Jcr Pharmaceuticals Co., Ltd. · TSE:4552

Jcr Pharmaceuticals — H1 FY2023 Earnings Presentation

44 slides from Jcr Pharmaceuticals's earnings presentation, Nov 2, 2023

  1. Slide 1: FY2023 First-Half Results Briefing Session

    FY2023 First-Half Results Briefing Session November 2, 2023 JCR Pharmaceuticals Co., Ltd. 【Securities code】4552, PRIME. TSE 【Contacts】ir-info@jp.jcrpharm.com Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved.

  2. Slide 2: Agenda

    Agenda 1. JCR’s Goal in Mid 2030s 2. FY2023 1st-Half Financial Results 3. JCR Activities for Further Growth 1. Domestic sales products 2. R&D Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. FY2023_2Q_1

  3. Slide 3: JCR's Goal in Mid 2030s

    JCR’s Goal in Mid 2030s Yoh Ito Senior Corporate Officer, Corporate Strategy Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. FY2023_2Q_2

  4. Slide 4: JCR will continue to focus on Rare Diseases

    JCR will continue to focus on Rare Diseases No fear of patent cliff: Low risk of biosimilar penetration in LSDs – Long product process know-how defines the product. life-cycles Robust income stream from Internal manufacturing capabilities domestic product sales, asset allow parallel progression of m

  5. Slide 5: JCR's Revenue Growth Strategy

    JCR’s Revenue Growth Strategy １ Domestic Products 2 Portfolio Assets 3 Platform Technologies Neuro-Degeneration, neuro-oncology, neuro-muscular, ocular diseases Continuous Revenue Advance existing Entry into indications with Stream portfolio exponential revenue to sustain investment into to leverage

  6. Slide 6: More Stable Profit Structure

    More Stable Profit Structure Enhanced sales from GROWJECT® enabled a more stable profit structure, allowing to cover R&D and SG&A expenses solely through revenue from domestic products. ～FY2022 FY2023～ Our core products, mainly GROWJECT®, sales increase Licenses R&D Products SG&A Gross Profit Cost G

  7. Slide 7: Estimated Timeline to Approval and Revenue from LSD Assets

    Estimated Timeline to Approval and Revenue from LSD Assets Revenue potential* Global Stage Estimated time to approval (bn yen; by mid-2030s) Partner FY2025 FY2030 FY2035 Japan Ex-Japan JR-141 P3 (Global) 8-10 14-18 (MPS II) Launched (JP) FY2027 JR-171 P1/2 (Global) 2-3 6-10 (MPS I) FY2028-2029 JR-44

  8. Slide 8: J-Brain Cargo as Door-Opener into broader CNS Indications

    J-Brain Cargo® as Door-Opener into broader CNS Indications 50 million patients (WW) J-Brain Cargo® to deliver drugs into the CNS $57 billion Neurodegenerative Fab Diseases Anti TfR mAb scFv • Alzheimer's Disease VHH • Parkinson's Disease • Epilepsy • ALS • Multiple Sclerosis • SMA • Huntington Disea

  9. Slide 9: Be a 100 bn Yen Company by mid-2030s based on Three Pillars

    Be a 100 bn Yen Company by mid-2030s based on Three Pillars Future Sources of Revenue 3 Revenue from platform technology licensing 2 Revenue from 34.3 billion yen portfolio licensing (Global LSD) 1 Revenue from Domestic Domestic products products FY2022 Mid 2030s Copyright © 2023 JCR Pharmaceuticals

  10. Slide 10: FY2023 1st-Half Financial Results

    FY2023 1st-Half Financial Results Yoshihiro Oota Director, Accounting Department, Corporate Strategy Division Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. FY2023_2Q_9

  11. Slide 11: Financial Highlights (Apr. 1, 2023 - Sep. 30, 2023)

    Financial Highlights（Apr. 1, 2023- Sep. 30, 2023） Strong sales of core products and higher contract income contributed to a significant year-on-year increase in revenues and profits. Net sales ： 24,272million yen （YoY +55.8％） Operating income ： 6,898million yen （YoY +826.2％） Ordinary income ： 7,126m

