The Forefront of Regenerative Medicine

The Forefront of Regenerative

Medicine

HEALIOS K.K.

(TSE: 4593)Life Explosion

The Forefront of

Regenerative MedicineLife Explosion

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Accordingly, actual results may differ materially from these forward-

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The information about regenerative medicine products (including

products currently in development) which is included in this material is

not intended to constitute an advertisement or medical advice.

2

The Forefront of

Regenerative Medicine

Transforming

lives through

the power of

Regenerative

Medicine

Our Mission

Life Explosion 3

The Forefront of


Company Name HEALIOS K.K.

Mission Transforming lives through the power of regenerative medicine

Head OfficeYurakucho Denki Bldg. North Tower 19F,

1-7-1 Yurakucho, Chiyoda-ku ,Tokyo 100-0006, Japan

Chairman and CEO

Hardy TS Kagimoto, MD

A serial entrepreneur and obsessed with the concept of curing as

many patients as possible through technology. He founded Healios

with the vision to create a world leading regenerative cell therapy

company.

Stock Code 4593 (Tokyo Stock Exchange - MOTHERS market)

Number of Employees 145 (December 31, 2020）

Research Institution Kobe and Yokohama

Affiliated Company Sighregen Co., Ltd. (Joint Venture with Sumitomo Dainippon Pharma Co., Ltd.)

Subsidiaries

Healios NA, Inc. (Established in February 2018)

Organoid Neogenesis Laboratory, Inc.

(Established in June 2018 to promote the practical use of organ bud technology)

About HEALIOS K.K.

• Company Profile

4

The Forefront of


Healios Key Strengths

• Unique hybrid strategy combining near-term commercialization in Japan with development

of world-leading iPSC technology platform.

• Proactively leveraging Japanese regulatory framework for regenerative medicine.

• Two clinical studies nearing full enrolment: HLCM051 (MultiStem®) for ischemic stroke and

acute respiratory distress syndrome (ARDS).

• Proprietary, gene-edited universal donor iPSC platform technology (UDC).

• Developing innovative, next generation pipeline assets in immuno-oncology,

ophthalmology and organ buds.

• Deep and diverse team, supportive manufacturing partnerships, and strong financial

resources.

Key Strengths

5

The Forefront of


Strategy Overview

Strategy Overview

• Reinvest somatic stem cell product earnings into innovative IPSC platform

MultiStem iPSC Platform

MultiStem®

(Somatic stem cell product

obtained from

adult bone marrow)

Manufactur ing

iPSC

x

Immuno

Oncology

iPSC

X

Gene

Edit ing

GlobalJapan

New Products

Immuno-oncology

Ophthalmology

Organ buds

Reinvest

Global

Reinvest

6

Engage deeply with regenerative medicine innovation around the world through our venture fund activities

Important

informational insightsBuilding relationships with

promising companies

・Saisei Bioventures, L.P. launched in January 2021 and has made its first investments in Japan and

US private venture companies.

High return investments

The Forefront of


Confirm effectiveness

and safety

Establish presumed

effectiveness and confirm safety Approval with conditions and time limit

After-sales effectiveness

and further safety verification

Proactively Leveraging Japanese Regulatory Framework

For Regenerative Medicine

“Conditional and Time-Limited Authorization System”

• Offers an attractive regenerative medicine commercialization path

• Provides drastic reduction in trial duration and number of patients

• Coverage by national insurance scheme is possible

Traditional development process

Development process upon

introduction of early approval system

Clinical Research SalesApprovalClinical Trial

Clinical Research SalesOfficial

ApprovalClinical Trial

Early

ApprovalSales

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The Forefront of


Pipeline in Inflammatory Conditions, Immuno-oncology, and

Replacement Therapies

Deep & Diverse Pipeline

8

*1) NK Cells: Natural Killer Cells

Inflammatory

Conditions

Development IndicationCountry /

RegionPre-clinical Clinical trial Preparation for Apply/ On Market Progress status

