Jcr Pharmaceuticals Co., Ltd. · TSE:4552
Jcr Pharmaceuticals — Q1 FY2025 Earnings Presentation
24 slides from Jcr Pharmaceuticals's earnings presentation, Jul 30, 2025
Slide 1: FY2025 First Quarter Results Conference Call
FY2025 First Quarter Results Conference Call July 30, 2025 JCR Pharmaceuticals Co., Ltd. [Securities code]4552, Prime. TSE [Contacts] ir-info@jp.jcrpharm.com
Slide 2: Disclaimer Regarding Forward-Looking Statement
Disclaimer Regarding Forward-Looking Statement • This material contains forecasts, projections, goals, plans, and other forward-looking statements regarding the Company’s financial results and other data. Such forward-looking statements are based on the Company’s assumptions, estimates, outlook, and
Slide 3: FY2025 First Quarter Consolidated Financial Results
FY2025 First Quarter Consolidated Financial Results Yoh Ito Senior Executive Officer Executive Director, Corporate Strategy Division
Slide 4: Overview: Consolidated Financial Results
Overview: Consolidated Financial Results (Unit：million yen) FY2024 FY2025 Additional Remarks Consolidated Year-on-year Progress Q1 YTD Q1 YTD Rate • Net Sales increased 5.2% year over year. Difference Ratio • Cost of sales ratio (Excluding income from Net Sales 8,145 8,569 +424 +5.2% 22.7% contractu
Slide 5: Breakdown of Net Sales (Consolidated)
Breakdown of Net Sales (Consolidated) (Unit: million yen) FY2024 FY2025 Additional Remarks Consolidated Year-on-year Progress Q1 YTD Q1 YTD Rate Difference Ratio • GROWJECT®, IZCARGO®, and TEMCELL® HS Inj. all outperformed GROWJECT® 4,649 4,495 (154) (3.3)% 25.3% internal budgets and maintained stro
Slide 6: Licensing-out of JUST-AAV
Licensing-out of JUST-AAV July 2025 License agreement with Alexion for JUST-AAV capsids JUST-AAV • Alexion may use the licensed capsids, which are part of the JUST-AAV platform, in up to five of Alexion's genomic AAV with directionality to target tissues/organs medicine programs and reduced migratio
Slide 7: Collaboration to Develop Therapy for Alzheimer's Disease
Collaboration to Develop Therapy for Alzheimer’s Disease July 2025 Joint collaboration, option and license agreement on J-Brain Cargo® with Acumen • To develop blood-brain barrier-penetrating treatment for Alzheimer’s disease - Combines JCR’s J-Brain Cargo® with Acumen’s AβO-selective antibodies - U
Slide 8: Selected for Japan's Regenerative Medicine CDMO Subsidy Program
Selected for Japan’s Regenerative Medicine CDMO Subsidy Program Regenerative CDMO Subsidy • Subsidy program by the Ministry of Economy, Trade and Industry • Supports the development of domestic CDMO facilities and talent related to regenerative, cell, and gene therapies Long-term product supply expe
Slide 9: Progress on Developmental Pipelines
Progress on Developmental Pipelines Anne Bechet Senior Executive Officer Executive Director, Development Division General Manager, JCR Europe B.V. General Manager, JCR USA Inc.
Slide 10: JR-141 Global Phase III STARLIGHT study Overview
JR-141 (pabinafusp alfa: BBB-penetrating ERT for MPS II) Global Phase III study（JR-141-GS31): STARLIGHT study Overview • Enrollment of the target number of 80 participants achieved • Constructive meeting took place with FDA in June 2025 - Ongoing discussion on path forward to BLA 9
Slide 11: Overview of Clinical or late Preclinical Pipeline
Overview of Clinical or late Preclinical Pipeline Status Code Indication Milestones/Comments Preclinical Phase 1 Phase 2 Phase 3 • Achievement of the patient enrollment JR-141 MPS II (Hunter syndrome) Global Ph3 • ~FY2027: Approval in US, EU, Brazil JR-142 Pediatric GHD Ph3 (Japan) • Dec 2024: Initi
Slide 12: Reach Beyond, Together
Reach Beyond, Together 2 ICR 50 th —IZ, TOKEN ” ~\ Pharmaceuticals ANNIVERSARY
Slide 13: Appendix
Appendix
Slide 14: GROWJECT Market Share Trends in Japan (Quarterly)
