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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