Jcr Pharmaceuticals Co., Ltd. · TSE:4552
Jcr Pharmaceuticals — Q2 FY2025 Earnings Presentation
30 slides from Jcr Pharmaceuticals's earnings presentation, Oct 30, 2025
Slide 1: FY2025 Second Quarter Results Briefing Session
Life is Rare FY2025 Second Quarter Results Briefing Session October 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 Life is Rare • 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,
Slide 3: FY2025 Second Quarter Financial Results
Life is Rare FY2025 Second Quarter Financial Results Yoh Ito Senior Executive Officer Executive Director, Corporate Strategy Division
Slide 4: Overview: Consolidated Financial Results
Overview: Consolidated Financial Results Life is Rare (Unit：million yen) FY2024 FY2025 Additional Remarks Consolidated Year-on-year Progress Q2 YTD Q2 YTD • Net Sales increased 28% year on year, mainly Difference Ratio Rate reflecting higher upfront and milestone Net Sales 16,657 21,362 +4,705 +28.2
Slide 5: Breakdown of Net Sales (Consolidated)
Breakdown of Net Sales (Consolidated) Life is Rare (Unit: million yen) FY2024 FY2025 Additional Remarks Consolidated Year-on-year Progress Q2 YTD Q2 YTD Rate Difference Ratio • GROWJECT®, IZCARGO®, and TEMCELL HS Inj. all outperformed ® GROWJECT® 9,401 8,915 (486) (5.2)% 50.1% internal budgets and m
Slide 6: Financial Status (Consolidated)
Financial Status (Consolidated) Life is Rare （Unit: million yen） Change Change End-Mar. End-Sep. End-Mar. End-Sep. • Main • Main 2025 2025 2025 2025 Increase/decrease Increase/decrease +4,776 +3,520 • Cash and deposits +2,425 Current • Short-term borrowings • Accounts receivable - trade, 43,988 47,5
Slide 7: Overseas Expansion of an Approved Product in Japan
Overseas Expansion of an Approved Product in Japan Life is Rare Agalsidase Beta BS I.V. Infusion [JCR] • Biosimilar therapeutic for Fabry disease • Commercially available in Japan since 2018 An exclusive licensing agreement for marketing authorization and commercialization was signed with Menagen Ph
Slide 8: Progress of Developmental Pipelines
Life is Rare Progress of Developmental Pipelines Anne Bechet Senior Executive Officer Executive Director, Development Division General Manager, JCR Europe B.V. General Manager, JCR USA Inc.
Slide 9: Overview of Clinical or Late Preclinical Pipeline
Overview of Clinical or Late Preclinical Pipeline Life is Rare Status Code Indication Milestones/Comments Preclinical Phase 1 Phase 2 Phase 3 • On track for ~FY2027: Approval in US, EU, JR-141 MPS II (Hunter syndrome) Global Ph3 Brazil JR-142 Pediatric GHD Ph3 (Japan) • Patient recruitment is on tra
Slide 10: Gene Therapy Platform Technology: Advancement of JUST-AAV Technology
Life is Rare Gene Therapy Platform Technology ~Advancement of JUST-AAV Technology~ Hiroyuki Sonoda, Ph. D. Director, Senior Managing Executive Officer Executive Director, Research Division
Slide 11: Challenges of AAV Gene Therapy
Challenges of AAV Gene Therapy Life is Rare Difficulty in delivery to target tissues Safety issues − Central nervous system, muscle, cartilage, − Acute liver toxicity, thrombotic etc. microangiopathy, neurotoxicity2 − AAVs do not cross the blood-brain barrier1 − Deaths due to liver injury have occur
Slide 12: JUST-AAV: Novel Platform Technology Applicable to Various Diseases
JUST-AAV：Novel Platform Technology Applicable to Various Diseases Life is Rare JCR JUST-AAV Ultimate destination of organ Safeguarding against off-target delivery Transformative technology AAV: Adeno-Associated Virus ex. CNS, Muscle AAV with directionality to target tissues/organs and reduced accumu
Slide 13: Evaluation of CNS-targeting JUST-AAV Delivery Efficiency
Evaluation of CNS-targeting JUST-AAV Delivery Efficiency Life is Rare AAV9 CNS-targeting JUST-AAV Transduction of brain regions (fold change compared with AAV9) 160 (Normalized to AAV9) 140 120 x114.8 100 mRNA x76.9 80 x34.8 x62.5 x65.8 x36.5 60 x44.6 x22.1 40 20 0 CNS-targeting JUST-AAV: n=2、AAV9:
Slide 14: Evaluation of Tropism Reduction for Specific Tissues
