Jcr Pharmaceuticals Co., Ltd. · TSE:4552
Jcr Pharmaceuticals — H1 FY2018 Earnings Presentation
19 slides from Jcr Pharmaceuticals's earnings presentation, Nov 2, 2018
Slide 1: FY2018 First-Half Results Briefing Session - Research and Development
FY2018 First-Half Results Briefing Session - Research and Development - November 2, 2018 JCR Pharmaceuticals Co., Ltd. Copyright © 2018 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 2: Development Pipeline in Japan
■ Lysosomal Storage Disorder (LSD) ■ Other Biopharmaceuticals Development Pipeline in Japan ■ Regenerative Medical Product As of Nov 2, 2018 Code Indication Pre-clinical PhⅠ/Ⅱ PhⅢ Filed Approved Remarks • Enzyme replacement Agalsidase Beta BS Fabry disease therapy (ERT) I.V. Infusion [JCR] • Biosimi
Slide 3: Research & Development News since May 22, 2018
Research & Development News since May 22, 2018 May 28 JCR Receives Approval in Brazil to Initiate Phase 2 Clinical Trial of JR-141 for Hunter Syndrome June 1 JCR Announces Completion of PMDA Review of Phase 3 Clinical Trial of JR-141 for Hunter Syndrome June 29 JCR Initiates Phase 2 Clinical Trial o
Slide 4: Key products and development status
Product Status Indication LSD Agalsidase Beta BS I.V. Infusion [JCR] Received Recombinant Agalsidase Beta marketing approval Fabry disease Biosimilar (rDNA origin) in Japan JR-441 Sanfilippo LSD BBB-Penetrating heparan N-sulfatase Preclinical syndrome type A (rDNA origin) JR-141 Japan: Phase III LSD
Slide 5: Agalsidase Beta BS I.V. Infusion: To be launched in Nov. 2018
Therapeutic enzyme for Fabry disease Biosimilar to Fabrazyme (α-galactosidase A) JCRʼs first enzyme replacement therapy for LSD Agalsidase Beta BS I.V. Infusion [JCR] 2015 Initiated clinical trial 2017 Sep. Submitted application for marketing approval in Japan 2018 Sep. Received marketing approval f
Slide 6: Agalsidase Beta BS I.V. Infusion [JCR]
Therapeutic enzyme for Fabry disease Biosimilar to Fabrazyme (α-galactosidase A) JCRʼs first enzyme replacement therapy for LSD Agalsidase Beta BS I.V. Infusion [JCR] ¾ Fabry disease MHLW designated intractable disease ■ Disease condition: - Skin : Angiokeratomas, lymphedema of the lower limbs - Cir
Slide 7: JR-441 BBB-penetrating heparan N-sulfatase
JR-441 BBB-penetrating heparan N-sulfatase NEW MHLW designated ¾ Sanfilippo syndrome Type A（ MPS IIIA ） intractable disease ■ Cause︓ an inborn deficiency or defect in heparan N-sulfatase within lysosomes in cells throughout the body ■ Disease condition︓ CNS disorders, sleep disorders, hepatosplenome
Slide 8: JR-141 BBB-penetrating iduronate-2-sulfatase
JR-141 BBB-penetrating iduronate-2-sulfatase ¾ Hunter syndrome (MPS type II) MHLW designated intractable disease ■Disease condition - Bone : characteristic face, bone deformity, arthrogryposis - Heart : cardiac valvular disease - Soft tissue : thick skin, hairiness, macroglossia - Liver : hepatomega
Slide 9: Proactive development of LSD pipeline
Proactive development of LSD pipeline -15 early to late stage J-Brain Cargo® programs- Niemann-Pick disease Fucosidosis Gaucher disease Batten disease late infantile Sanfilippo A GM1 Batten disease JR-441 syndrome gangliosidosis infantile type (MPS IIIA) Sanfilippo B Hurler Krabbe disease syndrome J
Slide 10: Potential of J-Brain Cargo
Potential of J-Brain Cargo® J-Brain Cargo® Therapeutic enzyme products for CNS Disorders treating LSDs （In-house development） （Out-licensing） J-Brain Cargo® potential applications to various CNS disorders 9 Copyright © 2018 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 11: Key products and development status
Product Status Indication LSD Agalsidase Beta BS I.V. Infusion [JCR] Received Recombinant Agalsidase Beta marketing approval Fabry disease Biosimilar (rDNA origin) in Japan JR-441 Sanfilippo LSD BBB-Penetrating heparan N-sulfatase Preclinical syndrome type A (rDNA origin) JR-141 Japan: Phase III LSD
Slide 12: JR-031EB: Expanded indication of TEMCELL HS Inj.
Expanded indication of JR-031EB TEMCELL®HS Inj. Oct. 2018︓ Designed the orphan regenerative medical product for EB in Japan ¾ Epidermolysis bullosa︓EB MHLW designated intractable disease ■ Cause: Hereditary disorder of abnormal gene expressed in the cutaneous basement membrane zone ■ Disease conditi
Slide 13: JTR-161/JR-161 Phase I/II started in Oct. 2018
Human dental pulp JTR-161/JR-161 stem cells (DPCs) a new product in cell therapy and regenerative medicine succeeded TEMCELL ® HS Inj. Co-development and license agreement with Teijin Limited in July 2017 Indication︓Acute cerebral infarction DPCs ¾ Acute cerebral infarction ■ Cause : Major risk fact
Slide 14: Key products and development status
Product Status Indication LSD Agalsidase Beta BS I.V. Infusion [JCR] Received Recombinant Agalsidase Beta marketing approval Fabry disease Biosimilar (rDNA origin) in Japan JR-441 Sanfilippo LSD BBB-Penetrating heparan N-sulfatase Preclinical syndrome type A (rDNA origin) JR-141 Japan: Phase III LSD
Slide 15: JR-131 Darbepoetin (Biosimilar to Nesp)
JR-131 Darbepoetin (Biosimilar to Nesp) ● Co-development agreement with Kissei Pharmaceutical Co., Ltd. in Sep. 2013 Leveraging JCRʼs proprietary Glycoengineering Technology to approach complex sugar chain structure Patent filed ・ Phase III study︓demonstrated equivalence in efficacy and safety compa
Slide 16: Growth Hormone
Growth Hormone JR-401X Somatropin Expanded Indication of GROWJECT® ¾ Indication︓ Short stature homeobox containing gene (SHOX) deficiency Phase Ⅲ started in July 2018 JR-142 Long-acting growth hormone ¾ JCRʼs proprietary half-life extension technology based on a novel modified albumin allows various
Slide 17: Development Pipeline in Japan
■ Lysosomal Storage Disorder (LSD) ■ Other Biopharmaceuticals Development Pipeline in Japan ■ Regenerative Medical Product As of Nov 2, 2018 Code Indication Pre-clinical PhⅠ/Ⅱ PhⅢ Filed Approved Remarks • Enzyme replacement Agalsidase Beta BS Fabry disease therapy (ERT) I.V. Infusion [JCR] • Biosimi
Slide 18: Copyright © 2018 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Copyright © 2018 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 19: 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,
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