Jcr Pharmaceuticals Co., Ltd. · TSE:4552

Jcr Pharmaceuticals — Q2 FY2024 Earnings Presentation

26 slides from Jcr Pharmaceuticals's earnings presentation, Oct 30, 2024

Slide 1: FY2024 Second Quarter Results Briefing Session
FY2024 Second Quarter Results Briefing Session October 30, 2024 JCR Pharmaceuticals Co., Ltd. [Securities code]4552, Prime. TSE [Contacts] ir-info@jp.jcrpharm.com Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.
Slide 2: FY2024 Second Quarter Financial Results
FY2024 Second Quarter Financial Results Yoh Ito Senior Executive Officer Executive Director, Corporate Strategy Division Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. 1
Slide 3: Overview: Consolidated Financial Results
Overview: Consolidated Financial Results （Unit：million yen） FY2023 FY2024 Additional Remarks ・Revenue and profit decreased year-on-year, primarily due Consolidated Year-on-year Progress Q2 YTD Q2 YTD to a reduction in contract revenue, which is expected to be Rate recorded in the second half of the
Slide 4: Breakdown of Net Sales (Consolidated)
Breakdown of Net Sales (Consolidated) （Unit: million yen) Additional Remarks FY2023 FY2024 ・GROWJECT® underwent a (4.9%) reduction in Consolidated Year-on-year Progress reimbursement prices in April, but increased Q2 YTD Q2 YTD Difference Ratio Rate sales volume led to a 7.5% revenue growth year- on
Slide 5: Financial Status (Consolidated)
Financial Status (Consolidated) （Unit: million yen） End-Mar. End-Sep. Change End-Mar. End-Sep. Change 2024 2024 • Main 2024 2024 • Main Increase/decrease Increase/decrease Total (1,274) Total +3,998 • Cash and deposits (484) • Short-term borrowings +7,312 Current • Accounts receivable - trade, and 3
Slide 6: Progress of Developmental Pipelines
Progress of Developmental Pipelines Anne Bechet Senior Executive Officer Executive Director, Development Division General Manager, JCR Europe B.V. General Manager, JCR USA Inc. Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. 5
Slide 7: 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 October 30, 2024) 24 sites 11 countries (USA, Europe, and Latin America) Overview ⚫ The trial site opened as planned
Slide 8: Progress of Developmental Pipelines
Progress of Developmental Pipelines BBB-penetrating heparan N-sulfatase (rDNA origin) JR-441 Indication: MPS type IIIA − Japan: Phase I study (JR-441-JP11) • Completed the clinical trial notification process in Japan − Germany: Phase I/II study (JR-441-101) • Progressing as planned • Enrollment of t
Slide 9: 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 JR-141 MPS II (Hunter syndrome) Global Ph3 • ~FY2027: Approval in US, EU, Brazil • Extension study ongoing JR-171 MPS I (Hurler syndrome etc.) Global Ph1/2 completed • Par
Slide 10: J-Brain Cargo Applicability to Multiple Modalities: Application to Gene Therapy
J-Brain Cargo® Applicability to Multiple Modalities ~Application to Gene Therapy~ Hiroyuki Sonoda, Ph. D. Director, Senior Managing Executive Officer Executive Director, Research Division Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved. 9
Slide 11: J-Brain Cargo Applicability
J-Brain Cargo® Applicability Oligonucleotide (ASO, siRNA) Delivery Enzymes and Protein Delivery to the brain and muscle to the brain and muscle to modify gene expression to replace deficient or missing enzymes and proteins Antibody Delivery LNP (mRNA, low-molecular to the brain compound) Delivery in
Slide 12: Applying J-Brain Cargo to Gene Therapy
Applying J-Brain Cargo® to Gene Therapy AAV capsid X Gene cassette AAV-production plasmid with JBC JBC-AAV sequence • JCR-proprietary gene therapy technology • J-Brain Cargo® domain embedded in AAV production plasmid for viral delivery into the CNS Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All
Slide 13: Advantages of J-Brain Cargo Applied Gene Therapy
Advantages of J-Brain Cargo® Applied Gene Therapy Novel mechanism with superior brain uptake compared with conventional AAV9 Same J-Brain Cargo® technology led to approval of pabinafusp alfa in Japan No added manufacturing complexity, stable production yields • JBC tags are included in the AAV capsi
Slide 14: Non-clinical Data: Evaluation of Brain in Mice by Immunohistochemical Staining
Non-clinical Data: Evaluation of Brain in Mice by Immunohistochemical Staining untreated AAV9 JBC-AAV Inserting J-Brain Cargo® tags into AAVs enables efficient gene delivery to the brain The 7th International Forum of Lysosomal Disorders, 2024 Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All right
Slide 15: 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 18: 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 （FY2022 Q4～FY2024 Q2）*On an NHI drug price basis General Practitioner(GP) 60.0% 30.6% 54.4% Hospital (HP) 48.6% 45.1% 50.0% 45.3% 43.7% 43.9% 41.6% 41.8% 40.0% Pharmacy(PH) 31.7% 24.3%
Slide 19: GROWJECT Sales Trends in Japan (Quarterly)
GROWJECT® Sales Trends in Japan (Quarterly) GH Sales Trends in Japan（FY2022 Q4～FY2024 Q2）*On an NHI drug price basis 9,000 18,000 GH Market in Japan （million yen） Sales amount of each product（million yen） 8,000 16,000 7,000 14,000 GH Market in Japan 6,000 12,000 JCR Growject® 5,000 10,000 Product A
Slide 20: IZCARGO Prescription Status in Japan
IZCARGO® Prescription Status in Japan 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) 1,600 100 92 92 90 88 85 90 1,400 83 77 74 80 71 1,200 75 75 70 72 73 1,000 69 70 60 64 62 60 800 50 40 600 30 400 20 200 10 1,047 1,249 1,047 1,215 1,342 1,413 1,2
Slide 21: Net Sales Trends by Product
Net Sales Trends by Product （Unit: million yen） Recombinant human growth hormone product 18,300 Recombinant treatment for mucopolysaccharidosis II (MPS II) 17,913 GROWJECT® IZCARGO® I.V. infusion 10mg From April 24, 2023: Co-promotion with Sumitomo Pharma Co., Ltd. 12,945 12,261 5,700 5,171 4,414 3,
Slide 22: Cash Flows (Consolidated)
Cash Flows (Consolidated) (Unit: million yen) FY2023 1H FY2024 1H 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 7,120 (556) (7,676) 財務活動によるCF Financing Activities Cash and Cash Equivalents at 期末現金及び現金同等物 Depreciation 1,335 1,667 +331 End of Perio
Slide 23: Handling of subsidies for the construction of the Kobe Science Park Center
Handling of subsidies for the construction of the Kobe Science Park Center • Of the depreciation at Kobe Science Park Center, approximately 300 million yen was recorded as SG&A expenses and approximately 100 million yen was recorded as Non-operating expenses. Transfer of items from As of March 31, 2
Slide 24: Glossary of abbreviations (A-G)
略語集（A～G） AAV Adeno-Associated Virus アデノ随伴ウイルス 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 European Medicines Agency 欧州医薬品庁 ERT Enzyme Replace
Slide 25: Glossary of abbreviations (H-Z)
略語集（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® - 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
Slide 26: 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