Code test （ Regenerative medical products） application Approved

HLCM051

Ischemic Japan

Patient enrollment progress exceeds

90%Stroke

ARDS Japan Patient enrollment is completed

Immuno-

Oncology


RegionPre-clinical Phase 1 Phase 2 Phase 3 Preparation for Apply/ On Market Progress status

Code test trial trial trial application Approved

HLCN061Solid

Tumors

Japan

Research and development of

genetically modified NK cells(*1)

US/EUJoint research with the National

Cancer Center Japan

Replacement

Therapies


RegionPre-clinical Phase 1 Phase 2 Phase 3

Preparation

for Apply/ On Market Progress statusCode test trial trial trial application Approved

HLCR011 Wet AMD Japan Undergoing preparation for clinical trial

Joint development with Sumitomo Dainippon Pharma

HLCR012 Dry AMD US/EU

HLCL041Metabolic Liver

Disease JapanJoint research with Yokohama City

University

phase 2

phase 2/3SAKIGAKE Designation

System

Orphan regenerative medicine product

HLCM051 (MultiStem)

Two Clinical Studies

Near-Term Product Candidate:

Life Explosion

The Forefront of


Somatic Stem Cell Product Derived From Adult Bone Marrow

(MultiStem)

• In-licensed for the Japanese market from Athersys (Cleveland, OH, US)

• A significantly de-risked asset based on completed, successful, placebo-

controlled trials

‒ US/UK phase 2 trial in acute ischemic stroke published in Lancet Neurology1

‒ US/UK phase 1/2 trial in ARDS presented in May 20192

• Product profile

‒ Cell therapy product based on patented technology

‒ Developing for “off-the-shelf” IV administration; no tissue matching needed

‒ Long shelf life (stable frozen for years)

‒ Consistent safety profile

‒ Promotes healing and tissue repair through multiple mechanisms

‒ Not a permanent transplant: cells cleared from the body over time

HLCM051

1. Hess DC, et al. Lancet Neurol. 2017;16(5):360-368. 2. Bellignan G, et al. Presented at the 2019 American Thoracic Society International Conference.

Dallas, TX; May 20, 2019.

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The Forefront of


Athersys’ Phase 2 Study Shows Statistically Significant Improvement

With MultiStem vs Placebo1

HLCM051 for Acute Ischemic Stroke

1. Hess DC, et al. Lancet Neurol. 2017;16(5):360-368. BI, Barthel index; mRS, modified Rankin scale; NIHSS, National Institutes of Health Stroke Score.

Percentage of patients achieving an Excellent

Outcome (primary endpoint) was significantly greater

at Day 90 and Day 365 in patients who received

MultiStem within 36 hours of onset vs placebo.

• Data from MASTERS-1 phase 2 trial

• Excellent outcome (requires all 3):

– mRS score ≤1 (scale, 0 to 6)

– NIHSS score ≤1 (scale, 0 to 42)

– BI score ≥95 (scale, 0 to 100)

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The Forefront of


We are assessing the impact of the continued COVID-19 pandemic on our clinical trial progress

HEALIOS’ TREASURE Study in Acute Ischemic Stroke in Japan


Clinical Trial

(>40 sites opened)

First patient enrolled

Nov 2017

ApplyPreparationApproval/

Sales

Approval expected to take 6 to 12 months

under the SAKIGAKE Designation System

Due to the increase in COVID-19 infections in Japan, recent

patient enrollment progress has been slower than expected.

The current enrollment progress exceeds 90%.

• Placebo-controlled, double-blind, phase 2/3 efficacy and safety trial of HLCM051 (MultiStem) in patients with ischemic

stroke (NCT02961504)

• Subjects: 220 patients with ischemic stroke treated within 18 to 36 hours, randomized 1:1 to HLCM051 or placebo

• Primary endpoint: proportion of subjects with an excellent outcome (mRS ≤1, NIHSS ≤1, and BI ≥95) at Day 90]

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The Forefront of


Period after onset

Clot-dissolving agent

Mechanical reperfusion

HLCM051

10h 20h 30h 40h

• Caused by blockage of blood flow in the brain, resulting in tissue loss

• Affects an estimated 230,000 to 330,000 patients in Japan annually*

– 130,000 severely affected*

– 62,000 seen within 36 hours*

• HLCM051 could enable treatment of many more patients than current therapies

Acute Ischemic Stroke: Unmet Need and Potential in Japan


*HEALIOS estimates.