  12. Slide 12: Breakdown of Net Sales

    Breakdown of Net Sales (Unit: million yen) FY2022 FY2023 Core products sales increased 31.1% YoY Six months Six months YoY Full year ended ended Forecast Progress due to strong sales of GROWJECT® and Difference Ratio Rate Sep. 30, 2022 Sep. 30, 2023 (Revised) other core products, IZCARGO® and Core p

  13. Slide 13: Operating Income

    Operating Income (Unit: million yen) FY2022 FY2023 Six months Six months Consolidated Six months Six months YoY Full year ended ended Progress ended ended Forecast Sep. 30, 2022 Sep. 30, 2023 Difference Rate Rate Sep. 30, 2022 Sep. 30, 2023 (Revised) Net Sales 15,581 24,272 +8,691 ＋55.8% 45,400 53.5

  14. Slide 14: JCR Activities for Further Growth: 1. Domestic sales products

    JCR Activities for Further Growth 1. Domestic sales products Toru Ashida Senior Vice President, Sales Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. 13

  15. Slide 15: Sales of Core Products

    Sales of Core Products Strong sales of core products contributed significantly to the large increase in profit and sales. FY2022 FY2023 Six months Six months YoY Full year Progress Full Year ended ended Forecast Rate Sep. 30, 2022 Sep. 30, 2023 Difference Rate (Revised) GROWJECT® 12,261 6,083 8,746

  16. Slide 16: GH Market Trends in Japan

    GH Market Trends in Japan Demand for GROWJECT® has surged since May 2023. JCR successfully achieved to increase production and stable supply of drug to patients. >50% GH Market Share in Japan Market share in the future 60% 50% 49.8% 40% FY2023 1st-Half Results* 30% • Number of units shipped YoY appr

  17. Slide 17: Hospital Market Characteristics

    Analysis of the Domestic GH Market* Domestic GH market* Hospital Market Characteristics ⚫ More opportunities for SGA, Turner and General Practitioner SHOX with higher-dose per weight. (GP) ⚫ Hospital specialists serve a KOLs to 28.5% influence the entire healthcare community. GH市場 GH Market Hospital

  18. Slide 18: GROWJECT Took the Top Market Share for All Sectors

    GROWJECT® Took the Top Market Share for All Sectors Significant expansion in the HP market, a long-standing challenge. Sep. 2022 Sep. 2023 HP Market HP Market Number of facilities supplied with GROWJECT®* (Compared to FY2022) JCR JCR HP：approx. 1.2x 15% 42% － New accounts ：180 GP : approx. 1.4x GP M

  19. Slide 19: Our Sales Strategy for Further Growth

    Our Sales Strategy for Further Growth Strong and stable supply Device strategy Two types of devices to meet the needs of ⚫ Already implemented a system to patients and healthcare professionals secure the production volumes necessary to maintain a market share of more than 50% ⚫ Full support for inje

  20. Slide 20: Motorized Digital Injector GROWJECTOR

    Motorized Digital Injector “GROWJECTOR®” Providing motorized digital injectors that suit the patient's needs/lifestyle Main features of motorized digital injector： Constant speed of drug infusion, pre-setting and automatic calculations of doses, automatic adjustment of doses during drug changes, rec

  21. Slide 21: IZCARGO Prescription Status

    IZCARGO® Prescription Status 100 売上高（百万円） Net Sales (million yen) 累積症例数（例） 投与症例数（例） Cumulative (case) Under medication (cases) 85 74 77 80 71 67 83 59 60 60 45 69 70 62 64 57 60 60 40 56 17 43 20 761 1,059 957 1,070 1,047 1,262 1,047 1,239 1,342 224 0 2021年度 FY2021 2021年度 FY2021 2021年度 FY2021 2021年度

  22. Slide 22: TEMCELL Prescription Status

    TEMCELL® Prescription Status Sales trends (Unit: million yen) 3,497 3,404 3,300 TEMCELL® sales status update 1,901 Available in more than 90% of target facilities With the cooperation of MEDICEO CORPORATION, the lead time was reduced from 3 to 2 days. Number of 331 326 172 Changes in the prevention

  23. Slide 23: JCR Activities for Further Growth: 2. R&D

    JCR Activities for Further Growth 2. R&D Mathias Schmidt,PD,Ph.D. Vice President, Clinical development (overall supervision) Business development and IR fields, excluding Japan Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. 22

