Slide 1: FY2022 Results Briefing Session - Research and Development Highlights
FY2022 Results Briefing Session - Research and Development Highlights - May 12, 2023 JCR Pharmaceuticals Co., Ltd. [Securities code]4552, Prime. TSE Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 2: Highlights (Oct. 27, 2022- May 11, 2023)
Highlights (Oct. 27, 2022- May 11, 2023) LSD：lysosomal storage disease 2022 MPS：mucopolysaccharidosis ♦Oct. －JR-471 for Fucosidosis enters Development －Exclusive Negotiation Rights for Global Commercialization Targeting Four Ultra-Rare Diseases and Conclude a Licensing Contract for the Commercializa
Slide 3: Agenda
Agenda ❚ Progress of Research and Development ❚ Upcoming Development Schedule Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. Business 2
Slide 4: Progress on R&D Products Applying J-Brain Cargo Technology
Progress on R&D Products Applying J-Brain CargoⓇ Technology Progress of internal R&D for LSD therapeutics Contributing to a wide range of disease areas J-Brain Cargo® other than LSDs Application to various modalities Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. Business 3
Slide 5: Progress on R&D Products Applying J-Brain Cargo Technology
Progress on R&D Products Applying J-Brain CargoⓇ Technology Progress of internal R&D for LSD therapeutics Contributing to a wide range of disease areas J-Brain Cargo® other than LSDs Application to various modalities Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. Business 4
Slide 6: JR-141 Long-term results①
JR-141 Long-term results①（Presented at WORLDSymposiumTM 2023） IZCARGO®（Brand name in Japan） JR-141 pabinafusp alfa: BBB-penetrating iduronate-2-sulfatase (rDNA origin) Please follow the link provided below BBB: Blood-brain barrier for the content of the WORLDSymposiumTM2023 presentation giugliani-16
Slide 7: JR-141 Long-term results②
JR-141 Long-term results②（Presented at WORLDSymposiumTM 2023） IZCARGO®（Brand name in Japan） JR-141 pabinafusp alfa: BBB-penetrating iduronate-2-sulfatase (rDNA origin) Please follow the link provided below for the content of the WORLDSymposiumTM2023 presentation giugliani-16-9-poster-05b.pdf Attenua
Slide 8: Progress on JR-171
Progress on JR-171 JR-171 lepunafusp alfa: BBB-penetrating α-L-iduronidase (rDNA origin) Design of Global Phase 1/2 Clinical Trial（JR-171-101) ◆ Apr. 2023: Dosing has been completed and data analysis is ongoing Part 1 N=4 Part 2 N=14 or more ✓ 18 years or older ✓ 0 years or older ✓ No or mild intell
Slide 9: JR-171 Ph1/2 Clinical trial results①
JR-171 Ph1/2 Clinical trial results① （Presented at WORLDSymposiumTM 2023） JR-171 lepunafusp alfa: BBB-penetrating α-L-iduronidase (rDNA origin) Summary of Global Phase 1/2 Clinical Trial（JR-171-101) All 7000 HS concentration in CSF (ng/mL) 6000 Low dose(n=6) 5000 High dose(n=8) 4000 3000 2000 1000 C
Slide 10: JR-171 Ph1/2 Clinical trial results②
JR-171 Ph1/2 Clinical trial results② （Presented at WORLDSymposiumTM 2023） JR-171 lepunafusp alfa: BBB-penetrating α-L-iduronidase (rDNA origin) Summary of Global Phase 1/2 Clinical Trial（JR-171-101) Low dose (n=6) High dose (n=7) 40 Serum heparan sulfate 40 Serum dermatan sulfate High dose; laronida
Slide 11: JR-171 Ph1/2 Clinical trial results③
JR-171 Ph1/2 Clinical trial results③ （Presented at WORLDSymposiumTM 2023） JR-171 lepunafusp alfa: BBB-penetrating α-L-iduronidase (rDNA origin) Summary of Global Phase 1/2 Clinical Trial（JR-171-101) Improvements observed in Phase 1/2 Language Motor Other domains – Expression and – Gait – Obstructive
Slide 12: JR-441 Preclinical Validation①
JR-441 Preclinical Validation① （Presented at WORLDSymposiumTM 2023） JR-441 BBB-penetrating heparan N-sulfatase (rDNA origin) • JR-441 dose-dependently decreased HS concentrations in CSF and brain • Both JR-441 and rhSGSH decreased HS concentrations in serum and peripheral tissues Brain Serum HS conc
