Jcr Pharmaceuticals Co., Ltd. · TSE:4552

Jcr Pharmaceuticals — FY2020 Earnings Presentation

33 slides from Jcr Pharmaceuticals's earnings presentation, May 17, 2021

  1. Slide 1: FY2020 Results Briefing Session - Research and Development Highlights

    FY2020 Results Briefing Session - Research and Development Highlights - May 17, 2021 JCR Pharmaceuticals Co., Ltd. Copyright © 2021 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 Approved MPS type II • ERT JR-141 Filed • J-Brain Cargo® (Hunter Syndrome) Phase 3 • ERT MPS type I JR-1

  3. Slide 3: Contents

    Contents Research & Development News (Nov.-Mar.) JR-141 Development Status JR-171 Development Status JR-142 Development Status Other Pipeline Products Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights reserved. R&D 2

  4. Slide 4: Research & Development News (Nov.-Mar.)

    Research & Development News (Nov.-Mar.) 2020 Oct. : First Patient Dosed in Phase 1/2 Global Clinical Trial of JR-171 Dec.：Completion of Phase 2 Clinical Trial Notification of JR-142 Dec.：JCR Files for Marketing Approval of JR-141 in Brazil Dec.：JCR Signs Provisional Production Master Service Agreeme

  5. Slide 5: Contents

    Contents Research & Development News (Nov.-Mar.) JR-141 Development Status JR-171 Development Status JR-142 Development Status Other Pipeline Products Copyright © 2021 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 of JR-141

    Progress of JR-141 IZCARGO®（Product name in Japan） JR-141 Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Study Design Japan Brazil • Phase 1/2 study • Phase 2 study • Phase 3 study Manufacturing and Application for marketing approval Global marketing approval （Mar. 23, 2

  8. Slide 8: JR-141 Study Designs in Japan and Brazil

    Progress of JR-141 IZCARGO®（Product name in Japan） JR-141 Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141-301(phase II/III) and JR-141-302(Extension) Study in Japan JR-141-301 JR-141-302 (Extension) Idursulfase or Naive 2.0 mg/kg/w 2.0 mg/kg/w 52 weeks JR-141-BR21(phase I

  9. Slide 9: JR-141 Phase 3 Results: Heparan Sulfate in CSF

    Progress of JR-141 IZCARGO®（Product name in Japan） JR-141 Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) Phase 3 trial・Extension study(JR-141-301/302) ：Results  Time course of Heparan Sulfate (HS) in CSF（severe） Baseline Week 25 Week 53 Week 105  The concentration of substrat

  10. Slide 10: JR-141 Developmental Trajectories of the Siblings

    Progress of JR-141 IZCARGO®（Product name in Japan） JR-141 Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) Phase 3 trial・Extension study (JR-141-301/302)：Results  Developmental Trajectories of the siblings Sibling 1 48 Sibling 2 Age equivalent score (months) 42 months Trajectori

  11. Slide 11: JR-141 Phase 2 Results (Brazil)

    Progress of JR-141 IZCARGO®（Product name in Japan） JR-141 Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) Phase 2 trial (JR-141-BR21/BR22)：Results  developmental assessment （BSID-III）  Developmental assessment was stabilized or improved, suggesting that JR-141 may improve cent

  12. Slide 12: JR-141 Global Development: Phase 3 Summary

    JR-141 Global development IZCARGO®（Product name in Japan） JR-141 Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) Global Phase 3 trial (JR-141-GS31)：Brief Summary Countries : USA, Brazil, EU (Germany, France, UK) Objective : To show efficacy on CNS and systemic symptoms. Design :

  13. Slide 13: JR-141 Global Phase 3 Design

    JR-141 Global development IZCARGO®（Product name in Japan） JR-141 Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) Global Phase 3 trial (JR-141-GS31)：Brief Summary  Cohort A： (neuronopathic patients) JR-141 2.0 mg/kg/w Standard of Care Possibility of Filing with interim switchabl

  14. Slide 14: JR-141 Regulatory Progress

    Progress of JR-141 IZCARGO®（Product name in Japan） JR-141 Pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) • Oct.2018: • Feb.2019: Orphan Drug Designation Orphan Drug Designation ・ Feb. 2021： • Mar. 2018: Designated under Fast Track Designation “SAKIGAKE Designation System” • Mar

