Rusfertide Investor Call on Phase 3 PV Data Presented at ASCO 2025

from the beginning

Rusfertide Investor Call on Phase 3 PV Data Presented at ASCO 2025

Important Notice

• Efficacy and Safety Results from the Ph3 Trial of Rusfertide in PV Patients

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

• Market Opportunity

PV patient journey highlights unmet need in current treatment paradigm as patients cycle through options with inconsistent HCT and tolerabilityRusfertide

Rusfertide aims to deliver rapid, consistent & sustained HCT control and is expected to be used at each step of the treatment landscape

Rusfertide has the potential to be a new standard of care in PV based on Ph3 data

Unlocking full potential of rusfertide for patients with PV

Q&A

• Q&A1

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• Q&A6

Medical Presentationas presented at ASCO 2025

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