Rusfertide 米国臨床腫瘍学会（ASCO）2025にて発表された真性多血症を対象とした臨床第3相試験データに関する投資家向けコール

最初から再生

Rusfertide 米国臨床腫瘍学会（ASCO）2025にて発表された真性多血症を対象とした臨床第3相試験データに関する投資家向けコール

重要な注意事項

• 真性多血症を対象とした臨床第3相試験におけるRusfertideの有効性および安全性の結果
  

Key Takeaway Points/Conclusions

Background

Polycythemia Vera and the Role of Iron and Hepcidin in Red Blood Cell Production

Rusfertide in Polycythemia Vera (PV)

Phase 3 VERIFY Study (NCT05210790) Design in PV

Phase 3 VERIFY Study (NCT05210790) in PV Prespecified Primary and Key Secondary Endpoints

Baseline Demographics and Disease Characteristics

Concurrent Cytoreductive Therapy During Part 1a

VERIFY Study Met Its Primary Endpoint During Weeks 20-32 (Part 1a)

Rusfertide + CSC Benefit Maintained vs. Placebo + CSC for Response* Across Subgroups, Including Risk Status and Concurrent Therapy

Rusfertide + CSC Reduced the Mean Number of PHL From Weeks 0-32 vs Placebo + CSC (p<0.0001): Key Secondary Endpoint #1

Rusfertide + CSC More Likely to Maintain Hct <45% From Weeks 0-32 vs Placebo + CSC: Key Secondary Endpoint #2

Rusfertide Demonstrated an Improvement in the PROMIS Fatigue SF-8a Total T-Score at Week 32 vs. Placebo: Key Secondary Endpoint #3

Rusfertide Demonstrated an Improvement in the MFSAF TSS7 at Week 32 vs. Placebo: Key Secondary Endpoint #4

Exposure and Treatment-Emergent Adverse Events (Part 1a)*

Cancer Events and Serious TEAEs (Part 1a)*

Conclusions

• 市場機会
  

真性多血症（PV）におけるペイシェント・ジャーニー：不安定なヘマトクリット（HCT）や忍容性が課題となり、患者さんが治療法を次々と変更せざるを得ない既存の治療体系にアンメット・ニーズが存在

Rusfertideは迅速、安定的かつ持続的なヘマトクリット（HCT）のコントロールを実現し、治療の各段階で使用される薬剤になると想定

臨床第3相試験のデータに基づきRusfertideは真性多血症（PV）の新たな標準治療となる可能性

真性多血症患者さんのために、Rusfertideの可能性を最大限に引き出す

質疑応答

• 質疑応答1

• 質疑応答2

• 質疑応答3

• 質疑応答4

• 質疑応答5

• 質疑応答6

ASCO 2025で発表したメディカルプレゼンテーション

Results From VERIFY, a Phase 3, Double-Blind, Placebo (PBO)-Controlled Study of Rusfertide for Treatment of Polycythemia Vera (PV)

Key Takeaway Points/Conclusions

Background

Polycythemia Vera and the Role of Iron and Hepcidin in Red Blood Cell Production

Rusfertide in Polycythemia Vera (PV)

Phase 3 VERIFY Study (NCT05210790) Design in PV

Phase 3 VERIFY Study (NCT05210790) in PV Prespecified Primary and Key Secondary Endpoints

VERIFY Patient Disposition and Analysis Sets: Part 1a

Baseline Demographics and Disease Characteristics

Concurrent Cytoreductive Therapy During Part 1a

VERIFY Study Met Its Primary Endpoint During Weeks 20-32 (Part 1a)

Rusfertide + CSC Benefit Maintained vs. Placebo + CSC for Response* Across Subgroups, Including Risk Status and Concurrent Therapy

Rusfertide + CSC Reduced the Mean Number of PHL From Weeks 0-32 vs Placebo + CSC (p<0.0001): Key Secondary Endpoint #1

Rusfertide + CSC More Likely to Maintain Hct <45% From Weeks 0-32 vs Placebo + CSC: Key Secondary Endpoint #2

Rusfertide Demonstrated an Improvement in the PROMIS Fatigue SF-8a Total T-Score at Week 32 vs. Placebo: Key Secondary Endpoint #3

Rusfertide Demonstrated an Improvement in the MFSAF TSS7 at Week 32 vs. Placebo: Key Secondary Endpoint #4

Exposure and Treatment-Emergent Adverse Events (Part 1a)*

Cancer Events and Serious TEAEs (Part 1a)*

Limitations

Conclusions

We would like to thank all patients and their caregivers who participated in this study along with all investigators, study staff, and clinical trial sites who contributed to VERIFY

Patient Lay Summary Slide