Treatment is time limited due to

risk of cerebral hemorrhage

Potentially longer treatment window

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The Forefront of


Results of Athersys’ Phase 1/2 Trial of MultiStem in ARDS• Athersys’ phase 1/2 clinical study in US and UK

shows further confirmation of tolerability and a

favorable safety profile associated with MultiStem

treatment

• Results from the double-blinded, placebo-controlled

study (MultiStem n=20; placebo n=10) after 28 days

of administration showed an improvement trend in

the group receiving MultiStem

• Post hoc analysis of patients in severe condition

with pneumonia-induced ARDS shows an even

greater difference

HLCM051 for ARDS

Overall analysis of phase 2

double-blind study

Post hoc analysis of patients in severe

condition with pneumonia-induced ARDS

MultiStem (n=20) Placebo （n=10) MultiStem Placebo

Mortality 25% 40% 20% 50%

Ventilator-free days 12.9 days 9.2 days 14.8 days 7.5 days

ICU-free days 10.3 days 8.1 days 12.0 days 5.0 days

（Source）Athersys

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The Forefront of


Clinical Trial

(~25 sites)

ApplyPreparationApproval/

Sales

HEALIOS’ ONE-BRIDGE Study in ARDS in JapanHLCM051 for ARDS

• Open-label, standard therapy-controlled, phase 2 efficacy and safety trial of HLCM051 (MultiStem) for Pneumonic Acute

Respiratory Distress Syndrome (NCT03807804)

• Subjects: 30 patients with pneumonia-induced ARDS randomized to HLCM051 (n=20) or standard therapy (n=10)

• Primary endpoint: number of days out of 28 in which a ventilator was not used (ventilator-free days)

April 2019

First patient enrolled

In November 2019, we were granted Orphan Regenerative Medicine Official Designation by the Ministry of

Health, Labour and Welfare.

In April 2020, we decided to make a protocol change to the ONE-BRIDGE study to include COVID-19 induced

ARDS patients as a separate cohort in the ongoing trial.

16

March 2021

Patient enrollment has been completed

The Forefront of


HEALIOS’ ONE-BRIDGE Study

(New Cohort for COVID-19 Induced ARDS Patients)

HLCM051 for ARDS

• Subjects: Approximately 5 Patients with pneumonia-induced ARDS caused by COVID-19

• Objective : Safety evaluation

The new group of patients with COVID-19 pneumonia (Cohort2) is separated from the ongoing treatment

group (Cohort1). The addition of this COVID-19 cohort should not effect the progress of the originally planned

clinical trial.

COVID-19

Test

Administration

of HLCM051

Cohort2

(The last patient was enrolled in August 2020)

Total enrollment: 5

Random

HLCM051

negative

Cohort1

(the ongoing clinical trial since April 2019)

20

10

ARDS

Patients

positive

Standard therapy

2:

1

17

The Forefront of


ARDS: Unmet Need and Potential in Japan

• Sudden onset of severe respiratory failure in seriously ill patients (typically 24 to

48 hours after illness or injury)

• Major causes include severe pneumonia, septicemia, trauma

• Activated inflammatory cells cause damage to lung tissue, leading to water

accumulation and acute respiratory failure

• Mortality rate: approximately 30% to 58%*1

• According to the data published on the initial group of cases of the new

coronavirus (COVID-19) in Wuhan, 31 to 41.8% of hospitalized patients

developed ARDS and ARDS complications were confirmed in 54 to 93% of fatal

cases *2 *3, indicating that ARDS is a major cause of mortality in COVID-19

patients.