JCR Pharmaceuticals — Q2 FY2024 Earnings Presentation on 6ix

Jcr Pharmaceuticals — Q2 FY2024 Earnings Presentation

Slide 1: FY2024 Second Quarter Results Briefing Session

Slide 2: FY2024 Second Quarter Financial Results

Slide 3: Overview: Consolidated Financial Results

Slide 4: Breakdown of Net Sales (Consolidated)

Slide 5: Financial Status (Consolidated)

Slide 6: Progress of Developmental Pipelines

Slide 7: JR-141 (pabinafusp alfa: BBB-penetrating ERT for MPS II)

Slide 8: Progress of Developmental Pipelines

Slide 9: Overview of Clinical or late Preclinical Pipeline

Slide 10: J-Brain Cargo Applicability to Multiple Modalities: Application to Gene Therapy

Slide 11: J-Brain Cargo Applicability

Slide 12: Applying J-Brain Cargo to Gene Therapy

Slide 13: Advantages of J-Brain Cargo Applied Gene Therapy

Slide 14: Non-clinical Data: Evaluation of Brain in Mice by Immunohistochemical Staining

Slide 15: Shaping the Future with our own Technology

Slide 16: Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.

Slide 17: Appendix

Slide 18: GROWJECT Market Share Trends in Japan (Quarterly)

Slide 19: GROWJECT Sales Trends in Japan (Quarterly)

Slide 20: IZCARGO Prescription Status in Japan

Slide 21: Net Sales Trends by Product

Slide 22: Cash Flows (Consolidated)

Slide 23: Handling of subsidies for the construction of the Kobe Science Park Center

Slide 24: Glossary of abbreviations (A-G)

Slide 25: Glossary of abbreviations (H-Z)

Slide 26: Disclaimer Regarding Forward-Looking Statement