  15. Slide 15: Contents

    Contents Research & Development News (Nov.-Mar.) JR-141 Development Status JR-171 Development Status JR-142 Development Status Other Pipeline Products Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights reserved. R&D 14

  16. Slide 16: Progress of JR-171

    Progress of JR-171 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） Disease overvie

  17. Slide 17: JR-171 Phase 1/2 Global Trial Summary

    Progress of JR-171 JR-171 BBB-penetrating α-L-iduronidase (rDNA origin) Phase 1/2 Global Clinical Trial（JR-171-101) ：Brief Summary Randomized Part 1 Part 2 allocation 4 weeks 12 weeks High dose High dose Extension Low dose Low dose Safety Evaluation Part1 Part2 Primary endpoint Safety Secondary and

  18. Slide 18: JR-171 Phase 1/2 Results Part 1

    Progress of JR-171 JR-171 BBB-penetrating α-L-iduronidase (rDNA origin) Phase 1/2 Global Clinical Trial（JR-171-101) ：Results Part 1(N=4) Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights reserved. R&D 17

  19. Slide 19: Contents

    Contents Research & Development News (Nov.-Mar.) JR-141 Development Status JR-171 Development Status JR-142 Development Status Other Pipeline Products Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights reserved. R&D 18

  20. Slide 20: Progress of JR-142: Phase 1 Summary

    Progress of JR-142 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 filed） Phase

  21. Slide 21: JR-142 Phase 1 Results

    Progress of JR-142 JR-142 Long-acting growth hormone (rDNA origin) Phase 1 Clinical Trial（JR-142-101): Results Change in serum IGF-1 SD score (1st dose) mean + SD placebo Time after administration (hour)  Dose-dependent increases in PK parameters and pharmacodynamic markers were observed.  No safe

  22. Slide 22: JR-142 Phase 2 Summary

    Progress of JR-142 JR-142 Long-acting growth hormone (rDNA origin) Phase 2 Clinical Trial（JR-142-201) ：Brief Summary 26weeks JR-142 0.75mg/kg/w Treatment group (Weekly) 0.5mg/kg/w Extension GROWJECT Treatment group 0.175mg/kg/w (Daily) First patient dosing in Phase 2 clinical trial scheduled for May

  23. Slide 23: Contents

    Contents Research & Development News (Nov.-Mar.) JR-141 Development Status JR-171 Development Status JR-142 Development Status Other Pipeline Products Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights reserved. R&D 22

  24. Slide 24: 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) ：Presentation at 17th Annual Filed JR-141 MPS type II WORLDSymposiumTM2021 Brazil （Hunter) JR-171 MPS I JR-141 MPS type II Clinical

  25. Slide 25: Expected timeline (Lysosome diseases)

    Other pipeline (Lysosome diseases) Expected timeline (Lysosome diseases) 2021 2022 2023 2024 JR-141 Japan：Approval pabinafusp alfa Brazil：To be approved （MPS II） Global：Initiation of Phase 3 trial JR-171 Global：Phase 1/2 trial Initiation of Phase 3 trial （MPS I） (ongoing) JR-441 Non-clinical (ongoin

  26. Slide 26: Other pipeline (GH and regenerative medicine area)

    Other pipeline (GH and regenerative medicine area) Expected timeline (GH area) 2021年 2022年 2023年 2024年 JR-142 （Pediatric short stature, Initiation of Phase 2 trial Initiation of Phase 3 trial GHD） JR-401X Phase 3 （Pediatric short stature, To be filed (ongoing) SHOX abnormalities) Note: Information a

  27. Slide 27: 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

  28. Slide 28: Appendix

    Appendix Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights reserved. R&D 27

  29. Slide 29: 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 : 30 (Japan : Total of Type A&B）, 4,000 (WW) est. Market size*² : No existing drug Disease overview : An autosomal recessive disease caused by

  30. Slide 30: 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

  31. Slide 31: Progress of Other Compounds JR-142, JR-401X

    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

  32. Slide 32: 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

  33. Slide 33: 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

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