Jcr Pharmaceuticals Co., Ltd. · TSE:4552

Jcr Pharmaceuticals — H1 FY2021 Earnings Presentation

28 slides from Jcr Pharmaceuticals's earnings presentation, Nov 2, 2021

  1. Slide 1: FY2021 First-Half Results Briefing Session – R&D Highlights

    FY2021 First-Half Results Briefing Session ーResearch and Development Highlightsー November 2, 2021 JCR Pharmaceuticals Co., Ltd Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights reserved. 【Securities code】4552, 1st Sec. TSE

  2. Slide 2: Contents

    Contents ❚ Research and Development Topics(Period：June to October 2021) ❚ Lysosome treatment pipeline ❚ JR-171 ❚ JR-141 ❚ Others, Expected timeline ❚ Growth hormone, Regenerative medicine area ❚ JR-401X ❚ Others, Expected timeline R&D 1 Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights reser

  3. Slide 3: Contents

    Contents ❚ Research and Development Topics(Period：June to October 2021) ❚ Lysosome treatment pipeline ❚ JR-171 ❚ JR-141 ❚ Others, Expected timeline ❚ Growth hormone, Regenerative medicine area ❚ JR-401X ❚ Others, Expected timeline R&D 2 Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights reser

  4. Slide 4: Research and Development Topics (June to October 2021)

    Research and Development Topics (Period：June to October 2021) 2021 ♦Jul. Presentation at 16th International Symposium on MPS and Related Diseases (MPS2021) Development pipeline for lysosomal storage diseases which apply the J-Brain Cargo® (JR-141, 171, 441, 446) were conducted oral and poster presen

  5. Slide 5: Development Pipeline

    Development Pipeline ■ Lysosomal Storage Diseases (LSDs) ■ Other Recombinant Protein Therapeutics ■ Regenerative Medical Product Code Indication Preclinical Cinical Trials Filed Approved Remarks MPS type II Filed • ERT JR-141 J-Brain Cargo® (Hunter Syndrome) • Phase 3 • ERT MPS type I JR-171 (Hurler

  6. Slide 6: Contents

    Contents ❚ Research and Development Topics(Period：June to October 2021) ❚ Lysosome treatment pipeline ❚ JR-171 ❚ JR-141 ❚ Others, Expected timeline ❚ Growth hormone, Regenerative medicine area ❚ JR-401X ❚ Others, Expected timeline R&D 5 Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights reser

  7. Slide 7: JCR's proprietary BBB-penetrating technology, J-Brain Cargo

    JCR’s proprietary blood-brain-barrier (BBB)-penetrating technology, J-Brain CargoⓇ Penetrating BBB through transferrin receptor to deliver the active ingredient into the brain Molecular + = concept TfR-targeting Effector J-Brain Cargo® mAb (enzyme) Antibody-enzyme fusion protein Transit mechanism Br

  8. Slide 8: Lysosomal Storage diseases (LSDs)

    Lysosomal Storage diseases（LSDs） LSD is a group of rare inherited diseases 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 acc

  9. Slide 9: MPS (Mucopolysaccharidosis)

    MPS (Mucopolysaccharidosis） ribosomes mitochondria rough-surfaced MPS is a group of LSD in which endoplasmic reticulum centrosome mucopolysaccharides such as dermatan sulfate (DS) and heparan sulfate (HS) accumulate. cell nucleus smooth-surfaced endoplasmic reticulum Accumulation of mucopolysacchari

  10. Slide 10: MPS type I (Hurler, Hurler-Scheie, Scheie syndrome) JR-171

    MPS type I（Hurler, Hurler-Scheie, Scheie syndrome） MPS I（ratio）*1 Symptoms*2 Central nervous system symptoms (CNS)*2 Conventional treatments*3 Hurler the most severe form, symptoms emerge significant developmental delay and （≒60％） shortly after birth and progress rapidly cognitive decline Hurler-Sch

  11. Slide 11: Progress of JR-171

    Progress of JR-171 JR-171 BBB-penetrating α-L-iduronidase (rDNA origin) Phase 1/2 Global Clinical Trial（JR-171-101) ：Brief Summary  JR-171 was granted Fast Track Designation from US FDA in Sep. 2021. 4 People 14 or more persons Part 1 Subjects Part 2 Subjects  18 years or older  0 years or older

  12. Slide 12: JR-171: CSF heparan sulfate levels in Part 1 subjects

    Progress of JR-171 JR-171 BBB-penetrating α-L-iduronidase (rDNA origin) Phase 1/2 Global Clinical Trial（JR-171-101) ： CSF*-heparan sulfate (HS) levels in Part 1 subjects *CSF: Cerebrospinal fluid 3000 HS concentration in CSF* (ng/mL) Subjects background（N＝4） 2500 • Scheie syndorome • No intellectual

  13. Slide 13: JR-171: HS/DS levels in Part 1 subjects

    Progress of JR-171 JR-171 BBB-penetrating α-L-iduronidase (rDNA origin) Phase 1/2 Global Clinical Trial（JR-171-101) ：heparan sulfate/dermatan sulfate (HS/DS) levels in Part1 subjects Serum HS concentration (ng/mL) Serum DS concentration (ng/mL) 1000 Subject 1 2000 Subject 1 HS Subject 2 DS Subject 2