• Standard treatment is ventilator use; requires ICU*4stay,

and prolonged ventilator use worsens prognosis

• No current therapeutic drugs

• Estimated 7,320 to 11,937 cases of ARDS annually in Japan (approximately 1/3

due to pneumonia)*5

HLCM051 for ARDS

*1 ARDS treatment guideline 2016 *2 Zhou F, et al. Lancet. 2020 Mar 11. pii: S0140-6736(20)30566-3 *3 Wu C , et al. JAMA Intern Med. 2020 Mar 13. doi: 10.1001*4 ICU, intensive care unit.*5 Respiratory Investigation; 55(4) : 257-263

(Source) Athersys

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The Forefront of


HLCM051: Preparation in advance of potential approval applications

HLCM051

19

① Basic business agreement with SPLine Corporation※ regarding the sale of pharmaceuticals ※SPLine Corporation is a wholly-owned subsidiary of MEDIPAL HOLDINGS CORPORATION ("MEDIPAL"). The MEDIPAL Group has storage

facilities and delivery systems capable of distributing specialty drugs, including regenerative medicine products. Its notable achievements in cell

medicine distribution include establishing the first wholesale distribution system in Japan for cells pharmaceuticals.

Wholly owned

Business & capital

allianceNikon

Nikon CeLL

innovation(Manufacturing in Japan)

Athersys

Product

manufacturing

Product

supply

Cost of

products

Sales royalty

Cold Chain Logistics

Hospitals

Distribution

② Healios applied for and obtained “GYOSHA Code” from the Ministry of Health, Labor and Welfare.

Gene Modified

iPSC Programs

Healios’s Cutting Edge Technology Platform

Life Explosion

The Forefront of


iPSC Platform: Leading the Development of Next-Generation iPSCsUniversal Donor Cells

21

・In October 2020, Healios established a clinical grade line that can

be clinically applied to humans in each of Japan, the United

States and Europe.

・Healios has led the development of high-quality, universal donor

iPS cells in accordance with global standards.

・Consultations with the FDA and PMDA led to no concerns in

relation to clinical use of UDC derived therapeutics.

・The UDC line differentiates readily into various in-house made

cells (e.g. NK cells, liver progenitor cells, vascular endothelial

cells, etc.).

・Active discussions with several companies and academic

institutions in relation to use with various therapeutic candidates.

Targeted cell programming through gene-editing

Allogeneic

hiPS cells

Immune response

• Heavy patient burden

• Short efficacy duration

• Reduce patient burden

• Increase efficacy

duration

Gene-

edited iPS

cell line

Reduce immune

response

Patient

Patient

Requires additional immunosuppressive drug

Healios Universal

Donor Cell Line

Allogeneic iPS Cell Line

Reduce or eliminate immunosuppressive drug requirement

Allogeneic

hiPS cells

World-leading engineered “universal” iPSC platform: “Universal Donor Cells” / “UDC”

The Forefront of



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Autologous

iPS cells

Allogeneic

iPS/ES cellsUDC

Immune

rejectionNone

Occurs

(Immunosuppressive drugs

are required）None

Manufacturi

ng term

Several months to 1 year

(Need to make

from each patient)

Off-the-shelf

（Single line）

Off-the-shelf

(Single line of

gene-edited cells)

Cost Very high Low Low

The Forefront of



23

Knock-out

HLA Class I

HLA Class II

Knock-in

gene X,Y,…

suicide gene

」

Universal iPSC regenerative medicines

・Off-the-shelf, scalable and cost-efficient

・Address broad population with single product

・Enhanced level and duration of efficacy

Clinical grade MCB HLA Class I/II KO

(intermediate)HEALIOS

Universal Donor Cells

house data

Engineered, universal iPS cells unlock full potential of iPSC therapies

The Forefront of


Universal Donor Cells

24

Parent-iPSC

Post-gene editing disappearance of HLA proteins and

enhanced expression of immunosuppression-related genes

■Control

■Target protein antibody

Clinical grade

UDC

less HighExpression

（Source）in-house data

HLA-A HLA-B HLA-C HLA-DR,DP,DQ Gene X Gene Y

HLA protein knock-outImmunosuppression-

related molecules knock-in

iPSC Platform: Removal of HLA Proteins and Addition of Immunosuppression-related Molecules