  24. Slide 24: R&D Pipeline

    R&D Pipeline Updated Code Indication Status Upcoming Milestones JR-141 MPS II (Hunter syndrome) Global Ph3 ~FY2027: Approval in US, EU, Brazil JR-171 MPS I (Hurler syndrome etc.) Global Ph1/2 completed FY2024: Ph3 JR-441 MPS IIIA (Sanfilippo syndrome type A ) Global Ph1/2 1st Half FY2024: LPI JR-446

  25. Slide 25: LSD Pipeline using J-Brain Cargo Technology

    LSD Pipeline using J-Brain Cargo® Technology Built an industry-leading portfolio in the LSD space of over $10billion. JR-141 MPS II Approved Japan （Hunter) JR-141 MPS II JR-171 MPS I JR-441 MPS IIIA Clinical Global （Hunter) Global （Hurler etc.) Global （Sanfilippo A) JR-162 Pompe MPS IIIB IND enablem

  26. Slide 26: MPS III Disease Overview

    MPS III Disease Overview LSD MPS I MPS III Type A • Autosomal recessive disorder causing accumulation • Congenital metabolic • Accumulation of II disease caused by mucopoly- of toxic heparan sulfate in several tissues and organs III Type B deficiency in lysosomal saccharides • CNS signs and symptoms

  27. Slide 27: JR-441 (BBB-penetrating ERT for MPS IIIA)

    JR-441 (BBB-penetrating ERT for MPS IIIA) July 2023 - Approval of Global Phase I/II study (JR-441-101) JR-441-101 study overview Achievements and next milestones Treatment Drug Jan -2022 period holiday Extension period EC grants Orphan Drug Designation Jul -2023 Dose 2 Approval of Global Ph I/II Cli

  28. Slide 28: JR-446 (BBB-penetrating ERT for MPS IIIB)

    JR-446（BBB-penetrating ERT for MPS IIIB) Sep-2023 MEDIPAL HOLDINGS and JCR Conclude Two Agreements on JR-446 Japan Rest of World JR-446 Status Agreement Collaboration agreement Licensing agreement Established molecular design, JCR MEDIPAL HD optimized for activity and MA holder (jointly developed wi

  29. Slide 29: JR-141 (pabinafusp alfa: BBB-penetrating ERT for MPS II)

    JR-141(pabinafusp alfa: BBB-penetrating ERT for MPS II ） Global Phase III study（JR-141-GS31): STARLIGHT study Overview Upcoming milestone 1QFY2024 - all patients enrolled necessary for interim analysis Current Status Recruiting Number of Clinical trial sites (as of Oct 2023): • USA: 5, Europe: 10, B

  30. Slide 30: JR-142 (Long-acting GH)

    JR-142（Long-acting GH） The growth promoting effect of JR-142 was confirmed in Phase II trial. Phase III trial scheduled to start in FY2024. JR-142 molecular design Phase 2 study design Modified albumin-fused GH 0.75mg/kg/w (n=9) JR-142 human albumin (weekly) Extension 0.5mg/kg/w (n=8) study hGH GROW

  31. Slide 31: JR-142 Ph2 results: Comparable Safety and Efficacy Profile

    JR-142 Ph2 results: Comparable Safety and Efficacy Profile Key outcomes: • Height velocity: Comparable between GROWJECT® and JR-142. • AEs/safety profile: Comparable between all groups. Outcome of Phase 2 Trial (height velocities) Next milestones ■他社持続型GH 2nd-Half FY2024 Start Ph3 Growth velocity (c

  32. Slide 32: Other Activities to enhance Access to Medicine

    Other Activities to enhance Access to Medicine JR-031HIE: Expanded indication of TEMCELL® HS inj. for Hypoxic ischemic encephalopathy in neonates (detail: jRCT1080224818) – Future development plan under consideration based on results of Ph I/II and 18-month observational studies. • Comparative study

  33. Slide 33: FY2023_2Q_32

    Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. FY2023_2Q_32

  34. Slide 34: Appendix

    Appendix Copyright © 2023 JCR Pharmaceuticals Co., Ltd. All rights reserved. FY2023_2Q_33