Slide 13: JR-441 Preclinical Validation②
JR-441 Preclinical Validation② （Presented at WORLDSymposiumTM 2023） JR-441 BBB-penetrating heparan N-sulfatase (rDNA origin) Biodistribution of JR-441 in the CNS of Cynomolgus Monkeys Cerebellum JR-441 Neuronal cells (Purkinje cells) rhSGSH JR-441 was distributed across various regions of the brain
Slide 14: Our responsibility for Ultra-Rare Diseases
Our responsibility for Ultra-Rare Diseases ✓ A particularly small group of patients with LSDs Difficult for large companies Generally no established to enter the market due to standard of care profitability “Creation of new therapeutics” and “System for providing global patients” Contribution to Ult
Slide 15: Global Approach to Ultra-Rare Diseases
Global Approach to Ultra-Rare Diseases Oct. 2022：Signed an agreement with MEDIPAL HOLDINGS for the global commercialization of biotherapeutics ultra-rare diseases Granting of exclusive negotiation rights for the global commercialization of four ultra rare diseases. Conclusion of licensing agreement
Slide 16: Progress on R&D Products Applying J-Brain Cargo Technology
Progress on R&D Products Applying J-Brain CargoⓇ Technology Progress of internal R&D for LSD therapeutics Contributing to a wide range of disease areas J-Brain Cargo® other than LSDs Application to various modalities Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. Business 15
Slide 17: Application for CNS Diseases other than LSDs①
Application for CNS Diseases other than LSDs① Mar. 2023：Concluded research collaboration, option and license agreement with Alexion to develop the treatment using J-Brain CargoⓇ for neurodegenerative disease The first international partnership to apply the J-Brain Cargo® technology for the treatment
Slide 18: Application for CNS Diseases other than LSDs②
Application for CNS Diseases other than LSDs② May 2023 ： Concluded an agreement of global collaboration for the development and commercialization of novel biologic therapies in epilepsy ✓ Epilepsy is thought to affect more than 50 million people worldwide* ✓ JCR will also be eligible to receive addi
Slide 19: Progress on R&D Products Applying J-Brain Cargo Technology
Progress on R&D Products Applying J-Brain CargoⓇ Technology Progress of internal R&D for LSD therapeutics Contributing to a wide range of disease areas J-Brain Cargo® other than LSDs Application to various modalities Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. Business 18
Slide 20: Application to Various Modalities
Application to Various Modalities Mar. 2023： Achievement of preclinical proof-of-concept milestone using J-Brain Cargo® technology for LSD in gene therapy collaboration with Takeda Enzymes and Oligonucleotide Proteins Delivery (ASO, siRNA) Delivery LNP (mRNA, low- Antibody Delivery molecular compoun
Slide 21: Agenda
Agenda ❚ Progress of Research and Development ❚ Upcoming Development Schedule Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. Business 20
Slide 22: JCR's LSD Pipeline Programs applied J-Brain Cargo
JCR's LSD Pipeline comprises 17 Programs applied J-Brain Cargo® Approved JR-141 Japan MPS II （Hunter） MPS II *1 MPS I Clinical JR-141 Global （Hunter） JR-171 Global （Hurler, etc） Non- JR-162 Pompe JR-441 MPS IIIA （Sanfilippo A） JR-446 MPS IIIB （Sanfilippo B） Clinical JR-443 MPS VII JR-479 GM2 Ganglio
Slide 23: Development Portfolio
Development Portfolio ★Updated Code Indication Status Next Milestones Remarks Approved - • SAKIGAKE/ Orphan Drug MPS type II • Conclusion of exclusive collaboration and license JR-141 (Hunter Syndrome) ~FY2027 agreement in Global specific regions with TAKEDA Phase 3 Approval in US, EU, Brazil • US:
Slide 24: Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 25: Forward-Looking Statement