Results of gene editing in clinical grade UDC

The Forefront of


iPSC Platform: Safe and Versatile iPS CellsUniversal Donor Cells

25

iPSC pluripotency maintained

OCT-3/4+NANOG

46 (X,Y) Expression of Pluripotency Markers

No post gene-editing karyotypic

aberrations

Differentiation

OCT-3/4

（ Undefined Factor）NANOG

（ Undefined Factor）

Endothelial cell

Hepatocyte

（Source）in-house data

UDC

Characteristics of Clinical grade UDC

The Forefront of


iPSC Platform: Evaluation of UDC Inducible Suicide Genes In VivoUniversal Donor Cells

26

UDC

Transplantation

Immunodeficient

mouse

Administration of Drug Death of transplanted

cells

Mechanism of Action

Suicide gene

Administration of a drug

induces dimerization

Induction of

Cell DeathDimerization

Confirmed suicide gene activity in immunodeficient mice

The Forefront of


iPSC Platform: Evaluation of UDC Inducible Suicide Genes In VitroUniversal Donor Cells

27

Death of UDCs

Culture of UDCs

0

0.2

0.4

0.6

0.8

1

1.2

0 0.003 0.01 0.03 0.1 0.3 1

ratio

(liv

e/t

ota

l)

Inducer of dimerization (nM)

Cell viability (ATP assay)

Reduction in Viability

After induction of suicide genes, target cells die by apoptosis （Source）in-house data

Administration

of Drug

The Forefront of


iPSC Derived Gene-Modified Natural Killer Cells iPS Gene Modified NK Cells

(Time)

(Technological development）

1st Generation 2nd Generation 3rd Generation

“Autologous”

Peripheral

blood derived

“Allogenic /

off-the-shelf”

Biomaterial or

iPSC derived

“Universal Donor Cell

derived"

Function enhanced by

multiple gene

modifications

• By using UDC (MHC class I/II KO), graft rejection may be avoided, and sustained efficacy may be expected.

• Stable production and quality with lower cost of goods are expected by using iPSC and cryopreservation is

available as an off-the-shelf product.

28

In June 2020 we started joint research with the National Cancer Center Japan (NCCJ)

on Healios’ allogeneic iPS cell-derived gene-edited NK cells

The Forefront of


Healios: Cancer is the Leading Cause of Death in Japan

iPS Gene Modifiied NK Cells

29

The No.1 cause of death in Japan is cancer

(approximately 90% of which are caused by solid tumors)

Mortality rate

Blood cancer

Solid Tumors

The Forefront of


Healios: Leading the Development of iPSC Derived Gene-Modified

Natural Killer CellsGene editing

Avoid rejection /

Functionally enhanced

UDC

NK cell

Solid Tumors

Dendritic Cell

Cytotoxic

T-lymphocyteDifferentiation

Activation


Migration /

Infiltration

Recognize and

attack

30

• Best in class, enhanced anti-tumor efficacy by

introducing/modulating various factors related to NK

cells killing activity

• Broad applicability across tumor types irrespective of

specific cancer antigens

NK cellsUDC

Production of NK cells

Induction of

differentiation

The Forefront of


Healios: Mechanism of NK Cell Attack of Cancerous or Virus-Infected

Cells


② Release the brake allowing for

the attack

Normal cells Cancerous or virus-infected cells

Normal cellNK cell

NK cells do not attack normal cells by

recognizing normal marker

NK cell Cancer cell

Virus-infected cell

① Activated by abnormal markers

③ Recognize the antibodies that are

attacking the cancer and further activate.