  35. Slide 35: Highlights (May 11, 2023 - Oct. 27, 2023)

    Highlights（May 11, 2023- Oct. 27, 2023) Category Date News R&D 11 May Novel Biologic Therapies in Epilepsy Collaboration with Angelini Pharma Sustainability 14 May LSD-related webinars for the general public Core products 23 May IZCARGO® Add of injectable drugs that can be administered by insurers t

  36. Slide 36: Product history of GROWJECT administration device

    Product history of GROWJECT® administration device Improved devices, a key factor in prescribing, at an unparalleled pace. As the only domestic manufacturer, continues to develop devices that meet the needs of healthcare professionals and patients in Japan. Only domestic motorized digital injector o

  37. Slide 37: Pipeline of LSDs

    Pipeline of LSDs JR-141 pabinafusp alfa: BBB-penetrating iduronate-2-sulfatase (rDNA origin) Indication : MPS type II (Hunter syndrome) Patient population*¹ : 150-200 (Japan) ,2,000-3,000 (WW) est. Est. Market size*² : 8.0-10.0 billion JPY (Japan), 90.0 billion JPY (WW) Disease overview : Hunter syn

  38. Slide 38: Pipeline of LSDs

    Pipeline of LSDs JR-441 BBB-penetrating heparan N-sulfatase (rDNA origin) Indication : MPS type III A (Sanfilippo A syndrome) Patient population*¹ : 10 (Japan）, 1,000-2,000 (WW) est. Est. Market size*² : 1.0-2.0 billion JPY (Japan), 60.0 billion JPY (WW) Disease overview : An autosomal recessive dis

  39. Slide 39: Pipeline of LSDs

    Pipeline of LSDs JR-443 BBB-penetrating β-glucuronidase (rDNA origin) Indication : MPS type VII（Sly syndrome） Patient population*¹ : <10 (Japan) , 100-200 (WW) est. Est. Market size*² : 3.0 billion JPY (WW） Disease overview : An autosomal recessive disease caused by deficiency of an enzyme, β-glucur

  40. Slide 40: Pipeline of LSDs

    Pipeline of LSDs JR-479 BBB-penetrating β-Hexosaminidase A (rDNA origin) Indication : GM2 gangliosidosis (Tay-Sachs disease, Sandohoff disease) Patient population*¹ : 20 (Japan), 1,000-2,000(WW) est. Est. Market size*² : 2.0-3.0 billion JPY (Japan), 55.0 billion JPY (WW) Disease overview : GM2 gangl

  41. Slide 41: Pipeline of Other Compounds

    Pipeline of Other Compounds JR-142 Long-acting growth hormone (rDNA origin) Indication : Pediatric growth hormone deficiency Note : JCR’s proprietary half-life extension technology, based on a novel modified albumin, allows significant increase in the half-life of various biotherapeutics （Patent fil

  42. Slide 42: Leveraging Growth Opportunities from J-Brain Cargo

    Leveraging Growth Opportunities from J-Brain Cargo® Partnerships are at the core of JCR’s growth and acceleration strategy Internal Expand/build footprint in USA and Europe Development LSD Portfolio Partner focus: Partnerships Synergy in capacity and commercialization Lysosomal Storage Muscle delive

  43. Slide 43: Abbreviations (A-G)

    Abbreviations （A～G） AE Adverse Events 有害事象 ANVISA Brazilian Health Surveillance Agency ブラジル国家衛生監督庁 BBB Blood-Brain Barrier 血液脳関門 CDMO Contract Development and Manufacturing Organization 医薬品開発製造受託機関 CNS Central Nervous System 中枢神経系 CSF Cerebrospinal fluid 脳脊髄液 DP Darbepoetin Alfa BS Inj. [JCR] ダルべポエチ

  44. Slide 44: Abbreviations (H-Z)

    Abbreviations （H～Z） HIE Hypoxic ischemic encephalopathy in neonates 低酸素性虚血性脳症 HS Heparan Sulfate ヘパラン硫酸 HSCT Hematopoietic Stem Cell Transplantation 造血幹細胞移植 i.v. Intravenous Injection 静脈注射 IAR Infusion-associated reactions 投与時反応 JBC J-Brain Cargo® Technology J-Brain Cargo®技術 KOL Key Opinion Leader 影

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