldisease

GBS-007
Ranibizumab

Ophthalmic
disease

GBS-008
Palivizumab

Infectious
disease

GBS-010
PEG-filgrastim

Oncology

GBS-011
Darbepoetin alfa

Renal disease

Sanwa Kagaku
Kenkyusho Co., Ltd.

GBS-012Aflibercept

Ophthalmic
diseaseKishi Kasei Co., Ltd.

Senju PharmaceuticalCo., Ltd.
Ocumension
Therapeutics

29New Biologics(antibody)Business

30

Outline of Development Plan
Challenge to create antibody drugs with completely new mechanisms of action（Goal）First-in-Class

Antibody drugs

Licensing-out and collaborative
development by FY2025
FY2022

Launching in early 2030s
FY2030

FY2025Circulatory diseases
 Therapeutic antibodies for pulmonary hypertension
Create new therapeutic antibodies by production of Therapeutic antibodies for malignant lymphoma
inhibitory antibodies for substances that are candidate ofCreate therapeutic antibodies with a new mechanism
root-cause substances for pulmonary hypertensionof action that does not depend on the patient's
 Therapeutic antibodies for vasculitis
immunity and induces direct cell death
Identify the causative agent that causes excessive
inflammation in the vascular wall and create therapeutic
antibodies with new mechanisms of action that inhibit
the substanceOncology

Project

Therapeutic Area

GND-004

Ophthalmic
disease, Oncology

GND-007

ImmunologicaldiseaseNew
Antibody

Basic
Research

NonClinical
Trial

Clinical Trial and Clinical Study
PhaseⅠ

PhaseⅡ

PhaseⅢ

Application/
MarketingApproval/
LaunchPartner

Oncology

Sapporo Medical
Univ.

Oncology

MabGenesis Co.,Ltd.
31

Malignant lymphoma
Create new therapeutic antibodies for malignant lymphoma【Target disease】【Market overview】

Malignant lymphoma• Lymphoma is a part of white blood cells (B cells, T/NK cells)
that become blood cancer

Existing treatment method: CAR-T
therapy• 10% of pediatric cancer【Challenges of current treatment】• Less radical cure and high mortality rate for highgrade of malignancy• CAR-T cell therapy has been developed for some B-cell
lymphomas, but side effects are strong. The cost of treatment
is high (drug price 33.49 million yen), putting pressure on
medical finances.

Though worldwide sales of Kymriah, Yescarta,
Tecartus (brexucabtagene autoleucel) etc.
reached 72 billion yen, expecting morethan 300 billion yen sales around 2026.
The domestic market for malignant
lymphoma of medicines including
chemotherapy is about 80 billion yen as of2021.

【KWB’s strategy】• Aim to create innovative therapeutic antibodies for malignant lymphoma that are highly effective inpatients with lowered immunizing power.
• Developing R&D of antibodies with a completely new mechanism of action that binds tomalignant lymphoma cells and directly kills them.

[Current]

Immune Cell

Cancer cell
Antibody

antibody
receptors

TargetantigenAttack[Future]Cancer cell

Cancer cell
Antibody
TargetantigenTargetantigenInducing direct cancer cell death32

Vasculitis
Create new therapeutic antibodies for vasculitis【Target disease】
Vasculitis（Kawasaki disease and others）• An intractable disease in which organ or tissue function is
reduced due to inflammation and bleeding in the vascularwall and clots• Classified by the type of blood vessel in whichinflammation occurs, Kawasaki disease is a pediatricvasculitis discovered in 1967 by Dr. Tomisaku Kawasaki• Kawasaki disease is a serious disease that highly
complicates coronary aneurysms in addition to symptoms
such as fever and rash due to excessive inflammation in
the vascular wall. The cause of disease has not clarified yet.

【Market overview (Estimates by
KWB)】
Kawasaki disease
Number of domestic patients is 15,000
patients per year. The current market
size is equivalent to about 4 billion yen.
Other vasculitis
Domestic market size will be more than
100 billion yen with the expand
indication.

【Challenges of current treatment】• Standard therapy is administration immune globulin.
Concerns about safety and radical treatment is urgent
issue because effect is not enough for 15 % of patients(children).

【KWB’s strategy】• Research on the causes of vasculitis• Aim to identify root-cause substances• After identifying the causative substance, aim to create an
inhibitory antibody and create an innovative new therapeutic
methods for vasculitis including Kawasaki disease.

Source:33

Pulmonary Hypertension
Creating new therapeutic antibodies for Pulmonary Hypertension【Target disease】
Pulmonary Hypertension• Pulmonary hypertension is a group of poorly prognostic
progressive diseases that result in high blood pressure in
the blood vessels "pulmonary arteries" that send bloodfrom the heart to the lungs and cause dysfunction of theheart and lungs.
• There are multiple causes such asleft ventricular dysfunction, pulmonary artery thickening,chronic obstructive pulmonary disease, etc., and 5 years
survival in the case of untreated is 50%.

【Market overview (Estimates by
KWB)】
The number of domestic potential
patients is estimated about 250,000
patients. If the annual treatment costwas 4 million yen and 25% of thepotential patients received treatment, it
would be a market size equivalent toabout 250 billion yen.

【Challenges of current treatment】• There is a treatment with vasodilator, but the therapeutic
effect is limited for patients with advanced disease states.
• The effect has not been confirmed for a large number of
pulmonary hypertension to the left heart dysfunction andchronic obstructive pulmonary disease.

【KWB’s strategy】• Create inhibitory antibodies for substances that arepotential root-cause of pulmonary hypertension• Verify the mechanism of action and its effectiveness in animalmodels and aim to create a completely new therapeutic methods for
pulmonary hypertension.

Increase
pulmonaryartery pressure

PulmonaryarteryLung
RightatriumPulmonaryvein
LeftatriumHeart

Enlarge rightventricle

Main veinLeftventricle

Rightventricle

MainarteryWhole
body

Source from website of Cardiovascular
Medicine, University Hospital Kyoto
Prefectural University of Medicine

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Cautionary Statement

This information material is provided for understanding Kidswell Bio
Corporation (“KWB”), not for soliciting investment in KWB shares.
Information provided in this material may contain so-called “forwardlooking statements.”
These statements are based on current
expectations, forecasts and assumptions that are subject to risks anduncertainties, which could cause actual outcomes and results to differ
materially from these statements. Risks and uncertainties include
success rate of R&D projects, new regulations and rules, relations withpartners in the future, etc.
This material includes information on pharmaceutical products andregenerative medicine (or related products), etc., which is being
developed or launched. However, this is not intended to promote our
products or provide medical advices.

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こどもの力になること、こどもが力になれること