Jcr Pharmaceuticals Co., Ltd. · TSE:4552
Jcr Pharmaceuticals — FY2019 Earnings Presentation
28 slides from Jcr Pharmaceuticals's earnings presentation, May 18, 2020
Slide 1: FY2019 Results Briefing Session - Research and Development Highlights
FY2019 Results Briefing Session - Research and Development Highlights - May 18, 2020 JCR Pharmaceuticals Co., Ltd. 【Securities code】 4552, 1st Sec. TSE Copyright © 2020 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 2: 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
Slide 3: Forward-Looking Statement
FORWARD-LOOKING STATEMENT The clinical development data mentioned in this document 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 advertise the efficacy of the product under development. T
Slide 4: Development Pipeline
■ Lysosomal Storage Disorders (LSDs) ■ Other Recombinant Protein Therapeutics Development Pipeline ■ Regenerative Medical Product As of May 18, 2020 Code Indication Preclinical Clinical trials Filed Approved Remarks MPS type II Phase 3 • ERT JR-141 • J-Brain Cargo® (Hunter Syndrome) Phase 2 • ERT JR
Slide 5: Development Progress
Development Progress ① Progress of JR-141 ② Progress of Other Compounds （JR-171, 441, 142, 401X, 031HIE, JTR-161/JR-161） Research Progress（JR-443, 446, 441, 162） 4 Copyright © 2020 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 6: Development Progress
Development Progress ① Progress of JR-141 ② Progress of Other Compounds （JR-171, 441, 142, 401X, 031HIE, JTR-161/JR-161） Research Progress（JR-443, 446, 441, 162） 5 Copyright © 2020 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 7: 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 wastes which fail to dissolve. Their symptoms vary depending on the affected enzymes and
Slide 8: Progress of JR-141
Progress of JR-141 JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) BBB：Blood Brain Barrier MPS I (Hurler syndrome etc.) MPS II (Hunter syndrome) MPS type II MPS Sanfillipo A syndrome Indication MPS III （Hunter syndrome） Sanfillipo B syndrome … MPS VII（Sly syndrome） … Patient population*¹
Slide 9: JR-141 Study Design
Progress of JR-141 JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) JR-141 Study Design Japan Brazil • Phase 1/2 study • Phase 2 study • Phase 3 study Global Application for Application for • Phase 3 study marketing approval marketing approval Application for marketing approval 8 Copyright
Slide 10: JR-141 Phase 3 Study Design
Progress of JR-141 JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) Phase 3 study（JR-141-301): Study design 4 weeks 52 weeks Naive or under stable Observation JR-141 (2.0 mg/kg/wk) idursulfase Primary Endpoint Change of Heparan Sulfate (HS) in Cerebrospinal Fluid (CSF) • Neurocognitive tes
Slide 11: JR-141 Phase 3 Results (52 weeks)
Progress of JR-141 JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) Phase 3 study（JR-141-301): Results（52 weeks） ＜Concentrations of HS and Dermatan Sulfate (DS) in CNS＞ HS and DS concentrations in CSF decreased in all subjects HS concentrations in CSF decreased significantly（-61.3％±12.1％,
Slide 12: JR-141 Phase 3 Results: Developmental Assessment
Progress of JR-141 JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) Phase 3 study（JR-141-301): Results（52 weeks） ＜New Kyoto Scale Developmental Assessment of Age Equivalence (AE)：after 52 weeks＞ Classification of Disease Number of Slope phenotype subjects Initial phase ： Attenuated 8 0.954
Slide 13: JR-141 Phase 3 Results: Behavioral Improvements
Progress of JR-141 JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) Phase 3 study（JR-141-301): Results（52 weeks） ＜Behavioral improvements in severe subjects（52 weeks）*＞ • Gradual increase in vocabulary • Willingness to communicate • Understood what the parents said • Speech of short senten
Slide 14: JR-141 Phase 3 Results Summary
Progress of JR-141 JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) Phase 3 study（JR-141-301): Results（52 weeks） All subjects completed the final observation ⇒ participated in the extension study Efficacy for CNS symptoms HS and DS concentrations in CSF decreased in all subjects ⇒ Pr
Slide 15: JR-141 Regulatory Status and Plans
Progress of JR-141 JR-141 BBB-penetrating iduronate-2-sulfatase (rDNA origin) • Oct.2018: • Feb.2019: Orphan Drug Designation Orphan Drug Designation • Mar. 2018: Designated under “SAKIGAKE Designation System” • Administration in all subjects completed in Phase 3 • The extension study ongoing Applic
