Jcr Pharmaceuticals Co., Ltd. · TSE:4552

Jcr Pharmaceuticals — H1 FY2020 Earnings Presentation

38 slides from Jcr Pharmaceuticals's earnings presentation, Nov 2, 2020

  1. Slide 1: FY2020 First-Half Results Briefing Session - R&D Highlights

    FY2020 First-Half Results Briefing Session - Research and Development Highlights - November 2, 2020 JCR Pharmaceuticals Co., Ltd. Copyright © 2020 JCR Pharmaceuticals Co., Ltd. All rights reserved. 【Securities code】 4552, 1st Sec. TSE

  2. Slide 2: Development Pipeline

    Development Pipeline ■■ Lysosomal Storage Disorders (LSDs) ■ Other Recombinant Protein Therapeutics Regenerative Medical Product Code Indication Preclinical Clinical trials Filed Approved Remarks MPS type II Filed • ERT JR-141 • J-Brain Cargo® (Hunter Syndrome) Phase 2 completed • ERT MPS type I JR-

  3. Slide 3: Contents

    Contents Research & Development News (Jun.-Oct.) JR-141 Development Status Other Pipeline Products Copyright © 2020 JCR Pharmaceuticals Co., Ltd. All rights reserved. R&D 2

  4. Slide 4: Research & Development News (Jun.-Oct.)

    Research & Development News (Jun.-Oct.) 2020 Jun. 17：Agalsidase Beta BS. I.V. Infusion for Fabry Disease: Publication of The Clinical Trial Results for Fabry Disease in Molecular Genetics and Metabolism Jul. 31：Completion of Phase 1/2 Clinical Trial Notification of JR-171 as a Global Clinical Trial

  5. Slide 5: Contents

    Contents Research & Development News (Jun.-Oct.) JR-141 Development Status Other Pipeline Products Copyright © 2020 JCR Pharmaceuticals Co., Ltd. All rights reserved. R&D 4

  6. Slide 6: Lysosomal Storage Disorders (LSDs)

    Lysosomal Storage disorders（LSDs） LSD is a group of rare inherited disorders in which one of enzymes in the lysosomes is congenitally missing or functionally deficient, resulting in the accumulation of metabolic waste which fails to dissolve. Symptoms vary depending on the affected enzymes and the a

  7. Slide 7: Progress on JR-141

    Progress on JR-141 Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Indication : MPS type II (Hunter Syndrome） Patient population*¹ : 250 (Japan), 7,800 (WW) Market size*² : 7.6 billion JPY est. (2019 Japan), 87 billion JPY est. (2019 WW) Di

  8. Slide 8: Unmet medical needs of Hunter Syndrome

    Unmet medical needs of Hunter Syndrome Intellectual disability: Yes Hunter Life expectancy: Approximately 10 years w/o treatment Syndrome*¹*² Attenuated Severe (2/3) Intellectual disability : None or moderate Life expectancy : Approximately 20 years w/o treatment  CNS symptoms disrupt QoL of patien

  9. Slide 9: JR-141 Summary and Estimated delivery route of J-Brain Cargo

    JR-141 Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan JR-141 Summary and Estimated delivery route of J-Brain Cargo® ＜JR-141＞ • Recombinant fusion protein of humanized antibody (J-Brain Cargo®) specifically binding J-Brain Cargo® (Anti huma

  10. Slide 10: History of JR-141 development

    History of JR-141 development 2005 • Initiation of BBB-penetrating technology research (2007) （ERT for Hunter syndrome launched in Japan） 2014 • Initiation of JR-141 development • Initiation of non-clinical testing • Confirmation of BBB penetration into CNS （Sonoda H, et al. Mol Ther. 2018; 26(5):13

  11. Slide 11: JR-141 non-clinical test: BBB penetration

    JR-141 non-clinical test Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan  Incorporation of JR-141 to neuronal tissues in of the brain following penetration via BBB intravenous administration into hTfR-KI Mice A Distribution to the brain B