Evaluation of Tropism Reduction for Specific Tissues Life is Rare AAV9 CNS-targeting JUST-AAV Liver Dorsal root ganglia Cervical Thoracic Lumbar 1.5E+07 2.5E+04 (copies/ug-tissue DNA) 2.0E+04 VG in tissue 1.0E+07 1.5E+04 1.0E+04 5.0E+06 11.7% 19.5% 14.1% 5.0E+03 0.3% 0.0E+00 0.0E+00 AAV9 JUST-AAV AA
Slide 15: Evaluation of Muscle-targeting JUST-AAV Delivery Efficiency
Evaluation of Muscle-targeting JUST-AAV Delivery Efficiency Life is Rare AAV9 Published capsid1 Muscle-targeting JUST-AAV Quadriceps Heart Liver 80 x 28.6 800 x 52.2 7E+6 (copies/μg-tissue DNA) Vector copy numbers 6E+6 60 600 5E+6 x 13.2 x 28.9 (µg/g-tissue) GFP Protein 4E+6 40 400 3E+6 2E+6 x0.2 20
Slide 16: Combining Tissue-specific Binders for Enhanced Utility
Combining Tissue-specific Binders for Enhanced Utility Life is Rare AAV9 CNS-targeting Muscle-targeting Dual-targeting Low High Low High Low Low High High 15
Slide 17: Exploring the Promise of Dual-targeting JUST-AAV
Exploring the Promise of Dual-targeting JUST-AAV Life is Rare AAV9 CNS-targeting Muscle-targeting Dual-targeting Brain Quadriceps Heart 40 x37.2 x35.3 20 x18.1 12 x10.5 (Normalized to AAV9) 10 30 15 x12.0 8 x7.6 mRNA 20 10 6 4 10 5 x1.0 2 x1.0 x0.7 x1.0 x0.9 x0.4 0 0 0 Barcode study (AAV pool was in
Slide 18: Shaping the Future with JCR's Proprietary Technologies
Shaping the Future with JCR’s Proprietary Technologies Life is Rare 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-
Slide 19: Life is Rare
ANNIVERSARY Life is Rare JYICR
Slide 20: Appendix
Life is Rare Appendix
Slide 21: GROWJECT Market Share Trends in Japan (Quarterly)
GROWJECT® Market Share Trends in Japan (Quarterly) Life is Rare ■ Human Growth Hormone Market in Japan GH Market Share in Japan （FY2023 Q4～FY2025 Q2）*On an NHI drug price basis General Hospital Practitioner(GP) (HP) 50.0% 31.2% 43.4% 43.9% 45.0% 41.8% 41.6% 41.2% 41.2% 40.3% 39.8% 40.0% 34.6% 35.5%
Slide 22: GROWJECT Trends of Market Share in Japan
GROWJECT® Trends of Market Share in Japan Life is Rare (billion yen) Weekly Sales 6.0 6,000,000,000 45.0% Daily Sales （ except GROWJECT® ） 40.0% GROWJECT® Sales 5,000,000,000 5.0 Daily Shares 35.0% （ except GROWJECT® ） GROWJECT® Shares 4.0 4,000,000,000 30.0% Weekly Shares 25.0% 3,000,000,000 3.0 Da
Slide 23: IZCARGO Prescription Status in Japan
IZCARGO® Prescription Status in Japan Life is Rare (cases) (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) 2,000 120 106 106 1,800 100 96 90 92 92 100 1,600 88 85 1,400 80 85 84 1,200 78 80 74 75 75 70 72 1,000 60 800 40 600 400 20 200
Slide 24: Cash Flows (Consolidated)
Cash Flows (Consolidated) Life is Rare (Unit: million yen) 営業活動によるCF Operating Activities 投資活動によるCF Investing Activities FY2024 Q2 YTD FY2025 Q2 YTD Difference 財務活動によるCF Financing Activities 期末現金及び現金同等物 Cash and Cash Equivalents at End of Period Profit before income taxes (556) 2,541 3,097 Depreciat
Slide 25: FY2025 Consolidated Financial Forecasts
FY2025 Consolidated Financial Forecasts Life is Rare (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 ye
Slide 26: Breakdown of Net Sales – FY2025 Consolidated Financial Forecasts
Breakdown of Net Sales – FY2025 Consolidated Financial Forecasts Life is Rare (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)% Treatm
Slide 27: Licensing-out of JUST-AAV
Licensing-out of JUST-AAV Life is Rare 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 red
Slide 28: Collaboration to Develop Therapy for Alzheimer's Disease
Collaboration to Develop Therapy for Alzheimer’s Disease Life is Rare 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 a
Slide 29: Selected for Japan's Regenerative Medicine CDMO Subsidy Program
Selected for Japan’s Regenerative Medicine CDMO Subsidy Program Life is Rare 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 produc
Slide 30: Abbreviations
Abbreviations Life is Rare 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 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 GFP Green fluorescent protein 緑色蛍光タンパク質 GHD Growth hormone defic
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