Jcr Pharmaceuticals Co., Ltd. · TSE:4552

Jcr Pharmaceuticals — FY2023 Earnings Presentation

22 slides from Jcr Pharmaceuticals's earnings presentation, May 13, 2024

Slide 1: FY2023 Results Briefing Session
FY2023 Results Briefing Session May 13th, 2024 JCR Pharmaceuticals Co., Ltd. [Securities code]4552, Prime. TSE Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 2: Key Messages
Key Messages FY2023 Revenue and earnings growth from increasing sales of core products FY2024 Strengthen organizational structure to support global clinical development and increase R&D investment − Expand clinical development team and establish an integrated structure of experts in Japan, US, Europ
Slide 3: Progress of Developmental Pipelines
Progress of Developmental Pipelines Anne Bechet Development (Acting Officer) General Manager, JCR Europe B.V. Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. FY2023_4Q_2
Slide 4: 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 Recruiting Number of Clinical trial sites (as of Apr 2024): • US: 5 • Europe: 12 • Latin America: 4 Trial is on going in 10 countries • Further sites to open in EU, U
Slide 5: JR-441 (BBB-penetrating ERT for MPS IIIA)
JR-441 (BBB-penetrating ERT for MPS IIIA) July 2023 – Started Global Phase I/II study (JR-441-101) in Germany JR-441-101 study overview Achievements and next milestones Treatment Drug Jan -2022 period Extension period holiday EC grants Orphan Drug Designation Jul -2023 Approval of Global Ph I/II Cli
Slide 6: R&D Pipeline
R&D Pipeline Code Indication Status Upcoming Milestones • Q1 FY2024: All patients enrolled JR-141 MPS II (Hunter syndrome) Global Ph3 necessary for interim analysis • ~FY2027: Approval in US, EU, Brazil • Extension study is ongoing JR-171 MPS I (Hurler syndrome etc.) Global Ph1/2 completed • Licensi
Slide 7: Next Wave of Technology Innovation
Next Wave of Technology Innovation Hiroyuki Sonoda, Ph. D. Vice President Executive Director, Research Division Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. FY2023_4Q_6
Slide 8: History of Technological Innovation
History of Technological Innovation Advanced technology and know-how in biopharmaceuticals developed since the company’s formation in 1975 Development of proprietary JCR technologies Stock solutions for COVID-19 Vaccine (AZD1222) J-Brain Cargo® Tech Biosimilars Stem Cell Therapies Recombinant Protei
Slide 9: Applying J-Brain Cargo to Gene Therapy
Applying J-Brain Cargo® to Gene Therapy Treatment with AAV expressing Treatment with modified AAV JBC-fusion protein expressing JBC tag for direct brain penetration AAV capsid JBC X X Therapeutic protein X Gene cassette JCR proprietary gene therapy technology applying J-Brain Cargo® Treatment strate
Slide 10: Characteristics of Gene Therapy used J-Brain Cargo
Characteristics of Gene Therapy used J-Brain Cargo® Treatment with AAV expressing Treatment with AAV General AAV gene therapy JBC-fusion protein carrying JBC surface tag • JBC-AAV can cross the blood- • High doses are • Transgene product, JBC-X, X brain barrier X required to penetrate can reach the
Slide 11: Shaping the Future with our own Technology
Shaping the Future with our own Technology Creation of breakthrough therapeutics in various disease areas • Expansion of partnering opportunities Lysosomal Storage Diseases • Increasing R&D collaborations Widely applicable technology Neurodegenerative based on J-Brain Cargo® Diseases Ocular Diseases
Slide 12: FY2023 Financial Results
FY2023 Financial Results Yoh Ito Senior Corporate Officer Executive Director, Corporate Strategy Division Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. FY2023_4Q_11
Slide 13: Consolidated Financial Results
Consolidated Financial Results （Unit: million yen) FY2022 FY2023 Consolidated Year-on-year Full-year results results forecast Progress rate Difference Ratio (Revised) Net sales 34,343 42,871 +8,528 ＋24.8% 45,400 94.4% Cost of sales 8,886 11,620 +2,733 ＋30.8% 12,400 93.7% Gross profit 25,456 31,251 +
Slide 14: Breakdown of Net Sales – Consolidated
Breakdown of Net Sales – Consolidated （Unit: million yen) FY2022 FY2023 Year-on-year Full year results results forecast Progress rate Difference Ratio (Revised) GROWJECT® 12,261 17,913 +5,652 +46.1% 19,500 91.9% IZCARGO®* 4,414 5,171 +757 +17.2% 5,200 99.4% TEMCELL®HS Inj. 3,404 3,236 (168) (4.9)% 3
Slide 15: Consolidated Financial Results FY2024 (Forecast)
Consolidated Financial Results FY2024 (Forecast) （Unit: million yen） FY2023 FY2024(forecast) Consolidated Year-on-year results forecast Difference Ratio Net sales 42,871 41,300 (1,571) (3.7)% Cost of sales 11,620 10,400 (1,220) (10.5)% Gross profit 31,251 30,900 (351) (1.1)% Selling, general and adm
Slide 16: Breakdown of Net Sales – Consolidated FY2024 (Forecast)
Breakdown of Net Sales – Consolidated FY2024 (Forecast) （Unit: million yen） FY2023 FY2024(forecast) Year-on-year results forecast Difference Ratio GROWJECT® 17,913 18,300 +387 +2.2% IZCARGO®* 5,171 5,700 +529 +10.2% TEMCELL®HS Inj. 3,236 2,800 (436) (13.5)% Treatments for renal anemia 4,652 4,200 (4
Slide 17: Aggressive R&D Investments
Aggressive R&D Investments Investments in global clinical development remarkably increased 14,000 13,000 R＆D Expenses (Unit: million yen) +15.7% 12,000 11,234 +27.6% 10,000 8,802 +22.7% 8,000 7,175 +33.9% in Research 6,000 5,360 287 people in Research in Research in Research in Research (Planned) 15
Slide 20: What is Named Patient Supply (NPS)?
What is Named Patient Supply (NPS)? Named Patient Supply (NPS) A program whereby a manufacturer makes individual drugs available to physicians who wish to use the drug to patients in countries where it is not approved. JCR IZCARGO® NPS A program to make IZCARGO® (Japanese product) available to patie
Slide 21: Abbreviations (A～G)
Abbreviations （A～G） AAV Adeno-Associated Virus アデノ随伴ウイルス 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 EC European Commission 欧州委員会 EMA European Medicines Agency 欧州医薬品庁 ERT Enzyme Replacement Therapy 酵素補充療法 EU European Union 欧州連合 FDA Food and Drug Admini
Slide 22: 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 i.v. Intravenous Injection 静脈注射 JBC J-Brain Cargo® - 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 Ph

