Slide 1: FY2018 Results Briefing Session - Research and Development Highlights
FY2018 Results Briefing Session - Research and Development Highlights - May 16, 2019 JCR Pharmaceuticals Co., Ltd. 【Securities code】 4552, 1st Sec. TSE Copyright © 2018 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 2: Development Pipeline
■ Lysosomal Storage Disorder (LSD) ■ Other Recombinant Protein Therapeutics Development Pipeline ■ Regenerative Medical Product As of May 16, 2019 Code Indication Pre-clinical Ph I/II Ph III Filed Approved Remarks • ERT JR-141 Hunter syndrome • J-Brain Cargo® • ERT JR-162 Pompe disease • J-Brain Car
Slide 3: Research & Development News since Nov. 28, 2018
Research & Development News since Nov. 28, 2018 2018 Nov. 28：Launch of Agalsidase Beta BS I.V. Infusion [JCR] for Fabry Disease Dec. 21：A blood-brain-barrier (BBB)-penetrating recombinant iduronate-2-sulfatase (JR-141): Notice on The Publication of The Phase 1/2 Clinical Trial Results for Hunter Syn
Slide 4: Product status and indication: JR-141, JR-171, JR-441
Product Status Indication JR-141 Japan: Phase III LSD ライソゾーム病 BBB-penetrating iduronate-2-sulfatase Brazil: Phase II Hunter syndrome (rDNA origin) JR-171 ライソゾーム病 LSD BBB-penetrating α-L-iduronidase Preclinical Hurler syndrome (rDNA origin) JR-441 Sanfilippo ライソゾーム病 LSD BBB-penetrating heparan N-sulf
Slide 5: JR-141 BBB-penetrating iduronate-2-sulfatase
JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) 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 - Live
Slide 6: JR-141 Application for marketing approval planned in FY2020 in Japan
JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) Hunter syndrome (MPS type II) MHLW designated intractable disease JCR USA, Inc. • Oct.2018: Designated under • Feb.2019: Designated under Orphan Drug Designation Orphan Drug Designation • Mar. 2018: Designated under “SAKIGAKE Designation S
Slide 7: JR-171 Phase I/II trial is planned in FY 2019
JR-171 BBB-penetrating α-L-iduronidase (rDNA origin) Hurler syndrome (MPS type I) MHLW designated intractable disease *Internal analysis ■ Patient population*： 60 (Japan), 3,600 (WW) est. ■ Market size* ： 1.5 billion JPY est. (2018 Japan) 24 billion JPY est. (2018 WW) ■ Disease condition - Bone :
Slide 8: JR-441 Phase I/II trial is planned in FY 2020
JR-441 BBB-penetrating heparan N-sulfatase (rDNA origin) MHLW designated Sanfilippo syndrome Type A（ MPS IIIA ） intractable disease *Total of Type A&B ■ Patient population*： 60 (Japan), 6,900 (WW) est. (Internal analysis) ■ Disease condition： CNS disorders, sleep disorders, hepatosplenomegaly, sei
Slide 9: JR-162 Pompe disease
JR-162 J-Brain Cargo®-applied acid α-glucosidase (rDNA origin) Pompe disease MHLW designated intractable disease ■ Patient population*： 80 (Japan), 10,600 (WW) est. *Internal analysis ■ Market size* ： 3 billion JPY est. (2018 Japan), 99 billion JPY est. (2018 WW) ■ Disease condition - Primarily af
Slide 10: Gene Therapy
Gene Therapy 【Human Cell】 lysosome 【Lysosome】 nucleus Rough endoplasmic reticulum ribosome mitochondrion Golgi apparatus human cell enzyme Membrane protein • Replacement therapy is not appropriate • Enzyme replacement therapy can be applied • The gene needs to be expressed in the cell J-Brain Cargo®
Slide 11: Progress of developmental stage of LSD pipeline
