Jcr Pharmaceuticals Co., Ltd. · TSE:4552
Jcr Pharmaceuticals — H1 FY2019 Earnings Presentation
30 slides from Jcr Pharmaceuticals's earnings presentation, Nov 6, 2019
Slide 1: FY2019 First-Half Results Briefing Session - Research and Development Highlights
FY2019 First-Half Results Briefing Session - Research and Development Highlights - November 6, 2019 JCR Pharmaceuticals Co., Ltd. 【Securities code】 4552, 1st Sec. TSE Copyright © 2019 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 2: Forward-Looking Statement
FORWARD-LOOKING STATEMENT This presentation contains, and answers given to questions that may be asked today may constitute, 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,
Slide 3: Forward-Looking Statement
FORWARD-LOOKING STATEMENT The clinical development data published in this document is the result at the time of the interim analysis. It does not guarantee future results, nor does it guarantee the efficacy or effects of products under development. This document is not intended to guarantee and adve
Slide 4: R&D News Release Highlights
R&D News Release Highlights （from May to October, 2019） R&D for Lysosomal Storage Disorders（LSDs） ① Report on Clinical Study of JR-141 ② Progress of Other LSDs Pipeline Progress of Other R&D Pipeline 3 Copyright © 2019 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 5: Development Pipeline
■ Lysosomal Storage Disorders (LSDs) ■ Other Recombinant Protein Therapeutics Development Pipeline ■ Regenerative Medical Product As of Nov. 6, 2019 Code Indication Preclinical Clinical trials Filed Approved Remarks Phase 3 • ERT JR-141 Hunter syndrome • J-Brain Cargo® Phase 2 • ERT JR-162 Pompe dis
Slide 6: R&D News Release Highlights
R&D News Release Highlights （from May to October, 2019） R&D for Lysosomal Storage Disorders（LSDs） ① Report on Clinical Study of JR-141 ② Progress of Other LSDs Pipeline Progress of Other R&D Pipeline 5 Copyright © 2019 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 7: R&D News Release Highlights: JR-142 Phase 1 initiation
R&D News Release Highlights （from May to October, 2019） May 21 JR-142：Initiation of Phase 1 study JR-142 Long-acting growth hormone (rDNA origin) Indication Pediatric growth hormone deficiency JCR’s proprietary half-life extension technology based on a novel modified albumin allows various biotherap
Slide 8: R&D News Release Highlights: JR-031HIE Phase 1/2 initiation
R&D News Release Highlights （from May to October, 2019） Jun. 10 JR-031HIE：Initiation of Phase 1/2 study Human mesenchymal stem cells JR-031HIE （ Expanded indication of TEMCELL®HS Inj. ） Indication Hypoxic ischemic encephalopathy in neonates ■ Cause：an insufficient supply of oxygen to the brain of a
Slide 9: JR-031EB: Withdrawal of Application for Additional Marketing Approval
R&D News Release Highlights （from May to October, 2019） Sep. 19 JR-031EB：Withdrawal of Application for Additional Marketing Approval Human mesenchymal stem cells JR-031EB （ Expanded indication of TEMCELL®HS Inj. ） Indication Epidermolysis bullosa(EB) ■ Cause： Hereditary disorder of abnormal gene exp
Slide 10: JR-131: Marketing Approval
R&D News Release Highlights （from May to October, 2019） Sep. 20 JR-131：Marketing Approval Darbepoetin Alfa (recombinant) [Darbepoetin Alfa Biosimilar 1] JR-131 Darbepoetin Alfa BS Injection [JCR] Indication Renal anemia ■ Disease overview：a subtype of anemia caused by insufficient production of eryt
Slide 11: R&D for Lysosomal Storage Disorders
R&D News Release Highlights （from May to October, 2019） R&D for Lysosomal Storage Disorders（LSDs） ① Report on Clinical Study of JR-141 ② Progress of Other LSDs Pipeline Progress of Other R&D Pipeline 10 Copyright © 2019 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 12: R&D for Lysosomal Storage Disorders
R&D for 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 its function is deficient, resulting in the accumulation of metabolic wastes which fail to dissolve. Its symptom
Slide 13: R&D for Lysosomal Storage Disorders: JR-141
R&D for Lysosomal Storage disorders（LSDs） hereditary metabolic disorders Sphingolipidosis Gaucher disease Fabry disease LSDs … Mucopolysaccharidosis MPS I (Hurler syndrome) Pompe disease MPS II (Hunter syndrome) Alpha-mannosidosis MPS III (Sanfillipo A syndrome) … … JR-141 BBB-penetrating iduronate-
Slide 14: Development strategy
Report on Clinical Study of JR-141 -Development strategy- JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) Indication MPS type II（Hunter syndrome） JR-141 Study Design Japan Brazil Phase 1/2 study Phase 2 study Phase 3 study Global Study Application for Application for marketing approval Ph
Slide 15: Summary of Ph1/2 study
Report on Clinical Study of JR-141 -Summary of Ph1/2 study- JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) Indication MPS type II（Hunter syndrome） Phase 1/2 study（JR-141-101): Study design 4 weeks 4 weeks Patients under Part 1 Part 2 stable idursulfase (1.0 mg/kg/wk, 2.0mg/kg/wk) • Adver
Slide 16: Summary of Ph1/2 study
Report on Clinical Study of JR-141 -Summary of Ph1/2 study- JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) Indication MPS type II（Hunter syndrome） Phase 1/2 study（JR-141-101): Part 2 Results Decrease of HS in CSF indicates BBB-penetration of JR-141 （1.0㎎/㎏/w: 25.1±12.9％ 2.0㎎/㎏/w: 31.5±3.
