Jcr Pharmaceuticals Co., Ltd. · TSE:4552
Jcr Pharmaceuticals — J.P. Morgan Healthcare Conference 2026
18 slides from Jcr Pharmaceuticals's conference presentation, Jan 16, 2026
Slide 1: JP Morgan Healthcare Conference 2026
Life is Rare JP Morgan Healthcare Conference 2026 JCR Pharmaceuticals Co., Ltd. Shin Ashida Chairman, President, and CEO Hiroyuki Sonoda, Ph.D. Director, Senior Managing Executive Officer
Slide 2: Shin Ashida (Founder) and Hiroyuki Sonoda
Life is Rare Shin Ashida (Founder) Hiroyuki Sonoda Representative Director, Chairman, President and CEO Representative Director, incoming President (as of Apr 1, 2026) 1
Slide 3: 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 4: 50 Years of JCR Pharma: A Journey of Growth and Innovation
50 Years of JCR Pharma: A Journey of Growth and Innovation JCR Europe JCR Europe Clin. Dev., Clin ops, regulatory affairs Head Office Kobe API Plant Bio Research Ctr. Murotani Plant JCR Lux JCR Luxembourg Packaging, labeling, distribution (planned) Seishin Plant Kobe Plant Research Institute Kobe Sc
Slide 5: JCR is Fully Integrated Bio-Pharma Company from Research to Commercialization
JCR is Fully Integrated Bio-Pharma Company from Research to Commercialization Revenue Stream from a Diversified Domestic Portfolio Percentage of Total Sales (FY2024) Allocation of FTEs (FY2024) Agalsidase Beta BS Japan Employees Others Epoetin Alfa BS 938 (Mar. 31, 2025) G&A R&D Pipeline 3 5 Female
Slide 6: JCR Developed the World's First Commercialized Brain Shuttle Medicine
JCR Developed the World’s First Commercialized Brain Shuttle Medicine 2026 Neuro Neuro 2021 Degeneration inflammation Pabinafusp alfa (IZCARGOTM) was approved Neuro Neuro-muscular in Japan for the treatment of MPS II. Oncology Diseases Therapeutic mAb Gene therapy delivery Platform delivery to the C
Slide 7: An Industry-Leading LSD Portfolio based on the J-Brain Cargo Technology
An Industry-Leading LSD Portfolio based on the J-Brain Cargo Technology Portfolio (preclinical and clinical) To all patients around the world MPS II Marketed JR-141 (Hunter) Fabry* Approved (JP) MPS II JR-141 (Hunter) Phase III (global) Clinical MPS I MPS IIIA MPS IIIB JR-171 JR-441 JR-446 Phase I/I
Slide 8: 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 • 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 on track. • Three-year
Slide 9: Partnerships are at the Core of JCR's Business Philosophy
Partnerships are at the Core of JCR’s Business Philosophy JBC partnership with Alexion (Oligonucleotides) Givinostat partnership (Japan) Gene therapy partnership Strategic partnership with Takeda (until 2024) JBC partnership with Gene therapy capsid partnership Alexion (Neuro) with Alexion Approval
Slide 10: JCR's Growth Strategy
JCR’s Growth Strategy Key initiatives for sustained company growth 1. Advance internal portfolio in LSDs and GH deficiency (JR-141, JR-441, JR-446, JR-142, others) 2. Expand international footprint through platform, portfolio and strategic partnerships (e.g. Italfarmaco) 3. Strengthen domestic portf
Slide 11: A Strategic Partnership between JCR and Italfarmaco
A Strategic Partnership between JCR and Italfarmaco Family-owned company Strong Founder engagement EUUS focus; presence in 90 countries Japan footprint Proprietary mall molecule product Biologics product portfolio portfolio Multiple Biotherapeutics platforms Potential future expansion of the partner
Slide 12: Givinostat Partnership with Italfarmaco
Givinostat Partnership with Italfarmaco Since its first approval in 2024, Givinostat has been administered to thousands of individuals diagnosed with Duchenne’s muscular dystrophy (DMD). In December 2025, JCR has acquired the development and commercialization rights for givinostat for Japan. Givinos
Slide 13: Commercial Potential of Givinostat for the Treatment of DMD Patients in Japan
Commercial Potential of Givinostat for the Treatment of DMD Patients in Japan Duchenne muscular dystrophy is an indication with high unmet medical need and a significant patient population in Japan • ~3,500 individuals in Japan diagnosed with DMD • Over 1,000 subjects meet eligibility criteria per E
Slide 14: JUST-AAV: Unlocking New Frontiers in Gene Therapy
JUST-AAV: Unlocking New Frontiers in Gene Therapy A novel AAV platform for targeted transgene delivery to the brain and other tissues and organs Creation of "tags" with high tropism to other organs Adeno-Associated Insertion of de novo 107 VG copies/ug liver DNA Virus (AAV9) mutations in capsid 106
Slide 15: Engineering Directionality to Desired Tissues and Organs
Engineering Directionality to Desired Tissues and Organs Transgene mRNA expression (normalized to AAV9) Quantitation of transduced cells1 Parietal cortex Frontal cortex Putamen 120 x114.8 2.0% 0.8% 0.5% CNS Delivery AAV9 100 x76.9 80 x62.5 x65.8 x34.8 x36.5 60 x44.6 x22.1 91.0% JUST-AAV 40 42.0% 51.
Slide 16: The First International Partnership on the JUST-AAV Platform
The First International Partnership on the JUST-AAV Platform July 2025 License agreement with Alexion for JUST-AAV capsids • Alexion may use the licensed capsids, which are part of the JUST-AAV platform, in up to five of Alexion's genomic medicine programs • Milestone payments of up to USD 825 milli
Slide 17: Shaping the Future with JCR's Proprietary Technologies
Shaping the Future with JCR’s Proprietary Technologies JBC and JUST-AAV enable multi-modal approaches and expand Neurodegeneration opportunities for out-licensing, driving long-term corporate value Muscular Diseases JUST-AAV Rare Diseases J-Brain Cargo ･･･ Neuroinflammation Neuro-oncology ･･･ AAV wi
Slide 18: Life is Rare
SO u, ANNIVERSARY ACA Life is Rare Pharmaceuticals
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