  14. Slide 14: Progress of JR-171

    Progress of JR-171 JR-171 BBB-penetrating α-L-iduronidase (rDNA origin) Phase 1/2 Global Clinical Trial（JR-171-101) ：Brief Summary Part1：Summary  CSF HS levels decreased from baseline in all subjects  There were no serious safety concerns. Current Status and Future Planning  JR-171-101 Part 2 wit

  15. Slide 15: Progress of JR-141: Clinical Development Package

    Progress of JR-141 IZCARGO®（Brand name in Japan） JR-141 pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141 Cinical Development Package Japan Global Brazil (US, Brazil, UK, France, Germany, Spain) • Phase 1/2 clinical trial • Phase 2 clinical trial • Phase 3 clinical trial •

  16. Slide 16: Progress of JR-141

    Progress of JR-141 IZCARGO®（Brand name in Japan） JR-141 pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) JR-141-301(phase 2/3) and JR-141-302(Extension) clinical trials 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(ph

  17. Slide 17: Progress of JR-141

    Progress of JR-141 IZCARGO®（Brand name in Japan） JR-141 pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) Phase 3 clinical trials in Japan (JR-141-301/302) Phase 2 clinical trials in Brazil（JR-141-BR21/BR22） Merged age equivalent score in severe patients overlaid with the natural

  18. Slide 18: Progress of JR-141: Age equivalent (months)

    Progress of JR-141 IZCARGO®（Brand name in Japan） JR-141 pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) Phase 2 clinical trials in Brazil（JR-141-BR21/BR22） Kaufman (KABCII)* Assessment in subjects with attenuated symptoms. Normal development 240 Observations and conclusions: • M

  19. Slide 19: JR-141 Clinical Trials: Narrative Reports

    JR-141 Clinical Trials IZCARGO®（Brand name in Japan） JR-141 pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin) Narrative Reports (JR-141-301 and JR-141-BR22) Japan (JR-141-301) Brazil (JR-141-BR21/BR22) Language General changes observed • 12/18 of narrative reports from severe pati

  20. Slide 20: JR-141 Global Trial Phase 3 (JR-141-GS31)

    JR-141 Global Trial Phase 3（JR-141-GS31） IZCARGO®（Brand name in Japan） JR-141 pabinafusp alfa (BBB-penetrating iduronate-2-sulfatase, rDNA origin)  Designation of PRIME from the EMA in Oct. 2021 （Summary） JR-141 2.0 mg/kg/w The application is Standard After 53 weeks, also considered  Cohort A： tre

  21. Slide 21: Lysosomal diseases: Expected timeline

    Lysosomal diseases：Expected timeline Note: Information after 2022 is a plan at this stage and is subject to change 2021 2022 2023 2024 • SAKIGAKE（PMDA） Brazil： Filed • Orphan Drug（PMDA） JR-141 • Orphan Drug（FDA） pabinafusp alfa Global：Initiation of • Fast Track（FDA） （MPS II） Phase 3 trial • Orphan D

  22. Slide 22: JCR's ERT pipeline for Lysosome diseases

    JCR’s 17 ERT pipeline for Lysosome diseases ：Presentation at 18th Annual WORLDSymposiumTM2022 ：Project with progress in development stage since May 2021 Indications with existing somatic ERT (Worldwide) Indications with no established standard of care (Worldwide) JR-141 MPS II Filed Brazil （Hunter)

  23. Slide 23: Contents

    Contents ❚ Research and Development Topics(Period：June to October 2021) ❚ Lysosome treatment pipeline ❚ JR-171 ❚ JR-141 ❚ Others, Expected timeline ❚ Growth hormone, Regenerative medicine area ❚ JR-401X ❚ Others, Expected timeline R&D 22 Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights rese

  24. Slide 24: JR-401X: About SHOX deficiency

    JR-401X： About SHOX deficiency JR-401X Expanded indication of GROWJECT® : SHOX deficiency  SHOX deficiency is a congenital disorder caused by micro deletions, duplications, or mutations that result in loss of function of a growth-gene SHOX (Short stature homeobox containing gene) present on the sex

  25. Slide 25: JR-401X Clinical Trials Phase 3

    JR-401X Clinical Trials Phase 3 JR-401X GROWJECT® additional indication: SHOX deficiency JR-401X-301：Design（JAPIC trial ID：JapicCTI-183946） NT/GH group Target number :16 No treatment 0.35 mg/kg/w 12 months 12 months GH/GH group Target number :16 0.35 mg/kg/w 24 months Efficacy endpoints  Primary en

  26. Slide 26: JR-401X: Results of efficacy data analysis

    JR-401X: Results of efficacy data analysis （At the end of 12 months） Long-acting recombinant growth hormone JR-401X Growject ® additional indication: SHOX deficiency GH/GH group NT/GH group Difference N=10 N=9 N 10 8 mean(SD) 0.932(0.213) 0.127(0.105) 0.796(0.174) Change in height SDS for median 0.9

  27. Slide 27: Other pipeline (Growth Hormone and regenerative medicine area)

    Other pipeline (Growth Hormone and regenerative medicine area) Expected timeline (Growth Hormone area) 2021 2022 2023 2024 JR-142* Phase 2 （Pediatric short stature, GHD） Initiation of Phase 3 trial (Ongoing） *Long-acting growth hormone JR-401X Phase 3 To be filed （SHOX deficiency) (Ongoing) Note: In

  28. Slide 28: Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights reserved.

    Copyright © 2021 JCR Pharmaceuticals Co., Ltd. All rights reserved.

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