Jcr Pharmaceuticals Co., Ltd. · TSE:4552
Jcr Pharmaceuticals — Q3 FY2025 Earnings Presentation
28 slides from Jcr Pharmaceuticals's earnings presentation, Jan 28, 2026
Slide 1: FY2025 Third Quarter Results Briefing Session
Life is Rare FY2025 Third Quarter Results Briefing Session January 28, 2026 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 Third Quarter Financial Results
Life is Rare FY2025 Third 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 Q3 YTD Q3 YTD Rate • Net Sales increased, driven by higher Difference Ratio upfront and milestone payments from Net Sales 25,880 30,353 +4,472 +17.3% 76.8% lic
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 Q3 YTD Q3 YTD Rate Difference Ratio • GROWJECTTM, IZCARGOTM, and GROWJECTTM 14,177 13,539 (638) (4.5)% 76.1% TEMCELLTM HS Inj. continued steady performance. IZCA
Slide 6: Revision of FY2025 Consolidated Financial Forecast
Revision of FY2025 Consolidated Financial Forecast Life is Rare Profit Attributable to Net Sales Operating Profit Ordinary Profit Earnings Per Share (Unit: million yen) (Unit: million yen) (Unit: million yen) Owners of Parent (Unit: yen) (Unit: million yen) Previously announced 37,800 2,600 2,400 3,
Slide 7: Revision of FY2025 Consolidated Financial Forecast
Revision of FY2025 Consolidated Financial Forecast Life is Rare (Unit: million yen) Previously Reference: Revised forecasts Change Change(%) announced forecasts FY2024 Results GROWJECTTM 17,800 17,800 ― ― 18,098 IZCARGOTM* 6,400 6,400 ― ― 5,718 TEMCELLTMHS Inj. 2,700 2,700 ― ― 2,904 Treatments for r
Slide 8: Agreement with Italfarmaco (Press Release in Dec, 2025)
Agreement with Italfarmaco (Press Release in Dec, 2025)1 Life is Rare • Development and Commercialization of Givinostat for DMD - Exclusive licensing agreement (Japan) • Strategic collaboration for the treatment of rare diseases - Enhancing both companies’ portfolios - Exploring joint opportunities
Slide 9: Givinostat: Oral Non-Steroidal Treatment for DMD
Givinostat: Oral Non-Steroidal Treatment for DMD Life is Rare 1 Distinct mechanism of action from other DMD therapies • HDAC inhibitor with mutation-agnostic mechanism of action 2 Regulatory approvals outside Japan • Approved in major markets, including the US and the EU1 • Clinical evidence demonst
Slide 10: 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 on track
Slide 11: Life is Rare
Confidential = O th ANNIVERSARY 2 JCR Life is Rare 7 ~ Pharmaceuticals
Slide 12: Appendix
JVCR Life is Rare ANNIVERSARY Appendix
Slide 13: GROWJECT Market Share Trends in Japan (Quarterly)
GROWJECTTM Market Share Trends in Japan (Quarterly) Life is Rare ■ Human Growth Hormone Market in Japan GH Market Share in Japan （FY2024 Q1～FY2025 Q3）*On an NHI drug price basis General Practitioner(GP) 45.0% Hospital(HP) 41.6% 41.8% 31.4% 41.2% 41.2% 42.9% 40.3% 39.8% 39.0% 40.0% 35.5% 36.1% 34.6%
Slide 14: GROWJECT Sales Trends in Japan (Quarterly)
GROWJECTTM Sales Trends in Japan (Quarterly) Life is Rare GH Sales Trends in Japan（FY2024 Q1～FY2025 Q3）*On an NHI drug price basis 7,000 16,500 GH Market in Japan （million yen） Sales amount of each product（million yen） 6,000 16,000 5,000 15,500 GH Market in Japan TM JCR GrowjectTM Daily 4,000 Produc
Slide 15: GROWJECT Trends of Market Share in Japan
GROWJECTTM Trends of Market Share in Japan Life is Rare (billion yen) Weekly Sales 7.0 7,000,000,000 45.0% Daily Sales （ except GROWJECTTM ） GROWJECTTM Sales 40.0% 6.0 6,000,000,000 Daily Shares 35.0% （ except GROWJECTTM ） 5.0 5,000,000,000 GROWJECTTM Shares 30.0% Weekly Shares 4.0 4,000,000,000 25.