  12. Slide 12: JR-141 non-clinical test: Effect on HS accumulation

    JR-141 non-clinical test Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Effect on HS accumulation in MPS II mice  JR-141 decreases HS in brain and Cerebral spinal Fluid (CSF)  A correlation between HS in brain tissues and CSF indicates u

  13. Slide 13: JR-141 Clinical trial in Japan: Brief Summary

    JR-141 Clinical trial in Japan Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Phase 3 trial (JR-141-301)：Brief Summary JR-141-301 JR-141-302 (extension) Idursulfase or Observation JR-141 2.0 mg/kg/w 2.0 mg/kg/w Naive 4 weeks 52 weeks Prima

  14. Slide 14: Clinical trial in Japan: Results, CSF HS at baseline

    JR-141 Clinical trial in Japan Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Phase 3 trial (JR-141-301)：Results Individual CSF HS Levels in Trial JR-141-301 at Baseline Findings  The CSF HS concentration is a biomarker that correlates we

  15. Slide 15: Clinical trial in Japan: Time course of HS reduction in CSF

    JR-141 Clinical trial in Japan Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Phase 3 trial (JR-141-301)：Results Time course of HS reduction in CSF Severe Attenuated Findings  CSF levels decreased in almost all patients to a level compara

  16. Slide 16: Clinical trial in Japan: Age Equivalent Score changes

    JR-141 Clinical trial in Japan Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Phase 3 trial (JR-141-301)：Results Mean Changes in Age Equivalent Score (AES) observed in various Disease Severity Groups Disease phenotype No Subjects Slope Att

  17. Slide 17: Clinical trial in Japan: HS and DS in blood

    JR-141 Clinical trial in Japan Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Phase 3 trial (JR-141-301)：Results HS and DS in blood Serum HS Serum DS Switch Naïve Findings:  Serum HS and DS remained stable in patients switched from IDS to

  18. Slide 18: JR-141 Clinical trial in Brazil: Brief Summary

    JR-141 Clinical trial in Brazil Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Phase 2 trial (JR-141-BR21)：Brief Summary JR-141-BR21 JR-141-BR22 (extension) 4.0 mg/kg/w 4.0 mg/kg/w Idursulfase or 2.0 mg/kg/w 2.0 mg/kg/w 2.0 mg/kg/w Naive 1

  19. Slide 19: Clinical trial in Brazil: HS in CSF

    JR-141 Clinical trial in Brazil Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Phase 2 trial (JR-141-BR21) :Results HS in CSF Findings  Decrease of HS concentration in CSF in all subjects of the 2.0 mg and 4.0 mg treatment group Copyright

  20. Slide 20: Clinical trial in Brazil: DS in serum

    JR-141 Clinical trial in Brazil Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Phase 2 trial (JR-141-BR21) :Results DS in serum <Switch> <Naïve> Findings:  Serum DS remained stable in patients switched from IDS to JR-141  Serum DS rapidl

  21. Slide 21: Investigators' reports on patients

    JR-141 Clinical trial Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Investigators’ reports on patients Phase 3 trial (JR-141-301) 【Speech】 Utters meaningful words, More verbally responsive to greetings, Resumes singing, Tries to speak in

  22. Slide 22: JR-141 has the potential to become a Breakthrough and first choice drug for MPS

    JR-141 Profile • No severe adverse event related to JR-141 reported • Efficacy on CNS signs and symptoms was demonstrated • Easy for initiation and continuation of treatment of MPS II patients: Intravenous infusion interval same as existing ERT. • Consistent findings of reduction in HS concentration

  23. Slide 23: Progress of JR-141: Study Design

    Progress of JR-141 Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan JR-141 Study Design Japan Brazil • Phase 1/2 study • Phase 2 study • Phase 3 study Application for Application for marketing approval Global marketing approval （Sep. 29, 202

  24. Slide 24: JR-141 Global development: Brief Summary

    JR-141 Global development Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Global Phase 3 trial (JR-141-GS)：Brief Summary Countries : USA, Brazil, EU (Germany, France, UK) Objective : To show efficacy on CNS and systemic symptoms. Design : •