Slide 16: Progress of Other Compounds
Development Progress ① Progress of JR-141 ② Progress of Other Compounds （JR-171, 441, 142, 401X, 031HIE, JTR-161/JR-161） Research Progress（JR-443, 446, 441, 162） 15 Copyright © 2020 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 17: JR-171 Phase 1/2 Study Planned
Progress of Other Compounds JR-171 BBB-penetrating α-L-iduronidase (rDNA origin) MPS type I Indication (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） An autosomal
Slide 18: JR-441 Phase 1/2 Study Planned
Progress of Other Compounds JR-441 BBB-penetrating heparan N-sulfatase (rDNA origin) Indication MPS type III A (Sanfilippo A syndrome) Patient population*¹ 60 (Japan）, 6,900 (WW) est. Market size*² No existing drug An autosomal recessive disease caused by a deficiency of the enzyme heparan- N-sulfat
Slide 19: JR-142 and JR-401X Progress
Progress of Other Compounds JR-142 Long-acting growth hormone (rDNA origin) Indication Pediatric growth hormone deficiency JCR’s proprietary half-life extension technology, based on Note a novel modified albumin, allows significant increase in the half-life of various biotherapeutics （Patent filed）
Slide 20: JR-031HIE and JTR-161/JR-161 Progress
Progress of Other Compounds JR-031HIE Human mesenchymal stem cells（Expanded indication of TEMCELL®HS Inj.） Indication Neonatal Hypoxic Ischemic Encephalopathy Prevalence* 2.5 of 1,000 live births *Internal analysis （Target: 150-200 patients per year with moderate-severe disease indicated for (WW) th
Slide 21: Research Progress
Development Progress ① Progress of JR-141 ② Progress of Other Compounds （JR-171, 441, 142, 401X, 031HIE, JTR-161/JR-161） Research Progress（JR-443, 446, 441, 162） 20 Copyright © 2020 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 22: JR-443 Clinical Study to Start Within 3 Years
Research Progress JR-443 BBB-penetrating β-glucuronidase (rDNA origin) MPS I (Hurler syndrome etc.) MPS II (Hunter syndrome) MPS type VII MPS MPS III Sanfillipo A syndrome Indication （Sly syndrome） … Sanfillipo B syndrome MPS VII（Sly syndrome） … Patient population*¹ several (Japan) , 200 (WW) est. M
Slide 23: JR-446 Clinical Study to Start Within 3 Years
Research Progress JR-446 BBB-penetrating α-N-acetylglucosaminidase (rDNA origin) MPS I (Hurler syndrome etc.) MPS II (Hunter syndrome) MPS type III B MPS MPS III Sanfillipo A syndrome Indication （Sanfillipo B syndrome） … Sanfillipo B syndrome MPS VII（Sly syndrome） … Patient population*¹ 60 (Japan）,
Slide 24: JR-446 Efficacy in MPSIIIB Model Mice
Research Progress JR-446 BBB-penetrating α-N-acetylglucosaminidase (rDNA origin) The results of drug efficacy study in MPSIIIB model mice ＜HS concentrations in Brain＞ μg/mg tissue weight WT Disease Dose1 Dose2 Enzyme mice control only JR-446 MPSIIIB model mice 23 Copyright © 2020 JCR Pharmaceutica
Slide 25: JR-162 Phase 1/2 Study Planned in 2023
Research Progress JR-162 J-Brain Cargo®-applied acid α-glucosidase (rDNA origin) Indication Pompe disease Patient population*¹ 80 (Japan), 10,600 (WW) est. Market size*² 3 billion JPY est. (2019 Japan), 110 billion JPY est. (2019 WW） An autosomal recessive disease caused by a deficiency of the enzym
Slide 26: Developmental Stages of the LSD Therapeutics by JCR
Developmental stages of the LSD therapeutics by JCR Krabbe disease α-Mannosidosis Compounds in red frames: Clinical studies to start within 3 years Niemann-Pick disease MPS IIIB Gaucher disease JR-446 (Sanfilippo B) Batten disease MPS VII MPS IIIA JR-441 late infantile JR-443 (Sly) (Sanfilippo A) Ba
Slide 27: R&D Target Timeline
R&D Target Timeline 2020 2021 2022 2023 Application for marketing approval JR-141 Application for marketing approval Global study initiated JR-171 Phase 1/2 initiated JR-142 Phase 1 Phase 2 initiated JTR-161 Phase 1/2 Phase 1/2 completed /JR-161 JR-441 Preclinical Phase 1/2 initiated Application for
Slide 28: Global specialty pharma in the rare disease arena
With all the strengths of "Team JCR“, we achieve Global specialty pharma in the rare disease arena Utilizing three platforms, JCR promotes its objective of “Realizing medical care for those living with rare diseases” Recombinant Cell Therapy Gene Protein Regenerative Therapies Therapeutics Medicine