Slide 17: Summary of Ph3 in Japan
Report on Clinical Study of JR-141 -Summary of Ph3 in Japan- JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) Indication MPS type II（Hunter syndrome） Phase 3 study（JR-141-301): Study design 4 weeks 52 weeks Naive or under stable idursulfase Observation JR-141 (2.0 mg/kg/wk) Primary Endpoin
Slide 18: Summary of Ph3 in Japan
Report on Clinical Study of JR-141 -Summary of Ph3 in Japan- Phase 3 study（JR-141-301): CSF HS concentrations (ng/mL) CSF HS 16000 14000 12000 10000 8000 6000 4000 2000 0 -4 26 Observation JR-141 （ Naive or idursulfase therapy ） HS concentrations in CSF decreased in all patients after infusions for
Slide 19: Summary of Ph3 in Japan
Report on Clinical Study of JR-141 -Summary of Ph3 in Japan- JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) Indication MPS type II（Hunter syndrome） Phase 3 study (JR-141-301): Interim evaluation at the completion of 26-week infusion Enrollment • 28 patients enrolled from 19 facilities Ef
Slide 20: Total scheme
Report on Clinical Study of JR-141 -Total scheme - JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) Indication MPS type II（Hunter syndrome） JCR USA, Inc. • Oct.2018: Designated under • Feb.2019: Designated under Orphan Drug Designation Orphan Drug Designation • Mar. 2018: Designated under
Slide 21: Progress of Other LSDs Pipeline
R&D News Release Highlights （from May to October, 2019） R&D for Lysosomal Storage Disorders（LSDs） ① Report on Clinical Study of JR-141 ② Progress of Other LSDs Pipeline Progress of Other R&D Pipeline 20 Copyright © 2019 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 22: Progress of Other LSDs Pipeline: JR-171
Progress of Other LSDs Pipeline JR-171 BBB-penetrating α-L-iduronidase (rDNA origin) Indication MPS type I (Hurler syndrome, Hurler-Scheie syndrome, Scheie syndrome) ■ Disease overview: A deficiency of α-L-iduronidase, causes an accumulation of Mucopolysaccharide, giving rise to multiple systemic an
Slide 23: Progress of Other LSDs Pipeline: JR-441 and JR-162
Progress of Other LSDs Pipeline JR-441 BBB-penetrating heparan N-sulfatase (rDNA origin) Indication MPS type III A (Sanfilippo syndrome Type A) ■ Disease overview: A deficiency of α-L-iduronidase, causes an accumulation of Mucopolysaccharide, leading to CNS symptoms mailny. *Total of Type A&B ■ Pati
Slide 24: Developing therapeutics for 15 types of LSDs in JCR
α-Mannosidosis Developing therapeutics for GM1 15 types of LSDs in JCR gangliosidosis Niemann-Pick disease Fucosidosis Gaucher disease Batten disease MPS IIIA JR-441 late infantile (Sanfilippo A) Batten disease MPS IIIB MPS I JR-171 infantile type (Sanfilippo B) (Hurler etc.) Metachromatic MPS VII P
Slide 25: Progress of Other R&D Pipeline
R&D News Release Highlights （from May to October, 2019） R&D for Lysosomal Storage Disorders（LSDs） ① Report on Clinical Study of JR-141 ② Progress of Other LSDs Pipeline Progress of Other R&D Pipeline 24 Copyright © 2019 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 26: Progress of Other R&D Pipeline: JTR-161/JR-161
Progress of Other R&D Pipeline JTR-161/JR-161 Human dental pulpstem cells (DPCs) Indication Acute cerebral infarction DPCs ■ Cause： hypertension, diabetes mellitus, smoking, obesity, and dyslipidemia ■ Prevalence* （Japan）: 300,000 est. per year. *Internal analysis ■ Treatment ： “thrombolytic therapy
Slide 27: Progress of Other R&D Pipeline: JR-401X
Progress of Other R&D Pipeline Somatropin (rDNA origin) JR-401X （Expanded Indication of GROWJECT® ） Indication Short stature homeobox-containing gene (SHOX) deficiency ■ Disease overview ：a congenital disorder caused by deletions or mutations of a SHOX gene (short stature homeobox containing gene) l
Slide 28: Development Pipeline
■ Lysosomal Storage Disorders (LSDs) ■ Other Recombinant Protein Therapeutics Development Pipeline ■ Regenerative Medical Product As of Nov. 6, 2019 Code Indication Preclinical Clinical trials Filed Approved Remarks Phase 3 • ERT JR-141 Hunter syndrome • J-Brain Cargo® Phase 2 • ERT JR-162 Pompe dis
Slide 29: JCR aims to develop first-in-class drugs from Japan to the world
Taking one step ahead, JCR aims to develop First-in-class drugs from Japan to the world Recombinant for achieving sustainable protein value creation therapeutics Rare Diseases Intractable Diseases Cell Therapy Gene Regenerative therapies Medicine 28 Copyright © 2019 JCR Pharmaceuticals Co., Ltd. All
Slide 30: Copyright © 2019 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Copyright © 2019 JCR Pharmaceuticals Co., Ltd. All rights reserved.
JCR Pharmaceuticals — H1 FY2019 Earnings Presentation on 6ix