FY2023 Results Briefing Session
May 13th, 2024
JCR Pharmaceuticals Co., Ltd.
[Securities code]4552, Prime. TSE
Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Key Messages
FY2023
Revenue and earnings growth from increasing sales of core products
FY2024
Strengthen organizational structure to support global clinical development and increase R&D investment
- Expand clinical development team and establish an integrated structure of experts in Japan, US, Europe and Brazil with JCR Europe playing a lead role
- JR-141:Complete patient enrollment for an interim analysis of the global Phase III
- JR-441:Phase I/II study on track (First patient dosed in Oct 2023)
- JR-446:Initiate Phase I/II study in 1st half of FY2024
- JR-171:Discussions on licensing-out ongoing
Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. | FY2023_4Q_1 | ||
Progress of Developmental Pipelines
Anne Bechet
Development (Acting Officer) General Manager, JCR Europe B.V.
Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. | FY2023_4Q_2 |
JR-141 (pabinafusp alfa: BBB-penetrating ERT for MPS II)
Global Phase III study(JR-141-GS31): STARLIGHT study Overview
Overview
1. | To assess the efficacy of JR-141 on CNS signs and symptoms in | |
Objectives | MPS-II subjects relative to standard ERT | |
2. | To assess control of somatic signs and symptoms by JR-141 | |
relative to standard ERT | ||
• Changes in HS in CSF, CNS symptoms (cognitive, behavior, | ||
Endpoints | attention) | |
• Control of systemic sign and symptoms | ||
Clinical Trails.gov | Identifier : NCT04573023 | |
Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Current Status
Recruiting
Number of Clinical trial sites (as of Apr 2024):
- US: 5
- Europe: 12
- Latin America: 4
Trial is on going in 10 countries
- Further sites to open in EU, US, Latin America to accelerate recruitment
Achievements
Oct -2018ODD by FDA
Feb -2019ODD by EMA
Feb -2021 Fast Track Designation by FDA Oct -2021 PRIME Designation by EMA
Feb -2022 First Patient dosed in JR-141-GS31
Dec -2022 Rare Pediatric Disease Designation by FDA
- Completed enrollment of all 20 eligible patients in Cohort B
- Enrollment in Cohort A on track
FY2023_4Q_3
JR-441(BBB-penetrating ERT for MPS IIIA)
July 2023 - Started Global Phase I/II study (JR-441-101) in Germany
JR-441-101 study overview
Treatment | Drug | |
Extension period | ||
period | holiday | |
Achievements and next milestones
Jan -2022
EC grants Orphan Drug Designation
Jul -2023
Dose 2
Screening
Dose 1
Baseline
6 | Optimal dose * |
weeks |
- Optimal dose will be determined after all patients' 27-week data are obtained and analyzed.
week
53 | 59 | 261 |
Approval of Global Ph I/II Clinical Trial in Germany
Oct -2023
First Patient First dosed
Dec -2023
FDA grants Orphan Drug Designation
1st Half -FY2024
Last Patient In
2nd Half -FY2025
Overview
Objectives | Safety, dose finding, exploratory efficacy |
No. of subjects | 12 subjects (≥1 year and ≤18 years) |
Clinical Trails.gov | Identifer : NCT06095388 |
1-year clinical data is expected
- The Phase I/II study is progressing well
- Started recruiting pediatric patients after approval from the independent data monitoring committee
Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. | FY2023_4Q_4 | ||
R&D Pipeline
Code
Indication
Status
Upcoming Milestones
• Q1 FY2024: All patients enrolled | |||||
JR-141 | MPS II (Hunter syndrome) | Global Ph3 | necessary for interim analysis | ||
• ~FY2027: Approval in US, EU, Brazil | |||||
JR-171 | MPS I (Hurler syndrome etc.) | Global Ph1/2 completed | • Extension study is ongoing | ||
• | Licensing out under negotiation | ||||
JR-142 | Pediatric GHD | Ph2 (Analysis completed) | • | FY2024: Phase 3 | |
JR-031HIE | Hypoxic ischemic encephalopathy in neonates | Ph1/2 (Analysis completed) | • | TBD | |
• 1st Half FY2024: LPI | |||||
JR-441 | MPS IIIA (Sanfilippo syndrome type A ) | Global Ph1/2 | • | 2nd Half FY2025: 1-year clinical data is | |
expected | |||||
JR-446 | MPS IIIB (Sanfilippo syndrome type B ) | Pre-clinical | • Under preparation for clinical trial | ||
• | 1st Half FY2024: FPI in Phase 1/2 | ||||
JR-471 | Fucosidosis | Pre-clinical | • | TBD | |
※Only high-priority projects in the clinical stage or soon to be in the clinical stage are listed in the above table.
Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. | FY2023_4Q_5 | ||
Next Wave of Technology Innovation
Hiroyuki Sonoda, Ph. D.
Vice President
Executive Director, Research Division
Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. | FY2023_4Q_6 |
History of Technological Innovation
Advanced technology and know-how in biopharmaceuticals
developed since the company's formation in 1975
Stock solutions for COVID-19
Vaccine (AZD1222)
J-Brain Cargo® Tech
Biosimilars
Stem Cell
Therapies
Recombinant Proteins
Human-derived therapeutics
Development of proprietary
JCR
technologies
Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. | FY2023_4Q_7 | ||
Applying J-Brain Cargo® to Gene Therapy
Treatment with AAV expressing
JBC-fusion protein
AAV capsid
JBC | X |
Therapeutic | |
Gene cassette | protein X |
Treatment with modified AAV
expressing JBC tag for direct brain penetration
X
JCR proprietary gene therapy technology applying J-Brain Cargo® Treatment strategies can be tailored depending on the disease characteristics
Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. | FY2023_4Q_8 | ||
Characteristics of Gene Therapy used J-Brain Cargo®
Treatment with AAV expressing | Treatment with AAV | General AAV gene therapy |
JBC-fusion protein | carrying JBC surface tag | |
- Transgene product, JBC-X, can reach the CNS
- Mechanism of action has been established in enzyme replacement therapy
AAV/JBC-X
JBC
X
• JBC-AAV can cross the blood- | X | • High doses are |
required to penetrate | ||
brain barrier | X | |
the CNS | ||
• Applicable to a membrane | ||
protein since treatment | ||
protein can be expressed in | ||
every organ including the | ||
CNS | Blood-Brain Barrier | |
JBC-AAV/X | AAV/X |
JBC
X
X
X
Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. | FY2023_4Q_9 | ||
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JCR Pharmaceuticals Co. Ltd. published this content on 13 May 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 13 May 2024 13:42:24 UTC.