Jcr Pharmaceuticals Co., Ltd. · TSE:4552
Jcr Pharmaceuticals — FY2024 Earnings Presentation
31 slides from Jcr Pharmaceuticals's earnings presentation, May 13, 2025
Slide 1: FY2024 Results Briefing Session
FY2024 Results Briefing Session May 13, 2025 JCR Pharmaceuticals Co., Ltd. [Securities code]4552, Prime. TSE [Contacts] ir-info@jp.jcrpharm.com
Slide 2: FY2024 Consolidated Financial Results / FY2025 Forecasts
FY2024 Consolidated Financial Results FY2025 Consolidated Financial Forecasts Yoh Ito Senior Executive Officer Executive Director, Corporate Strategy Division
Slide 3: Overview: Consolidated Financial Results
Overview: Consolidated Financial Results (Unit：million yen) FY2023 FY2024 Additional Remarks • Both revenue and profit declined year on year. Consolidated Year-on-year Results Results • The primary factors were: (1) the absence of expected Difference Ratio licensing income following the failure to f
Slide 4: Breakdown of Net Sales (Consolidated)
Breakdown of Net Sales (Consolidated) (Unit: million yen) FY2023 FY2024 Additional Remarks Consolidated Year-on-year Results Results • GROWJECT® revenue increased despite Difference Ratio the NHI price revision in April 2024, supported by higher sales volume. GROWJECT® 17,913 18,098 +184 +1.0% • IZC
Slide 5: Financial Status (Consolidated)
Financial Status (Consolidated) （Unit: million yen） Change Change End-Mar. End-Mar. End-Mar. End-Mar. • Main • Main 2024 2025 2024 2025 Increase/decrease Increase/decrease Total (6,524) Total +13,852 • Cash and deposits (5,559) Current • Short-term borrowings • Accounts receivable - trade, 30,135 43
Slide 6: 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, as
Slide 7: 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 renal
Slide 8: 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 9: 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 Current Status Number of clinical trial sites 28 sites 12 countries Achieved over 95% of patient recruitment ⚫ Close collaboration with Overview agencies to target to file as soon a
Slide 10: Other Pipelines for Lysosomal Storage Disorders
Other Pipelines for Lysosomal Storage Disorders Achieved steady progress in clinical development for rare diseases with no available treatment BBB-penetrating heparan N-sulfatase (rDNA origin) JR-441 Indication: MPS type IIIA − Oct 2023 Initiation of Phase I/II study (Germany) − Completed patient en
Slide 11: Growth Hormone Product JR-142
Growth Hormone Product Long-acting growth hormone (rDNA origin) JR-142 Indication: Pediatric growth hormone deficiency Phase III study design Modified albumin-fused GH 0.75mg/kg/week human albumin JR-142 group (weekly) Extension 0.5mg/kg/week period hGH 1 3 GROWJECT® group 0.175mg/kg/week Switch to
Slide 12: 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 • Q3 FY2025: Enrollment completion JR-141 MPS II (Hunter syndrome) Global Ph3 • ~FY2027: Approval in US, EU, Brazil JR-142 Pediatric GHD Ph3 (Japan) • Dec 2024: Initiation
Slide 13: Progress on Research
Progress on Research Hiroyuki Sonoda, Ph. D. Director, Senior Managing Executive Officer Executive Director, Research Division
Slide 14: 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 • Q3 FY2025: Enrollment completion JR-141 MPS II (Hunter syndrome) Global Ph3 • ~FY2027: Approval in US, EU, Brazil JR-142 Pediatric GHD Ph3 (Japan) • Dec 2024: Initiation
Slide 15: Evaluation in a Mouse Model of GM2 gangliosidosis
Evaluation in a Mouse Model of GM2 gangliosidosis BBB-penetrating β-Hexosaminidase A (rDNA origin) Enzyme JR-479 replacement Indication: GM2 gangliosidoses (Tay-Sachs disease, Sandhoff disease) therapy ** ** Brain ## ## ++ ++ (weekly) HexA: β-Hexosaminidase A (weekly) ** ** Spinal cord N=9-10/group
Slide 16: Collaboration through JCR's Proprietary Technologies
Collaboration through JCR’s Proprietary Technologies Broad applicability of J-Brain Cargo® to various modalities Enzymes Oligonucleotides Proteins （ASO, siRNA） Antibodies LNP (mRNA, low-molecular weight compounds) Gene/Cell therapy Practical use of J-Brain Cargo® technology J-Brain Cargo® partnershi
Slide 17: 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 18: 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 19: Gene Therapy with JUST-AAV: Survival in a Mouse Model of CLN2
Gene Therapy with JUST-AAV: Survival in a Mouse Model of CLN2 28 weeks 22 weeks Tail coding Black: Healthy at 28 weeks Healthy Red: JUST-AAV-treated at 28 weeks None: Disease control at 22 weeks Disease JUST-AAV Nov 20, 2024_R&D meeting JUST-AAV resulted in survival of more than 370 days, similar to
Slide 20: 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 21: Reach Beyond, Together
Reach Beyond, Together MIC?