GROWJECT® Market Share Trends in Japan (Quarterly) GH Market Share in Japan （FY2023 Q3～FY2025 Q1）*On an NHI drug price basis ■ Human Growth Hormone Market in Japan General 50.0% Practitioner(GP) 45.3% 31.0% Hospital 43.9% (HP) 45.0% 41.6% 41.8% 41.2% 41.2% 43.7% 40.3% 40.0% 34.6% 35.0% 32.9% 31.7% 3
Slide 15: GROWJECT Sales Trends in Japan (Quarterly)
GROWJECT® Sales Trends in Japan (Quarterly) ◆Breakdown of Shares in June 2025 Daily ：79% Weekly ：21% Copyright © 2025 IQVIA. Own analysis based on JPM (Oct 2023-Jun 2025). Reprinted with permission 14
Slide 16: GROWJECT Trends of Market Share in Japan
GROWJECT® Trends of Market Share in Japan (billion yen) 6,000,000,000 6.0 45.0% 40.0% 5.0 5,000,000,000 35.0% Weekly Sales 4.0 4,000,000,000 30.0% Daily Sales （ except GROWJECT® ） 25.0% GROWJECT® Sales 3.0 3,000,000,000 Daily Shares 20.0% （ except GROWJECT® ） GROWJECT® Shares 2.0 2,000,000,000 15.0%
Slide 17: IZCARGO Prescription Status in Japan
IZCARGO® Prescription Status in Japan (million yen) 売上高（百万円） Sales(million yen) 投与症例数（例） Administered cases 累積症例数（例） Cumulative cases (cases) (cases) (cases) 1,800 120 106 1,600 100 96 92 92 100 88 90 1,400 85 83 1,200 80 85 78 80 1,000 74 75 75 70 72 69 60 800 600 40 400 20 200 1,215 1,342 1,413 1,
Slide 18: FY2025 Consolidated Financial Forecasts
FY2025 Consolidated Financial Forecasts (Unit：million yen) Additional Remarks • Net sales is expected to increase year on year, as growth FY2024 FY2025 (Forecast) in IZCARGO® sales and higher licensing income are likely to outweigh. Consolidated • Cost of sales is expected to decline year on year, a
Slide 19: Breakdown of Net Sales – FY2025 Consolidated Financial Forecasts
Breakdown of Net Sales – FY2025 Consolidated Financial Forecasts (Unit：million yen) FY2024 FY2025 (Forecast) Consolidated Year-on-year Results Forecast Difference Ratio GROWJECT® 18,098 17,800 (298) (1.6)% IZCARGO®* 5,718 6,400 +681 +11.9% TEMCELL®HS Inj. 2,904 2,700 (204) (7.0)% Treatments for rena
Slide 20: JUST-AAV: Novel Platform Technology Applicable to Various Diseases
JUST-AAV：Novel Platform Technology Applicable to Various Diseases JCR JUST-AAV Ultimate Destination of Organ Safeguarding against off-target delivery Transformative Technology AAV: Adeno-Associated Virus JBC: J-Brain Cargo® AAV with directionality to target tissues/organs and reduced migration to sp
Slide 21: Mechanism of Action of JUST-AAV
Mechanism of Action of JUST-AAV (in case of brain-targeting type) AAV capsid Therapeutic gene X X Transgene expression in the brain X AAV-production Brain plasmid with JBC tag JUST-AAV BBB sequence Transferrin Vasculature receptor Transferrin Transition of blood-brain barrier via transferrin recepto
Slide 22: Shaping the Future with JCR's Proprietary Technologies
Shaping the Future with JCR’s Proprietary Technologies Lysosomal Storage Partnering our groundbreaking technologies and creating Disorders breakthrough therapies in various disease areas beyond rare Neurodegeneration JUST-AAV Muscular Diseases J-Brain Cargo® ･･･ Neuroinflammation Neuro-oncology ･･･
Slide 23: Abbreviations (A～G)
Abbreviations (A～G) AAV Adeno-Associated Virus アデノ随伴ウイルス AβO Amyloid Beta Oligomer アミロイドベータオリゴマー API Active Pharmaceutical Ingredient 原薬 ASO Antisense oligonucleotides アンチセンス核酸 BBB Blood-Brain Barrier 血液脳関門 BLA Biologics License Application 生物製剤承認申請 CDMO Contract Development and Manufacturing Organi
Slide 24: Abbreviations (H～Z)
Abbreviations (H～Z) HS Heparan Sulfate ヘパラン硫酸 i.v. Intravenous Injection 静脈注射 JBC J-Brain Cargo® - LNP Lipid nanoparticle 脂質ナノ粒子 MPS Mucopolysaccharidosis ムコ多糖症 NPS Named Patient Supply 特定の患者への医薬品提供プログラム ODD Orphan Drug Designation 希少疾病用医薬品指定 Ph I Phase I 臨床第１相試験 Ph II Phase II 臨床第２相試験 Ph III Phase
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