FORWARD-LOOKING STATEMENT This presentation contains forward-looking statements that are subject to a number of risks and uncertainties, many of which are outside our control. All forward-looking statements regarding our plans, outlook, strategy and future performance are based on judgments derived
Slide 26: Forward-Looking Statement
FORWARD-LOOKING STATEMENT The clinical development data mentioned in this document do not guarantee future results, nor do they guarantee the efficacy or effects of products under development. This document is not intended to guarantee or advertise the efficacy of the product under development. The
Slide 27: Appendix
Appendix Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 28: Pipeline of LSDs
Pipeline of LSDs JR-141 pabinafusp alfa: BBB-penetrating iduronate-2-sulfatase (rDNA origin) Indication : MPS type II (Hunter syndrome) Patient population*¹ : 250 (Japan) , 7,800 (WW) est. Est. Market size*² : 7.6 billion JPY (2019 Japan), 87.0 billion JPY (2019 WW) Disease overview : Hunter syndrom
Slide 29: Pipeline of LSDs
Pipeline of LSDs JR-441 BBB-penetrating heparan N-sulfatase (rDNA origin) Indication : MPS type III A (Sanfilippo A syndrome) Patient population*¹ : 30 (Japan : Total of Type A&B）, 4,000 (WW) est. Est. Market size*² : >70.0 billion JPY (2019 WW: Total of Type A&B) Disease overview : An autosomal rec
Slide 30: Pipeline of LSDs
Pipeline of LSDs JR-443 BBB-penetrating β-glucuronidase (rDNA origin) Indication : MPS type VII（Sly syndrome） Patient population*¹ : several (Japan) , 200 (WW) est. Est. Market size*² : 9.8 billion JPY est.（2019 WW） Disease overview : An autosomal recessive disease caused by deficiency of an enzyme,
Slide 31: Pipeline of LSDs
Pipeline of LSDs JR-479 BBB-penetrating β-Hexosaminidase A (rDNA origin) Indication : GM2 gangliosidosis (Tay-Sachs disease, Sandohoff disease) Patient population*¹ : 30 (Japan), TBD(WW) est. Est. Market size*² : TBD Disease overview : GM2 gangliosidosis is an autosomal recessive LSD caused by a def
Slide 32: Pipeline of Other Compounds
Pipeline of Other Compounds JR-142 Long-acting growth hormone (rDNA origin) Indication : Pediatric growth hormone deficiency Note : JCR’s proprietary half-life extension technology, based on a novel modified albumin, allows significant increase in the half-life of various biotherapeutics （Patent fil
Slide 33: Progress on JR-141 (JR-141-GS31)
Progress on JR-141 (JR-141-GS31) IZCARGO®（Brand name in Japan） JR-141 pabinafusp alfa: BBB-penetrating iduronate-2-sulfatase (rDNA origin) ◆ Expansion of clinical trial sites in the US, Brazil, and Europe and patient enrollment is in progress. Also, prepare the initiation of the study in several new
Slide 34: Progress on JR-141 (JR-141-GS31)
Progress on JR-141 (JR-141-GS31) IZCARGO®（Brand name in Japan） JR-141 pabinafusp alfa: BBB-penetrating iduronate-2-sulfatase (rDNA origin) Objective : JR-141-GS31 1. To demonstrate the significant efficacy of JR-141 on CNS signs and symptoms in MPS-II subjects relative to standard ERT. JR-141 is exp
Slide 35: Leveraging Growth Opportunities from J-Brain Cargo Technology
Leveraging Growth Opportunities from J-Brain Cargo® Technology Partnerships are at the core of JCR’s growth and acceleration strategy LSD: Lysosomal diseases CNS: Central central nervous system Internal Expand/build footprint in USA and Europe Development LSD Portfolio Partner focus: Partnerships Sy
Slide 36: Design Uniqueness of the J-Brain Cargo Technology
Design Uniqueness of the J-Brain Cargo® Technology Versatile binders, affinities, Affinity to TfR: epitopes 0.1 nM 10~100x higher affinity to BBB Applicable to multiple modalities Affinity to M6PR: P P 1 ~ 10 nM TfR: Transferrin receptor M6PR: Mannose-6-phosphate receptor BBB: Blood-brain barrier Di