Slide 16: IZCARGO Prescription Status in Japan
IZCARGOTM 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) 2,000 120 109 106 106 1,800 100 96 90 92 92 100 1,600 88 1,400 80 85 84 86 1,200 78 80 74 75 75 72 1,000 60 800 40 600 400 20 20
Slide 17: Partnerships are at the Core of JCR's Business Philosophy
Partnerships are at the Core of JCR’s Business Philosophy Life is Rare JBC partnership with Alexion (Oligonucleotides) Givinostat partnership (Japan) Gene therapy partnership Strategic partnership with Takeda (until 2024) JBC partnership with Gene therapy partnership Approval of IZCARGOTM Alexion (N
Slide 18: Italfarmaco S.p.A
Italfarmaco S.p.A Life is Rare Italfarmaco S.p.A • Private global pharmaceutical company Employees Business founded in 1938 (Milan, Italy) >4000 >90 contries • Development, manufacturing, marketing and sales of branded prescription & non- prescription products • Proven success in many therapeutic ar
Slide 19: 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 20: 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 CargoTM with Acumen • To develop blood-brain barrier-penetrating treatment for Alzheimer’s disease - Combines JCR’s J-Brain CargoTM with Acumen’s AβO-selective
Slide 21: Regenerative CDMO Subsidy
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 22: 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 23: Givinostat: Non-Steroidal Treatment for DMD
Givinostat: Non-Steroidal Treatment for DMD Life is Rare 1 INN: Givinostat (Brand name: Duvyzat) • Histone deacetylase (HDAC) inhibitor • Oral, non-steroidal therapy (twice daily dosing) 2 Overseas indication • DMD patients ≥6 years of age (EU: Ambulatory patients ≥6 years on concomitant steroid the
Slide 24: Role of HDACs in Regulating Muscle Homeostasis
Role of HDACs in Regulating Muscle Homeostasis Life is Rare HDACs help mediate muscle homeostasis via cytoplasmic and nuclear activity1,2 Cytoplasm3 Nucleus3 HDAC Nonhistone proteins HDAC HAT Nucleus closes chromatin, opens chromatin, repressing gene allowing gene transcription transcription RNA pol
Slide 25: HDAC Inhibition Targets the Pathophysiology in DMD
HDAC Inhibition Targets the Pathophysiology in DMD1,2 Life is Rare HDAC inhibition Counteracts the Pathological Events in DMD1,3 Restoration of motor function4-8 Reduced activation of immune cells4,6,7 Increased muscle repair and regeneration4,6,7,9 HDAC inhibition Reduced fibrogenesis and adipogene
Slide 26: EPIDYS Study: Primary Endpoint—4SC at Week 72
EPIDYS Study: Primary Endpoint—4SC at Week 721 Life is Rare Mean change from baseline in time to perform the 4SC test at 72 weeks (non-log transformed) Δ = 1.78 s ▪ At week 72, givinostat plus 5.0 P=0.037* corticosteroids reduced the 4.5 GLS mean time to perform decline in time to perform the 4.0 4S
Slide 27: Givinostat and Permanent Loss of Ambulation
Givinostat and Permanent Loss of Ambulation1,2 Life is Rare ▪ Patients receiving givinostat plus corticosteroids (SoC) preserved their ability to walk for an additional 2.9 years (HR, 0.42; 95% CI, 0.23-0.76; P=0.004) compared with patients receiving SoC alone Givinostat Control Parameter (n=142) (n
Slide 28: 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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