JCR Pharmaceuticals — FY2023 Earnings Presentation on 6ix

Jcr Pharmaceuticals — FY2023 Earnings Presentation

Slide 1: FY2023 Results Briefing Session

Slide 2: Key Messages

Slide 3: Progress of Developmental Pipelines

Slide 4: JR-141 (pabinafusp alfa: BBB-penetrating ERT for MPS II)

Slide 5: JR-441 (BBB-penetrating ERT for MPS IIIA)

Slide 6: R&D Pipeline

Slide 7: Next Wave of Technology Innovation

Slide 8: History of Technological Innovation

Slide 9: Applying J-Brain Cargo to Gene Therapy

Slide 10: Characteristics of Gene Therapy used J-Brain Cargo

Slide 11: Shaping the Future with our own Technology

Slide 12: FY2023 Financial Results

Slide 13: Consolidated Financial Results

Slide 14: Breakdown of Net Sales – Consolidated

Slide 15: Consolidated Financial Results FY2024 (Forecast)

Slide 16: Breakdown of Net Sales – Consolidated FY2024 (Forecast)

Slide 17: Aggressive R&D Investments

Slide 18: Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.

Slide 19: Appendix

Slide 20: What is Named Patient Supply (NPS)?

Slide 21: Abbreviations (A～G)

Slide 22: Abbreviations (H～Z)