Slide 22: Appendix
Appendix
Slide 23: GROWJECT Market Share Trends in Japan (Quarterly)
GROWJECT® Market Share Trends in Japan (Quarterly) ■ Human Growth Hormone Market in Japan GH Market Share in Japan （FY2023 Q2～FY2024 Q4）*On an NHI drug price basis General 60.0% Practitioner(GP) Hospital(HP) 30.8% 44.3% 48.6% 50.0% 45.3% 43.9% 41.6% 41.8% 41.2% 41.2% 40.0% 32.9% 31.7% 30.1% Pharmacy
Slide 24: GROWJECT Sales Trends in Japan (Quarterly)
GROWJECT® Sales Trends in Japan (Quarterly) GH Sales Trends in Japan（FY2023 Q2～FY2024 Q4）*On an NHI drug price basis 8,000 16,000 GH Market in Japan （million yen） Sales amount of each product（million yen） 7,000 15,500 6,000 15,000 GH Market in Japan 5,000 JCR Growject® Daily 14,500 Product A Daily 4
Slide 25: GROWJECT Breakdown of Market Share in Japan
GROWJECT® Breakdown of Market Share in Japan (billion yen) 6,000,000,000 6.0 45.0% 40.0% 5,000,000,000 5.0 35.0% Weekly Sales 4,000,000,000 4.0 30.0% Daily Sales （ except GROWJECT® ） 25.0% GROWJECT® Sales 3,000,000,000 3.0 Daily Shares 20.0% （ except GROWJECT® ） GROWJECT® Shares 2,000,000,000 2.0 15
Slide 26: 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 1,600 100 96 92 92 100 88 90 1,400 85 83 77 1,200 80 78 80 1,000 74 75 75 70 72 69 60 64 800 600 40 400 20 200 1,047 1,215 1,342 1,4
Slide 27: Net Sales Trends by Product FY2025 (Forecast)
Net Sales Trends by Product FY2025 (Forecast) Recombinant human growth hormone product Recombinant treatment for mucopolysaccharidosis II (MPS II) （Unit: million yen） GROWJECT® IZCARGO® I.V. infusion 10mg 17,913 18,098 17,800 From April 24, 2023: Co-promotion with Sumitomo Pharma Co., Ltd. 6,400 5,7
Slide 28: Cash Flows (Consolidated)
Cash Flows (Consolidated) (Unit: million yen) FY2023 FY2024 Difference 営業活動によるCF Operating Activities 投資活動によるCF Investing Activities Profit before income taxes 7,244 (6,414) (13,658) 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 Depreciation 3,197 3,374 +176 End of Period D
Slide 29: Abbreviations (A～G)
Abbreviations (A～G) AAV Adeno-Associated Virus アデノ随伴ウイルス 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 CNS Central Nervous System 中枢神経系 CSF Cerebrospinal fluid 脳脊髄液 CTN Clinical Trial Notification 治験計画届 EC European Commission 欧州委員会 EMA
Slide 30: 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
Slide 31: 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