④ Release the degrading enzymes

and destroy cancer cells

• NK cells are large granular lymphocytes (LGL) and critical to the innate immune system. The role of NK cells is to

recognize and attack abnormal cells, such as cancer cells and virus-infected cells.

31

The Forefront of



Enhancement of Anticancer Functions through Gene Modification

32

HLCN061

Cancer-Immunity Cycle

NK cells recognize and kill cancer cells

Exposed to cancer antigen

Activation of the cancer immunity cycle and induction of cytotoxic T-lymphocytes

Degeneration of cancer

Antigen

presentation

T-cell activation

Migration

Invasion

Recognition

Cellular dysfunction

vessel

（Source） This material was based on Daniel S.Chen and Ira Mellman.,Immunity. 2013;39(1):1-10.

Enhancing Anticancer Function

at Each Stage of the Cancer-Immunity Cycle

Antigen exposure by injury

cancer

lymph

The Forefront of


Healios: Joint research with the National Cancer Center Japan


34

Gene edited

NK cellsPDX

National Cancer

Center Japan

Research utilizing PDX（Patient-Derived Xenograft）

*1 PDX models

Transplant human patient cancer tissue into immunodeficient mice

Dramatically improves the predictability of clinical response

PDX models ＊１ will be used to consider what solid cancers we

should target with our therapy.

Based on the results of these studies

・Clarify the characteristics of solid cancers to which HLCN061

exerts antitumor effects

・Investigate the expression of several molecules recognized

by HLCN061

Healios joint research with the NCCJ started in June 2020 and will last for about one year.

The Forefront of


iPSC Regenerative Medicine

35

iPSC Regenerative Medicine Treatments to Replace Damaged Cells

or Organs

HLCR011/HLCR012:

Ophthalmology

HLCL041: Liver DiseaseGenerating “organ buds” by

co-culturing 3 types of cells

iPS cell

MSC, mesenchymal stem cell; RPE, retinal pigment epithelium.

The Forefront of



36

HLCL041: Liver Organ Bud Platform

Transplanted to mice

Green：Cells of each organ

Red：Vascular endothelial cell

Black：MSC

*movie

The vascularization was confirmed in vivo by transplantation to mice.

Cell derived from

various organs

Vascular

endothelial cellMSC

（Sours） 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

By creating an "Organ Bud" of each organ with iPS cells, we have laid the groundwork for paradigm shifting therapies to

emerge for various severe diseases.

UDCs allow for the realization of organ replacement using organ buds.

（Sours）Modified from Takebe T. et al., Cell Stem Cell, 2015

The Forefront of



37

HLCL041: Liver Organ Bud Platform

Treatment effects of liver bud transplantation to mouse using hiPSC

（Source） Adapted by Healios from Takebe. T, et al. Nature, 499 (7459),（2013)

Process by which organ forms from

organ bud links mouse‘s vascular

network autonomously

Su

rviv

al ra

te (

%)

100

80

60

40

20

2010 300Days

Non-transplantation

Liver organ budtransplantation group using human iPS cells

Human adult liver cell transplantation group

Liver organ budtransplantation group using human fetus iPS cells

Survival rate improves significantly in transplantation experiments

（Source）Takebe,T., et al. Nature Protocols, 9, 396–409 (2014)

Process

Healios Resources

Life Explosion

The Forefront of


Diverse Leadership Team

Jun

Narimatsu

Richard

Kincaid

David

Smith

Michael

Alfant

Gregory

Bonfiglio

Yoshinari

Matsuda

Seigo

Kashii

Accountant

Supporting various

venture companies in

the field of IT/

Healthcare

Chief Financial

Officer

Member of Board of

Directors

Cell manufacturing

KOL

.

Group Chairman &

CEO, Fusion

Systems, Co., Ltd.

