Jcr Pharmaceuticals Co., Ltd. · TSE:4552
Jcr Pharmaceuticals — Corporate Update (January 2026)
20 slides from Jcr Pharmaceuticals's corporate update, Jan 23, 2026
Slide 1: Briefing Session on the Agreement with Italfarmaco
Life is Rare Briefing Session on the Agreement with Italfarmaco January 23, 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: Agreement with Italfarmaco (Press Release on Dec 24, 2025)
Agreement with Italfarmaco (Press Release on Dec 24, 2025) 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 opportunitie
Slide 4: In-licensed asset: Givinostat
Life is Rare In-licensed asset: Givinostat
Slide 5: Overview: Duchenne Muscular Dystrophy (DMD)
Overview: Duchenne Muscular Dystrophy (DMD) Life is Rare DMD, duchenne muscular dystrophy Desmet T, et al., Front Pharmacol. 2025; 16: 1662586. 4
Slide 6: Overview: Duchenne Muscular Dystrophy (DMD)
Overview: Duchenne Muscular Dystrophy (DMD) Life is Rare 1 Etiology1 • Caused by mutations in the dystrophin gene, leading to the absence of dystrophin protein beneath the muscle fiber membrane 2 Patient population2 • approx. 3,500 (Japan) 3 Clinical course1 • Onset at 3–5 years of age • Progressive
Slide 7: Current status of DMD treatment
Current status of DMD treatment Life is Rare Approval status Note1 Japan US EU ・Negative for anti-AAVrh74 antibodies Gene therapy ✓3 ✓ ・Ambulatory patients ・3years to less than 8 years ・Patients with specific genetic Exon skipping ✓3 ✓ mutations2 Steroid ✓ ✓ ✓ ー Approaches DMD through a distinct mec
Slide 8: 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 9: 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 10: 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 11: EPIDYS: Phase 3 Study Design
EPIDYS: Phase 3 Study Design1-3 Life is Rare • Randomized, double-blind, parallel-group, placebo-controlled study with a total of 179 ambulant boys randomized 2:1 (givinostat:placebo) • Givinostat or placebo were administered in addition to corticosteroids Givinostat oral plus corticosteroids Givino
Slide 12: EPIDYS Study: 4SC From Baseline to Week 72
EPIDYS Study: 4SC From Baseline to Week 721 Life is Rare Mean change from baseline over 72 weeks in the 4SC test* 6.0 Mean change from baseline in time to Givinostat 5.0 Placebo 4.0 perform 4SC (s) 3.0 2.0 1.0 0.0 -1.0 0 12 24 36 48 60 72 Time (weeks) Mean change from baseline (95% CI), s Givinostat
Slide 13: 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 14: 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 15: Commercial Potential of Givinostat for DMD Patients in Japan
Commercial Potential of Givinostat for the Treatment of DMD Patients in Japan Life is Rare 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 DMD1 • Over 1,000 subjects meet eligibility
Slide 16: JCR's Competitive Strengths in Pediatric and Rare Disease Areas
JCR’s Competitive Strengths in Pediatric and Rare Disease Areas Life is Rare Rare disease IZCARGOTM • Pediatric portfolio advantage metabolism - Established presence in pediatrics neurology GROWJECTTM Givinostat - >60% coverage of DMD-treating institutions with existing products endocrinology (inter
Slide 17: Key Takeaways
Key Takeaways Life is Rare Givinostat: Oral non-steroidal treatment for DMD 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 EU • Clinical evi
Slide 18: Life is Rare
Ou, ANNIVERSAR’ JVC Life is Rare
Slide 19: Appendix
Pharmaceuticals Appendix
Slide 20: 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
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