FY2021 Q3 Financial Results

Company

HEALIOS K.K. (TSE 4593)

Date

November 12, 2021

1.

Strategy/Updates

| 02

2.

HLCM051 ARDS

| 11

3.

HLCM051 Stroke

| 19

4.

iPSC Platform

| 24

5.

HLCN061 Immuno-oncology (NK Cells)

| 33

6.

HLCR011 AMD/

| 42

HLCL041 Liver Organ Bud Platform

7.

Financial Highlights

| 46

8.

Appendix

| 51

1

Hybrid Strategy

  • Generate near term profits in stroke and ARDS indications
  • Reinvest profits in our world-leading engineered iPSC platform to create next generation therapies for the global market

HLCM051

iPSC Platform

New Products

Global

Japan

Global

iPSC

Immuno-oncology

X

Ophthalmology

Gene

STROKE

Editing

Organ buds

ARDS

…

(Somatic stem cell product

iPSC

obtained from

Reinvest

Manufacturing

x

Reinvest

…

adult bone marrow)

Immuno

Oncology

…

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

Important

Building relationships

High return

informational insights

with promising companies

investments

Saisei Ventures LLC (US based) has made its initial investments in Japan and US private venture companies.

© HEALIOS K.K. All rights reserved.

2

Update

Pipeline

Clinical pipeline

Development

Country/ Pre-clinical

Clinical trial

Preparation for

Apply/

On Market

Indication

（ Regenerative medical products）

Code

Region

application

Approved

test

Inflammatory

Ischemic

Phase2/3

SAKIGAKE Designation

Conditions

Japan

Stroke

System

HLCM051

Non-clinical / CMC package submitted

Orphan regenerative

ARDS

Japan

Phase2

medicine product

Progress status

Patient enrollment completed Rolling submission in progress via SAKIGAKE Designation System

Preparing for application

Development

Indication

Country/

Pre-clinical

Phase 1

Phase 2

Phase 3

Preparation for

Apply/

On Market

Progress status

Code

Region

test

trial

trial

trial

application

Approved

Immuno-

Japan

Research and development of

Oncology

HLCN061

Solid Tumors

genetically modified NK cells

US/EU

Joint research with the National

Cancer Center Japan

* NK Cells: Natural Killer Cells

Development

Indication

Country/

Pre-clinical

Phase 1

Phase 2

Phase 3

Preparation for

Apply/

On Market

Progress status

Code

Region

test

trial

trial

trial

application

Approved

Replacement

HLCR011

Wet AMD

Japan

Preparing for clinical trial

by Sumitomo Dainippon Pharma

Therapies

HLCR012

Dry AMD

US/EU

HLCL041

Metabolic Liver

Japan

Joint research with Yokohama City

Disease

University

Research pipeline

Development

Target Organ

Country/

Pre-clinical

Phase 1

Phase 2

Phase 3

Preparation for

Apply/

On Market

Progress status

Code

Region

test

trial

trial

trial

application

Approved

UDC Platform

－

Pancreatic β

－

Joint research with the National Center

Cells

for Global Health and Medicine

－

Photoreceptor

－

Cells

© HEALIOS K.K. All rights reserved.

3

Update

HLCM051 Stroke: TREASURE Study

Based on the advice of the regulatory authority, in order to avoid any potential bias to the 365 -day data (and related secondary endpoints) that could result from unblinding and disclosure of 90 -day data (primary endpoint), the decision was made that the 90 -day unblinding, data analysis and release would

take place after the 365-day data is locked.

Overview

Development Plan

March 2022

March 2021

Last patient final hospital

Last patient treated

follow up visit targeted

November 2017

August 2021

Patient enrollment

Q2 2022

First patient enrolled

completed

Key-open*

Observation

Data analysis

Approval /

Patient enrolment

Preparation

Apply

Sales

Non-clinical / CMC package submitted Consulting with regulatory authorities

The approval period may be shortened from 12 months to 6 months by the

SAKIGAKE Designation System

  • "Key-open"is the process of unblinding the data, after which analysis can be completed. Results will be released promptly post key-open and completion of analysis.

Trial

Placebo-Controlled,Double-Blind,

Phase 2/3 Efficacy and Safety Trial

of HLCM051 (MultiStem®) in

Patients With Ischemic Stroke

（TREASURE study）

Subjects

Ischemic stroke within 18 to 36

hours

Conditions

Placebo-Controlled,Double-Blind

Enrollment

220 (HLCM051 [n=110], placebo

[n=110], randomized)

Primary

Proportion of subjects with an

Endpoints

excellent outcome defined by

functional assessments

[ Time Frame: Day 90 ]

Secondary

Proportion of subjects with an

Endpoint

excellent outcome defined by

(one among

functional assessments

several)

[ Time Frame: Day 365 ]

"Excellent Outcome" is defined as achieving mRS ≤1, NIHSS ≤1, and BI ≥95. mRS, NIHSS, and BI are the three major indices of functional assessment for stroke patients.

© HEALIOS K.K. All rights reserved.

4

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Healios KK published this content on 12 November 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 12 November 2021 06:36:16 UTC.