Presidents Emeriti,

ACCJ

Founder & Managing

Partner of Proteus,

LLC. (Investment in

RM ventures)

Chairman of the

Board of CCRM

Attorney-at-Law,

Senior Management

Partner of Uruma

Law Offices Legal

Professional

Corporation

Ex-corporate auditor

of Astellas Pharma

Masanori

Sawada

Hardy TS

Kagimoto

Kouichi

Tamura

Michihisa

Nishiyama

Koji

Abe

Executive Vice President

Chief Medical Officer

Administrative field

Chairman and CEO Executive officer,

Research and

Manufacturing field

Executive officer,

Development field

Executive Officer

HR & GA field

MD, PhD, MBA MD, Founder Ex-Astellas US Director

of Laboratories

Expertise in

Immunosuppressant

Research

Constructed network for

tacrolimus approval and

sales at Astellas (US and

Europe)

Over 30 years experience

in pharmaceutical HR field

39

The Forefront of


Capabilities

Total Number of Staff 89 (As of September 30, 2020)

Ph.D. Holders Over 30 people

Kobe Research Institute

Affiliate company for Manufacturing: Sighregen

Sighregen, a joint venture with Sumitomo Dainippon Pharma, is

establishing a manufacturing facility Sighregen rents the facility in

SMaRT and is establishing the iPSC-RPE manufacturing system

and facility for iPSC-RPE cell products.

40

Research and Manufacturing Capabilities

The Forefront of


Capabilities

Business and Capital Alliance with Nikon

41

Expanded the alliance with Nikon in July, 2019 to pursue regenerative medicine growth opportunities

Overview of the Business Alliance

<Healios>

- Promoting the search and development of new seeds in the regenerative medicine field

- Cooperating in relation to manufacturing currently being undertaken by Nikon

- Beginning of discussions to consider cell contract manufacturing for various products

Healios is developing in house

<Nikon>

- Supporting the development from the perspective of cell contract manufacturing and image

evaluation for cells

History of Partnership with Nikon

2017

2017

2019

2013

Nikon CeLL innovation entered into

Agreement to support MultiStem

commercial manufacturing efforts

in Japan

Concluded the business and capital

alliance Allotted 1,037,400 shares of

Healios stock and received 2 billion yen

from Nikon

Expanded the business and capital

Alliance Allotted 40 units of convertible

bonds and received 4 billion yen

from Nikon

Started R&D using Nikon’s

image evaluation technology

Allotted 500,000 shares of Healios

stock and received 500 million yen

from Nikon

Wholly owned

Business & capital

alliance

HLCM051 Manufacturing Framework

Nikon

Nikon CeLL innovation

(Manufacturing in Japan) Athersys

Product

manufacturing

Product

supplyCost of products

Sales royalty

sales

The Forefront of


• Founded in 2011, A-round in 2013, TSE listed in 2015

• Over $300 million raised since inception

• Approximately $134.53 million of cash on balance sheet as of December 31, 2020 (burn rate

approximately $10-15 million per quarter)

• July 2019 cap raise via Goldman Sachs was world’s first zero-coupon global convertible

bond financing for pre-revenue Japanese biotech. Approx. $107mn total net proceeds raised

from combination of CBs and equity to international investors along with CB issued to Nikon.

Cash Position

Strong Financial Resources

42

Conclusion

Life Explosion

The Forefront of


Conclusion

• Unique hybrid strategy combining near-term commercialization in Japan with

development of world-leading iPSC technology platform.

• Proactively leveraging Japanese regulatory framework for regenerative medicine.

• Two clinical studies nearing full enrolment: HLCM051 (MultiStem®) for ischemic stroke

and acute respiratory distress syndrome (ARDS).

• Proprietary, gene-edited universal donor iPSC platform technology (UDC).

• Developing innovative, next generation pipeline assets in immuno-oncology,

ophthalmology and organ buds.

• Deep and diverse team, supportive manufacturing partnerships, and strong financial

resources.

44

The Forefront of Regenerative Medicine

Corporate Communications

HEALIOS K.K.

E-mail: [email protected]

https://www.healios.co.jp/en

The Forefront of Regenerative Medicine

45

mailto:[email protected]

https://www.healios.co.jp/en

Documents

The Forefront of Regenerative Medicine