Progress of developmental stage of LSD pipeline -15 early to late stage J-Brain Cargo® programs- * Developmental stage moved forward from Nov. 2018. * α-Mannosidosis GM1 * gangliosidosis Niemann-Pick disease Fucosidosis Gaucher disease Sanfilippo A Batten disease JR-441 syndrome late infantile (MPS
Slide 12: Potential of J-Brain Cargo
Potential of J-Brain Cargo® Collaboration PeptiDream Inc., 2016 Started the joint research collaboration Successful development 2019 of constrained peptides across the BBB potential applications to various CNS disorders 11 Copyright © 2018 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 13: Product status and indication: JR-141, JR-171, JR-441
Product Status Indication JR-141 Japan: Phase III LSD ライソゾーム病 BBB-penetrating iduronate-2-sulfatase Brazil: Phase II Hunter syndrome (rDNA origin) JR-171 ライソゾーム病 LSD BBB-penetrating α-L-iduronidase Preclinical Hurler syndrome (rDNA origin) JR-441 Sanfilippo ライソゾーム病 LSD BBB-penetrating heparan N-sulf
Slide 14: JR-031EB Marketing approval planned in FY 2019
Expanded indication of TEMCELL®HS Inj. JR-031EB Human mesenchymal stem cells MHLW designated Epidermolysis bullosa：EB intractable disease ■ Cause: Hereditary disorder of abnormal gene expressed in the cutaneous basement membrane zone ■ Disease condition: Slight friction may cause the skin to detac
Slide 15: JTR-161/JR-161 Started administration in Phase I/II
JTR-161 Human dental pulpstem cells (DPCs) /JR-161 Acute cerebral infarction DPCs ■ Cause : Major risk factors are generally the same as for atherosclerosis (high blood pressure, diabetes mellitus, tobacco smoking, obesity, and dyslipidemia) ■ Patient population* (Japan): 300,000 est. *Internal an
Slide 16: Product status and indication: JR-141, JR-171, JR-441
Product Status Indication JR-141 Japan: Phase III LSD ライソゾーム病 BBB-penetrating iduronate-2-sulfatase Brazil: Phase II Hunter syndrome (rDNA origin) JR-171 ライソゾーム病 LSD BBB-penetrating α-L-iduronidase Preclinical Hurler syndrome (rDNA origin) JR-441 Sanfilippo ライソゾーム病 LSD BBB-penetrating heparan N-sulf
Slide 17: JR-131 Marketing approval planned in FY 2019
JR-131 Darbepoetin alfa (rDNA origin) Renal anemia ● Co-development agreement with Kissei Pharmaceutical Co., Ltd. in Sep. 2013 Leveraging JCR’s proprietary Glycoengineering Technology to approach complex sugar chain structure Patent filed Phase III study：demonstrated equivalence in efficacy and
Slide 18: JR-142 Phase I planned in May 2019
JR-142 Long-acting growth hormone (rDNA origin) Pediatric growth hormone deficiency JCR’s proprietary half-life extension technology based on a novel modified albumin allows various biotherapeutic products to increase drug half-life significantly Patent filed Prominently extended circulatory hal
Slide 19: Development Pipeline
■ Lysosomal Storage Disorder (LSD) ■ Other Recombinant Protein Therapeutics Development Pipeline ■ Regenerative Medical Product As of May 16, 2019 Code Indication Pre-clinical Ph I/II Ph III Filed Approved Remarks • ERT JR-141 Hunter syndrome • J-Brain Cargo® • ERT JR-162 Pompe disease • J-Brain Car
Slide 20: Bio Drugs, Gene Therapy: 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 Bio Drugs Rare Diseases Intractable Diseases Cell Therapy Gene Regenerative Medicine Therapy 19 Copyright © 2018 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 21: Copyright © 2018 JCR Pharmaceuticals Co., Ltd. All rights reserved.
20 Copyright © 2018 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 22: 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,