  25. Slide 25: Global Phase 3 trial (JR-141-GS) design

    JR-141 Global development Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan Global Phase 3 trial (JR-141-GS)：Brief Summary  Cohort A： (neuronopathic patients) JR-141 2.0 mg/kg/w Standard of Care Possibility of Filing with interim switchable

  26. Slide 26: Progress of JR-141

    Progress of JR-141 Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Status : Filed for MAA in Japan • Oct.2018: • Feb.2019: Orphan Drug Designation Orphan Drug Designation • Mar. 2018: Designated under “SAKIGAKE Designation System” • All patients enrolled in Phase 3 trial

  27. Slide 27: Contents

    Contents Research & Development News (Jun.-Oct.) JR-141 Development Status Other Pipeline Products Copyright © 2020 JCR Pharmaceuticals Co., Ltd. All rights reserved. R&D 26

  28. Slide 28: Progress of Other Compounds: JR-171

    Progress of Other Compounds JR-171 BBB-penetrating α-L-iduronidase (rDNA origin) Indication : MPS type I (Hurler syndrome, Hurler-Scheie syndrome, Scheie syndrome) Patient population*¹ : 60 (Japan), 3,600 (WW) est. Market size*² : 1.6 billion JPY est.（2019 Japan）, 28 billion JPY est.（2019 WW） Diseas

  29. Slide 29: JCR's pipeline for Lysosome diseases

    Other pipeline (Lysosome diseases) JCR’s pipeline for Lysosome diseases Indications with existing somatic ERT (WW) Indications with no established standard of care (WW) MPS type II Red frames: Clinical stucies to start Filed JR-141 within 3years （Hunter) MPS I Clinical JR-171 (Hurler etc.) MPS IIIA

  30. Slide 30: Expected timeline (Lysosome diseases)

    Other pipeline (Lysosome diseases) Expected timeline (Lysosome diseases) 2020 2021 2022 2023 JR-141 Japan：Filed Global： Pabinafusp alfa Initiation of （MPS II） Brazil：To be filed Phase 3 trial JR-171 Global：Phase 1/2 trial Initiation of Phase 3 （MPS I） (ongoing) trial JR-441 Non-clinical (ongoing) In

  31. Slide 31: Expected timeline (GH and regenerative medicine area)

    Other pipeline (GH and regenerative medicine area) Expected timeline (GH area) 2020年 2021年 2022年 2023年 JR-142 Phase 1 Initiation of Initiation of （Pediatric short stature, (Complete) Phase 2 trial Phase 3 trial GHD） JR-401X Phase 3 （Pediatric short stature, File (ongoing) SHOX abnormalities) Note: I

  32. Slide 32: Global specialty pharma in the rare disease arena

    With all the strengths of "Team JCR“, we propel to the forefront as a Global specialty pharma in the rare disease arena Leveraging the three platforms, JCR is committed to its objective of “Realizing medical care for those living with rare diseases” Recombinant Cell Therapy Gene Protein Regenerative

  33. Slide 33: Appendix

    Appendix Copyright © 2020 JCR Pharmaceuticals Co., Ltd. All rights reserved. R&D 32

  34. Slide 34: Progress of Other Compounds: JR-441, JR-162

    Progress of Other Compounds JR-441 BBB-penetrating heparan N-sulfatase (rDNA origin) Indication : MPS type III A (Sanfilippo A syndrome) Patient population*¹ : 60 (Japan）, 6,900 (WW) est. Market size*² : No existing drug Disease overview : An autosomal recessive disease caused by a deficiency of the

  35. Slide 35: Progress of Other Compounds: JR-443, JR-446

    Progress of Other Compounds JR-443 BBB-penetrating β-glucuronidase (rDNA origin) Indication : MPS type VII（Sly syndrome） Patient population*¹ : several (Japan) , 200 (WW) est. Market size*² : 1.4 billion JPY est.（2019 WW） Disease overview : An autosomal recessive disease caused by deficiency of an e

  36. Slide 36: Progress of Other Compounds: JR-142, JR-401X, JR-031HIE, JTR-161

    Progress 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

  37. Slide 37: 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

  38. Slide 38: 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

JCR Pharmaceuticals — H1 FY2020 Earnings Presentation on 6ix